Silicon Investor - Sangamo Therapeutics, Inc. SGMO

[scaram(o)uche] Sold 75% of that position soon after taking it, modest gain. Sold remainder thi...

scaram(o)uche — 2/7/2019 1:19:40 PM

Sold 75% of that position soon after taking it, modest gain. Sold remainder this morning, making every mistake. Saw pre-market action, got anxious. Didn't sell. Then read PRs after they came out, not realizing that Feuerstein's embargoed story was immediately available. First try to sell was into the first trading halt. Embarrassing. Second attempt, considered a market sell, but got greedy and set a limit. Didn't hit, and whammy, second trading halt. Embarrassing X2. Finally selected "market", and someone got caught out with a decent bid.

Took a loss, all shares considered. That's not as bad as the fashion in which I took it, and that after investing in a management team in which I'd lost confidence.

Fail.

[scaram(o)uche] Looked like the final stages of a bail, to me, this afternoon. Took on a few sh...

scaram(o)uche — 5/9/2018 1:57:45 PM

Looked like the final stages of a bail, to me, this afternoon. Took on a few shares.

[more100] $SGMO closed up 21.71 percent on Thursday, September 10, 2015, on 7.27 times nor...

more100 — 9/11/2015 7:29:53 AM

$SGMO closed up 21.71 percent on Thursday, September 10, 2015, on 7.27 times normal volume. It ran into resistance at its 50 day moving average.

[jim heger] Good buying op today. It hardly matters that the platform may not work as adver...

jim heger — 4/28/2015 12:42:32 PM

Good buying op today. It hardly matters that the platform may not work as advertised. Barton Biggs once said about the uncertainty of investing in experimental bios, "No one knows what those guys are doing."
Lanphier has shown, if nothing else, that he can sell stock.

[PLegee] Another huge step forward for Sangamo, bigger than the Shire deal in early 2012....

PLegee — 1/9/2014 4:06:56 PM

Another huge step forward for Sangamo, bigger than the Shire deal in early 2012. A first tier biotech company has just validated their platform by partnering up with them and allowing a share of royalties for any market successful outcomes. Huge. Congrats Sangamo and congrats to all you long time shareholders. More monogenetic disease drug partnerships look to be possible after this huge win for SGMO. Plus, we have strong leadership on the BOD with Ringo et all; missteps are behind us, the future looks so promising.

[nigel bates] CAMBRIDGE, Mass. and RICHMOND, Calif., Jan. 9, 2014 /PRNewswire/ -- Biogen Idec ...

nigel bates — 1/9/2014 8:18:42 AM

CAMBRIDGE, Mass. and RICHMOND, Calif., Jan. 9, 2014 /PRNewswire/ -- Biogen Idec (BIIB) and Sangamo BioSciences, Inc. (SGMO) announced today an exclusive worldwide collaboration and license agreement focused on the development of therapeutics for hemoglobinopathies, inherited conditions that result from the abnormal structure or underproduction of hemoglobin. The agreement will enable Biogen Idec to further enhance its expertise in non-malignant hematology by leveraging Sangamo's proprietary genome-editing technology platform to develop treatments targeting sickle cell disease (SCD) and beta-thalassemia.

"Our collaboration with Sangamo is expected to help us expand our capabilities to develop treatments for people with serious, inherited hematologic conditions," said Douglas E. Williams, Ph.D., Biogen Idec's executive vice president of research and development. "Building upon emerging science related to fetal hemoglobin regulation, we intend to develop Sangamo's novel gene-editing technology to create a single approach that has the potential to functionally cure both sickle cell disease and beta-thalassemia."

Sangamo's proprietary zinc finger nuclease (ZFN) genome-editing technology enables multiple pathways to treat SCD and beta-thalassemia. The technology can be used to precisely target and knock out key regulators of gene expression, or can be used to precisely insert a new corrective gene to replace the defective copy.

"We are delighted to partner our hemoglobinopathies programs with Biogen Idec," said Edward Lanphier, Sangamo's president and chief executive officer. "Biogen Idec is a leader in drug development and has a history of successfully translating cutting edge science into treatments that provide life-changing clinical benefit for patients. This alliance is further validation of our ZFP platform as a transformative technology and accelerates our goal of developing a novel class of therapeutics which has the potential to revolutionize the treatment of genetic diseases."

Under the terms of the agreement, Sangamo is responsible for all research and development activities through the first clinical proof of concept trial in beta-thalassemia, and both companies will perform activities to enable submission of an Investigational New Drug (IND) application for SCD. Biogen Idec will be responsible for subsequent worldwide clinical development and commercialization of products arising from the alliance. Sangamo retains an option to co-promote any licensed product to treat SCD and beta-thalassemia in the United States.

Biogen Idec will provide Sangamo with an upfront payment of $20 million and will reimburse Sangamo for its internal and external research and development program-related costs. Sangamo may also receive additional payments of approximately $300 million based on the achievement of certain development, regulatory, commercialization and sales milestones, as well as double digit royalties on product sales.

[nigel bates] RICHMOND, Calif., Dec. 10, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. ( SGMO...

nigel bates — 12/10/2013 8:59:08 AM

RICHMOND, Calif., Dec. 10, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. ( SGMO) announced the presentation of new pre-clinical data demonstrating therapeutic levels of gene modification in non-human primates (NHPs) from its In Vivo Protein Replacement Platform. Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the platform enables the permanent production of therapeutic proteins from a specific genomic site in the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases such as hemophilia and lysosomal storage disorders (LSD) including Gaucher and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life. The data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) which is being held in New Orleans.

"These data provide proof of concept for this broadly applicable genome editing strategy by demonstrating that our process is scalable to large animals and by validating the use of the albumin safe harbor as a site for expression of therapeutic proteins," said Philip Gregory. D. Phil., Sangamo's vice president of research and chief scientific officer. "We have further optimized Sangamo's ZFN system and demonstrated that a single systemic treatment enables stable liver-specific production of replacement protein. Early data in primates suggest that we can achieve circulating levels of protein above the threshold for therapeutic effect, which we believe are sufficient for the correction of a range of monogenic diseases. Our data demonstrate expression of replacement enzymes for multiple different proteins, including those deficient in lysosomal storage disorders, which serves to demonstrate the potential of this approach for a broad range of other monogenic diseases."

Sangamo's In Vivo Protein Replacement Platform makes use of a highly expressed and liver specific genomic "safe-harbor site" that can be edited with ZFP nucleases (ZFNs) to accept and express any therapeutic gene and thus permanently produce high levels of the missing protein with a single treatment. The gene encoding albumin, the most abundant protein in blood serum, was chosen as a safe harbor site because it is active exclusively in the liver. The albumin promoter is highly active, continuously producing large amounts of albumin protein (approximately 15g/day) which is in excess of the body's requirements. With such a large capacity for protein production, targeting and co-opting a very small percentage of the albumin gene's production capacity is sufficient to safely produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production. The study was performed as part of Sangamo's collaboration with Shire and in the laboratory of Katherine A. High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia. Dr. High is a Howard Hughes Medical Institute Investigator, and Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

"These data represent an important milestone in the progress of our monogenic disease programs towards the clinic," stated Edward Lanphier, Sangamo's president and chief executive officer. "Our In Vivo Protein Replacement Platform is a highly leverageable and disruptive application of our ZFN-mediated genome editing technology and can potentially be applied to any disease-relevant gene where enabling the liver to provide a stable source of corrective replacement protein will be therapeutic. The NHP data presented at ASH represent a significant de-risking step and provide important proof of concept and validation for this entire strategy."

The data were presented in an oral presentation:

Abst. #720 ZFN Mediated Targeting of Albumin "Safe Harbor" Results in Therapeutic Levels of Human Factor VIII in a Mouse Model of Hemophilia A
Session: 801
Monday, December 9, 2013: 5:45 PM

Scientists demonstrated efficient ZFN-mediated gene addition into the albumin locus in adult mouse models of hemophilia A and B with a single systemic administration of ZFNs and a donor DNA sequence encoding a corrective human Factor VIII or IX gene, respectively. This permanent correction resulted in the production of robust, stable levels of circulating active Factor VIII and Factor IX protein that normalize clotting times in hemophilic mice.

Data were presented demonstrating the broad utility of this method for the potential treatment of a wide range of monogenic diseases. In these additional studies, the albumin-specific ZFN strategy was used in combination with appropriately designed donor DNA sequences encoding a diverse range of protein factors including alpha-galactosidase which is deficient in Fabry disease, beta-glucosidase (Gaucher disease), Iduronate-2 Sulfatase (Hunter disease) and alpha-L-Iduronidase (Hurler disease).

The feasibility and scalability of the approach was also evaluated in NHPs (Rhesus monkeys), an important large animal model that has been previously used in the development of novel gene-based therapies for monogenic diseases such as hemophilia. These NHP data revealed specific ZFN-mediated modification of the albumin locus by deep-sequencing analysis of liver biopsy samples from treated animals. There were no significant alterations in circulating albumin levels. Analysis of T-cells isolated from both spleen and lymph node, demonstrated that the animals did not mount an immune response to either the ZFNs or the vector used for delivery. Importantly, ZFN-mediated modification of the albumin locus in NHPs resulted in levels of modification which are sufficient to provide a therapeutic effect. In a program update, early experiments demonstrate expression of the encoded therapeutic protein at levels that were up to three-fold the amount required for therapeutic effect.

[nigel bates] Seemingly have pulled off the trick of a fundraising followed by a rise in the s...

nigel bates — 9/18/2013 2:39:04 PM

Seemingly have pulled off the trick of a fundraising followed by a rise in the stock price...

RICHMOND, Calif., Sept. 18, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. ( SGMO) announced today the sale of 6,100,000 shares of its common stock in an underwritten public offering at a price to the public of $10.58 per share, the closing price of the stock on the previous day. The gross proceeds to Sangamo from the public offering are expected to be approximately $65 million, before deducting underwriting discounts and commissions and other estimated offering expenses. In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 915,000 shares of common stock on the same terms and conditions, to cover over-allotments, if any. The offering is expected to close on or about September 23, 2013, subject to customary closing conditions....

[nigel bates] Yet another technique... pipeline.corante.com

nigel bates — 9/6/2013 7:41:57 AM

[DewDiligence_on_SI] Maybe it's about issuing a PR just for the sake of "news flow." I'm highly skept...

DewDiligence_on_SI — 8/27/2013 1:22:31 PM

Maybe it's about issuing a PR just for the sake of "news flow." I'm highly skeptical of SGMO, as you may have guessed.

[nigel bates] Who knows ? At least it's rather cheap.

nigel bates — 8/27/2013 12:31:28 PM

[mopgcw] it has been interesting to follow the discussion on whether this move is about z...

mopgcw — 8/27/2013 12:00:05 PM

it has been interesting to follow the discussion on whether this move is about zfp not working and sgmo getting an alternative or about solving the delivery problem via this acquisition.

[nigel bates] RICHMOND, Calif., Aug. 26, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (SGMO)...

nigel bates — 8/27/2013 11:32:22 AM

RICHMOND, Calif., Aug. 26, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (SGMO) announced that it signed a definitive agreement to acquire Ceregene, Inc., a privately held biotechnology company focused on developing adeno-associated virus (AAV) gene therapies. Sangamo will host a teleconference at 8:30 am ET tomorrow, Tuesday, August 27, 2013, to discuss the acquisition and provide a general business overview.

"Ceregene is a leader in development and manufacturing of AAV-based therapies with significant clinical development experience," said Edward Lanphier, Sangamo's president and CEO. "Since their inception in 2001, the company has safely treated over 115 subjects in four clinical trials. Sangamo has acquired all of Ceregene's AAV assets including CERE-110, AAV delivery of nerve growth factor (NGF) to the brain for the treatment of Alzheimer's disease. CERE-110 is being evaluated in a fully enrolled and fully funded Phase 2 clinical trial. In addition to the AAV platform, the assets also include one of the world's largest databases of AAV GMP manufacturing know-how, toxicology data, and safety data from their human clinical trials, which will be an invaluable resource as we advance our ZFP Therapeutics."

Under the terms of the definitive agreement Sangamo will issue to the stockholders of Ceregene 100,000 shares of Sangamo's common stock, which represents less than 0.2 percent of Sangamo's total shares outstanding. In addition, Sangamo has agreed to make contingent earn-out payments to the stockholders of Ceregene based upon revenues generated from license or sales transaction of certain existing products of Ceregene. The acquisition is expected to close in September 2013, subject to customary closing conditions. The Company does not expect the acquisition, including the ongoing Phase 2 clinical trial, to have any impact on its financial guidance regarding 2013 operating expenses or yearend cash. A more detailed description of the terms of the definitive agreement is available in the Form 8-K filed by the Company with the Securities and Exchange Commission on this date.

Sangamo will receive over 120 issued, pending or in-licensed patents that include patent families covering the AAV vector platform and manufacturing methods, therapeutic transgenes, and technology for direct administration of AAV to the brain. Sangamo will also have access to GMP master cell banks, materials and manufacturing know-how that will expand its capabilities in AAV manufacturing as well as a database of preclinical efficacy and toxicology studies and other documentation supporting Ceregene's Investigational New Drug (IND) applications. These materials provide valuable reference materials for Sangamo in the preparation and filing of IND applications for its in vivo ZFP Therapeutics®, particularly those that target the brain. In addition, Sangamo will acquire all of Ceregene's preclinical and clinical therapeutic programs including its ongoing double-blind, placebo-controlled Phase 2 trial to evaluate its NGF-AAV (CERE-110) in Alzheimer's disease (AD) and the proprietary needle device for brain delivery of AAV with supporting regulatory documentation and clinical experience.

In 2008, Ceregene, and its collaborators at the Alzheimer's Disease Cooperative Study (ADCS), based at the University of California, San Diego (UCSD), were awarded a $5.4 million grant from the National Institute of Aging of the National Institutes of Health (NIH) to support the double-blind, placebo-controlled, Phase 2 clinical trial of CERE-110 in 50 subjects with mild to moderate AD. Using AAV to produce a steady supply of NGF in a specific area of the brain, the treatment is designed to address the loss of cholinergic neurons which is associated with memory loss and cognitive decline in AD. The trial is fully enrolled and the subjects, half of whom were treated with CERE-110 and half with sham surgery (placebo), are in the follow-up stage of the study which will evaluate the effect of treatment on established clinical end-points in cognitive function and quality of life. The results of this trial are expected in 2015. ...

[nigel bates] RICHMOND, Calif., May 20, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. ( SGMO)...

nigel bates — 5/20/2013 7:28:31 AM

RICHMOND, Calif., May 20, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. ( SGMO) announced the presentation of new data demonstrating the successful application of Sangamo's proprietary In Vivo Protein Replacement Platform (IVPRP) to produce therapeutically relevant levels of Factor VIII in a mouse model. Sangamo has partnered with Shire AG (LSE: SHP, NASDAQ: SHPG) to develop ZFP Therapeutics for both hemophilia A and B using this approach. The data were presented at the 16th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) which was held in Salt Lake City from May 15-18, 2013.

"Our IVPRP provides a potentially curative therapeutic solution for hemophilia A – a monogenic disease caused by the absence of the coagulation protein, Factor VIII," said Philip Gregory. D. Phil., Sangamo's vice president of research and CSO. "These data provide further proof-of-concept for the broad application of our IVPRP strategy for monogenic diseases currently treated using protein replacement. Our data demonstrate that a single systemic treatment results in the stable production of therapeutically relevant levels of functional Factor VIII."

Hemophilia A, the most common form of the bleeding disorder, is caused by a genetic defect in the gene encoding Factor VIII, a blood clotting factor. Sangamo's IVPRP approach enables the precise insertion of a replacement Factor VIII gene into the genome at what is known as a "safe harbor site." Specifically, Sangamo's IVPRP uses the albumin gene as the site of insertion ensuring that Factor VIII will be highly expressed at stable levels exclusively in the liver.

Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the IVPRP enables the permanent production of therapeutic proteins from the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases including hemophilia and lysosomal storage disorders (LSD) such as Gaucher and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life.

Data were presented on the production of Factors VIII and IX for the correction of hemophilia A and B, as well as four enzymes absent in a panel of LSDs by Sangamo's collaborators from the laboratory of Katherine A. High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, and Investigator, Howard Hughes Medical Institute. The abstract for this presentation, entitled "In Vivo ZFN Mediated Targeting of Albumin as a Platform for Expression of Multiple Therapeutic Genes," was chosen as one of the six Excellence in Research Awards presented at the meeting.

"The ASGCT meeting provides a great forum to highlight our latest data demonstrating the potential of our ZFP platform to provide novel therapeutic solutions to a broad range of diseases," said Edward Lanphier, Sangamo's president and CEO. "Our presentations included data from clinical and preclinical programs to develop ZFP Therapeutics for indications including HIV/AIDS, hemophilia A and B, lysosomal storage disorders and other monogenic diseases, and cancer.

In one of the first sessions of the ASGCT meeting, we presented data from our SB-728-T clinical trials in HIV-infected subjects demonstrating unprecedented increases in total CD4 T-cells and reduction in HIV reservoir as measured by proviral DNA, and encouraging preliminary data from our ongoing studies. Data demonstrating the successful production of Factor VIII demonstrates the unique qualities of our IVPRP approach to deliver potentially curative solutions for many monogenic diseases. We look forward to presenting further data from these programs and others in our ZFP Therapeutic pipeline at scientific and medical meetings throughout the year."

A total of thirteen data presentations were made by Sangamo scientists and their collaborators at the 16th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) which was held in Salt Lake City from May 15-18, 2013.

Presentations included data from the following programs and collaborations in the following areas:

HIV/AIDS

Clinical Studies of the Infusion of ZFN CCR5 Modified Autologous CD4 T cells (SB-728T) in HIV Subjects, presented by Dale Ando, M.D. who also chaired the session entitled, Challenges and Success of Gene Therapy Product Approval.Long Term CD4 Reconstitution in HIV Subjects Receiving ZFN CCR5 Modified CD4 T-Cells (SB-728-T) May Be Attributed to the Sustained Durability of the Central Memory T-Cell Subset. (Abstract #58)T-Cells Edited To Express the C34 Peptide from gp41 Heptad Repeat-2 Fused to CCR5 or CXCR4 Exhibit Robust Protection from Diverse HIV-1 Isolates. (Abstract #46)Next Generation TALENs Mediate Efficient Disruption of the CCR5 Gene in Human HSCs. (Abstract # 184)Monogenic Diseases

In Vivo ZFN Mediated Targeting of Albumin as a Platform for Expression of Multiple Therapeutic Genes. (Abstract #488)Allele-Preferred Targeted Correction of CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells. (Abstract # 107)Rescue of T-cell deficiency in Prkdc SCID mice by transplantation of gene-repaired haematopoietic stem cells. (Abstract # 678)Cancer

TCR Gene Editing To Treat Acute Leukemia and Multiple Myeloma. (Abstract #504)Technology Development

Electroporation of ZFN mRNA Enables Efficient CCR5 Gene Disruption in Mobilized Blood Hematopoietic Stem Cells at Clinical Scale. (Abstract #183)Efficient Site-Specific Integration and In Situ Gene Correction of Human Long-Term Repopulating Hematopoietic Stem Cells by Zinc Finger Nucleases. (Abstract #486)Optimizing Plasmid DNA Templates for Homologous Recombination-Mediated Gene Modification Following Zinc Finger Nuclease Treatment of Hematopoietic Stem Cells. (Abstract #487)Enhancing the Efficiency of Zinc Finger Nuclease Delivery by Integrase-Defective Lentiviral Vectors Using Valproic Acid. (Abstract #492)In Vivo Cleavage of Transgene Donors Promotes Nuclease-Mediated Targeted Integration. (Abstract#568)All abstracts for the meeting are available online at 2013 ASGCT Meeting Abstracts

[fred hayes] thanks nigel...

fred hayes — 3/19/2013 11:42:15 AM

[nigel bates] Pretty binary, though, and if the HIV treatment whiffs.... Longer term, here's ...

nigel bates — 3/19/2013 10:21:05 AM

Pretty binary, though, and if the HIV treatment whiffs....

Longer term, here's a potentially (very) competitive platform technology:
forbes.com

[fred hayes] Here's an article by PropThink posted on SA. Easily fooled, I am impressed with...

fred hayes — 1/16/2013 11:09:25 AM

Here's an article by PropThink posted on SA. Easily fooled, I am impressed with the stuff PropThink puts out. Always seems to be comprehensive...

seekingalpha.com

[scaram(o)uche] Thanks for your prompt answer and accompanying thoughts, very considerate! Rick

scaram(o)uche — 1/16/2013 10:15:35 AM

[PLegee] You have it correct, there is no definitive date and/or conference when the data...

PLegee — 1/16/2013 8:54:36 AM

You have it correct, there is no definitive date and/or conference when the data will be made public. In addition, other than the subjective term "update", there is no set expectation as of what or how much detail will actually be disclosed. SGMO is already on record stating that full results will be posted by YE 2013. Since these trials are `unblinded' and undoubtedly SGMO is already evaluating findings in real time, I am hoping that the interim report at a minimum, improves the current probability of a decisive clinical outcome; a true functional cure. Ongoing patient CD4-T levels and detectable viral loads versus number of treatments at a minimum would be nice; hopefully the data presented will be much more robust than that. I dislike the deliberate strategy of keeping we investors guessing, but after the fiasco three years ago on the DN (SB 509) announcement, Ed has since chosen the low bar tactic on setting expectations.

Some are speculating that CROI in March could contain a late entry for an announcement, but others seem to point to conferences in the 2nd quarter as being more likely. The official public company statement form Jan 9th was:

"We expect to present preliminary data from these trials in the first half of 2013 and to have the full data set by the end of 2013."

[scaram(o)uche] I need to listen to the presentation again. ICAAC this year.... was there an...

scaram(o)uche — 1/15/2013 8:50:41 PM

I need to listen to the presentation again. ICAAC this year.... was there any exact language re. what to expect? Sort of sounded like we should expect data, but... is it THE data?

I'll listen again. Thanks, if you know the answer.

Rick

[PLegee] Very positive in all regards, especially "Proof of Concept" of their SB 728T HI...

PLegee — 1/15/2013 8:33:51 PM

Very positive in all regards, especially "Proof of Concept" of their SB 728T HIV clinical trials. Ed stated that additional updates from the trials will be presented in 1st half 2013 . . . . possibly at the upcoming CROI conference in March (as late breaking results). Phase I results released in September of 2012 showed an unprecedented and durable growth in CD4+ T cells and Ed seemed to build upon that at the JPM conference. They did not just stop there in their presentation, they also indicated that they have a workable delivery system that until this point, hampered the ZFN technology a bit. I'm not clinically versed to explain how this is accomplished, but it represents a big advancement for all future ZFN gene edited infusions. Also, mentioned that all commercial partnerships (DOW, SIAL, KBLB and Shire) are all progressing or expanding nicely. 2012 had record royalties for SGMO.

Second straight day of double digit % gains on significant volume. With any follow through and cooperation from the markets, we might be able to establish a higher base and finally leave the $6's behind us for good. We should be trading in the mid teens IMO with just the sheer potential this company has.

The replay of the JPM conference is here:
investor.sangamo.com

A recent news report of expanded clinical trials (also discussed in the JPM conference) is below:
RICHMOND, Calif., Jan. 9, 2012 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the initiation of two new Phase 2 clinical studies (SB-728-1101 and SB-728-902, Cohort 5) in its program to develop a "functional cure" for HIV/AIDS. Sangamo's ZFP Therapeutic® approach (SB-728-T) generates T-cells that are resistant to HIV infection using its zinc finger nuclease (ZFN) technology to permanently disrupt the DNA sequence encoding CCR5, a co-receptor used by HIV to enter cells. The company expects to present data from its SB-728-T HIV clinical trials at appropriate medical meetings in 2012.

Full transcript here:
http://investor.sangamo.com/common/mobile/iphone/releasedetail.cfm?ReleaseID=705990&CompanyID=SGMO&mobileid=

[PCskibum] Didn't catch the presentation. What did they have new to say?

PCskibum — 1/14/2013 9:54:42 PM

[PLegee] 1.86M shares traded today, new 52 week high, 6X normal volume, a 17% increase in...

PLegee — 1/14/2013 9:01:45 PM

1.86M shares traded today, new 52 week high, 6X normal volume, a 17% increase in price. No news to account for the big day as of this time, seems to me that a big fund started to establish a position and then others joined the frenzy. At 10 am EST, volume appeared fairly normal with the stock only marginally above the previous close. Shortly thereafter, a major volume spike occurred (I haven't pulled the block trades yet) which seem to fuel the stock momentum for the remainder of the day. Volume in the 2nd half of the day continued to increase with the last half hour showing numerous high volume trades.

Possibly the recent and impressive presentation at the JPM Healthcare Conference finally incited some funds to act. Regardless, the recent Shire deal along with a rejuvenated board (since the Bill Ringo addition in 2010; undoubtedly the driving force in the Shire deal) proves to me that the "new" SGMO is on the right track. The company seems to have a sense of purpose that it had lacked in the past.

The near term catalyst is obviously the HIV update that will be disclosed in the first half of 2013; this will be the real determinant of where this company is headed near term. SGMO needs to continue to operate with the same sense of urgency it has embraced as of late. Talens, CRSPRS and who knows what other form of gene editing is currently being developed, and beginning to appear on the horizon. We have the IP locked up for ZFN, a minimum two year lead in development; lets use it to our/patient advantage.

[nigel bates] Sangamo BioSciences Provides Comprehensive Overview Of Company's ZFP Therapeutic...

nigel bates — 12/7/2012 4:41:41 PM

Sangamo BioSciences Provides Comprehensive Overview Of Company's ZFP Therapeutic® Development Programs At Its 2012 Analyst BriefingCompany Introduces Novel "In Vivo Protein Replacement Platform" Leverageable Across Many Monogenic Diseases Potentially Enabling Multiple New INDs by the End of 2015

NEW YORK, Dec. 6, 2012 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO), announced that the company is providing an update on its technology platform advancements and pipeline of ZFP Therapeutics® as well as near- and mid-term operating goals during an Analyst and Investor Briefing being held today in New York City. The presentation, by members of Sangamo management team, will be webcast live beginning at 5:00 pm ET and can be accessed via a link on the Company's website.

"We are very excited to share the progress that we have made in advancing our zinc finger DNA binding protein (ZFP) technology and ZFP Therapeutic programs, particularly in the area of monogenic diseases where we have developed a new, highly disruptive therapeutic platform for protein replacement therapies," said Edward Lanphier, president and CEO of Sangamo. "This approach can be leveraged across multiple diseases with the goal of engineering genetic cures."

"Sangamo's proprietary gene-editing and gene-regulation technology is unique in its ability to generate novel, highly differentiated therapies that act at the DNA-level," stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "This capability enables us to address the source of a wide variety of genetic diseases and potentially cure them. In addition to two ongoing Phase 2 clinical trials of SB-728-T, which may afford a functional cure for HIV/AIDS, we have a rich pipeline of preclinical programs. These include ZFP Therapeutics for hemophilia and Huntington's disease, which we are developing in collaboration with Shire, as well as Sangamo's programs in hemoglobinopathies (sickle cell disease and beta-thalassemia) and lysosomal storage diseases (LSDs). Based upon our ZFP Therapeutic approach and the disease targets that we have selected, we expect to file up to seven new INDs by 2015."

"Commercially, our business model has enabled us to monetize the value of our ZFP technology platform and maintain a strong balance sheet through partnerships with Dow AgroSciences in plant agriculture and Sigma-Aldrich Corporation in life science research reagents, while making significant progress in advancing our ZFP Therapeutics programs," continued Mr. Lanphier. "Beyond our very significant collaboration with Shire, we plan to establish additional high-value strategic partnerships around selected therapeutic programs at points of realizable value inflection while continuing to advance proprietary programs and increasingly forward integrate."

Program Highlights
During the briefing members of Sangamo's management team will discuss recent achievements and objectives for the 2013-2015 time period including:

Clinical

Sangamo plans to present preliminary data in the first half of 2013 from its clinical trials of SB-728-T (SB-728-1101 and SB-728-902 Cohort 5) for the treatment of HIV/AIDS and expects to have the full data set from these trials by the end of 2013. Both studies are designed to maximize the engraftment of T-cells in which both copies of the CCR5 gene have been disrupted making the cells resistant to infection by HIV. This creates a reservoir of cells that cannot be infected by the virus but are available to mount an immune response with the goal of providing a "functional cure" for HIV.Preclinical / Research

Sangamo will outline its strategy to enable the potential filing of seven Investigational New Drug (IND) Applications by the end of 2015.The Company will introduce a new, highly disruptive "In Vivo Protein Replacement Platform" driven by Sangamo's ZFP nuclease (ZFN) technology which is potentially curative and leverageable across many monogenic diseases, such as hemophilia and LSDs (e.g. Gaucher, Fabry, and Pompe disease) that are currently treated by regular infusions of enzyme replacement therapy (ERT). Sangamo's novel approach uses a naturally highly expressed gene to drive continuous production of active forms of these proteins from the liver such that they are present at stable levels in the bloodstream thus reducing or eliminating the need for ERT.The Company will update investors on recent preclinical progress in ZFP Therapeutics programs with the goal of engineering a genetic cure for hemophilia, Huntington's disease and hemoglobinopathies (sickle cell disease and beta-thalassemia) and outline the key next steps and timelines to IND filing.

Financial

Sangamo will provide a near- and mid-term financial overview including the anticipated amount of cash and cash equivalent of $40-$45 million by the end of 2015 assuming completion of milestones associated with existing partnerships but not including any additional funding from new partnerships, research grants or equity financing transactions.Sangamo's 2012 Analyst and Investor Briefing is being webcast live and will be available on Sangamo's website at 5:00pm ET via a link in the Investor section at investor.sangamo.com under "Events and Presentations". A replay of the webcast will be archived and available on the website approximately two hours after the presentation.

[PLegee] The Shire deal changes everything in my opinion, plus gives me a much needed boo...

PLegee — 2/21/2012 3:34:44 AM

The Shire deal changes everything in my opinion, plus gives me a much needed boost regarding managerial competence. I think the recent inclusion of Ringo on the Board has already started paying big dividends and his influence had a lot to do with this deal reaching a successful conclusion. This is finally a validation of the company's clinical platform based upon the accolades of Wall St and pharma alike. The partnership is better and more significant than I was previously giving the company's management the credit to achieve. Congrats to Sangamo on this huge step forward, it was long, long overdue.

Looking at the stock going forward, it will be up from here. The upcoming CROI will also unveil some additional HIV updates which the company has strategically maintained complete control over; it is not part of the Shire hemo related deal. Ed has already implied that the HIV trials have been designed to produce maximum effect, I think Phase II updates will be very encouraging (as do most investors). Looks like the newfound momentum continues near term.

Now that the "ice has been cracked", SGMOs potential on hundreds of further mono genetic target opportunities can at least be put into play for other partnership discussions/deals. The significance of the Shire deal cannot be understated; it is a huge step forward for the company. Though Ed will maintain this has been his corp strategy all along, it was the new blood on the B of D that actually influenced the successful outcome of the partnership. This truly is a major milestone for the company and ultimately for patient access to game changing science.

[Cautious_Optimist] Up 26% today... Any thoughts?? I

Cautious_Optimist — 2/17/2012 4:50:32 PM

[PLegee] Obviously `dead money' for another year until something surfaces from the HIV ef...

PLegee — 11/20/2011 10:47:56 AM

Obviously `dead money' for another year until something surfaces from the HIV efforts. Very, very disappointed in the SB509 results. Ed's earlier comments regarding 2011 being a transformative year really suckered me in to buying more stock in anticipation of this groundbreaking medical treatment; regenerating nerves has never been accomplished for DN patients. Comments made by management leading up to the DN announcement all seemed to be inferring positive results (I realize there was an imposed blackout period); I blame only myself for my huge loss.

On the bright side, a ZFN monogenetic focus is what this company needed all along. The longer they screwed around and were distracted with non-ZFN disease states, the more likely someone would eventually come along and crash onto their hallowed HIV IP turf with a disruptive fast-track FDA approved vaccine. Knowing the cost restraints of all governments and health providers, a vaccine would be far more cost effective scenario than a $100K per patient functional ZFN cure; especially if those dependent on recurring drug cocktails can then be quantitatively/financially defined as justifiable by gov't or Big Pharma.

Additionally, there seems to be a notable uptick from their current partnerships in Dow Agro and SIAL. Looks like a record breaking Royalty quarter coming up in 2011. The 2011 financing now looks like a marvelous deal for the company, another secondary is highly unlikely at this stage. All is far from lost with this recent DN set-back.

So, have to believe the damage is done and the bottom is predominantly in place. Though we can go slightly lower from here, the company has adequate cash on hand, proven ZFN technology, a potential new platform in combating disease, a promising pipeline (though at a woefully slow pace; absolutely no sense of urgency), and on the radar screen of every big pharmaceutical company worldwide. At a market cap of < $140M, any number of companies could take SGMO out for literally a balance sheet rounding entry.

Would like to see Ed step aside (fat chance of that with his hand picked directors; he certainly is not CEO material but is one hell of a visionary) and to more aggressively develop early stage partnerships on at least two disease states. This technology will eventually pay off for some company in the field; so far SGMO's chosen `all or nothing' business strategy has been proven to be seriously deficient. Aggressive competition is only a couple years behind and gaining ground rapidly (ie TALENs, HIV vaccines), not sure if SGMO is up to the test. Nonetheless, the recent addition of Bill Ringo to the Board gives me hope that serious competence has arrived and material change is in store. Compensation for company officials is way out of line with results rendered to date, there needs to be a major shake-up.

I will continue to hold on to my stock, feel that the worst is over, and hope my kids get rich off of my decision. HIV news is a year away, I believe it will be different next time around; Drs Cannon and Lazzard (sp) results are more than encouraging. Not a slam dunk by any means, but SGMO still has the IP edge, just wondering if it will ever be commercially deployed.

[mopgcw] still trying to figure that out.

mopgcw — 10/4/2011 12:00:15 PM

[Cautious_Optimist] So what do y'all think??? Buy, sell or hold???

Cautious_Optimist — 10/4/2011 11:52:20 AM

[mopgcw] Sangamo BioSciences, Inc. (SGMO) Special Call October 3, 2011 8:30 AM ET Opera...

mopgcw — 10/4/2011 11:40:30 AM

Sangamo BioSciences, Inc. (SGMO) Special Call October 3, 2011 8:30 AM ET

Operator

Good morning and welcome to the Sangamo BioSciences Teleconference to discuss data from the company’s Phase 2b clinical trial subject to the diabetic neuropathy. This call is being recorded.

I would now pass you over to the coordinator of this event, Dr. Elizabeth Wolffe, Senior Director of Corporate Communications.

Dr. Elizabeth Wolffe – Senior Director of Corporate Communications

Thank you, (Sharon). Good morning and thank you for joining Sangamo’s management team on our conference call to discuss top line data from the company’s Phase 2b clinical trial SB-509-901 which we released this morning.

Also present during this call are several members of Sangamo’s senior management including Edward Lanphier, President and Chief Executive Officer; Geoff Nichol, Executive Vice President of Research and Development; Dale Ando, Vice President of Therapeutic Development and Chief Medical Officer; and Ward Wolff, Executive Vice President and Chief Financial Officer. Following this introduction, Edward and Dale will summarize the data and outline our products forward. Following that, we will open up the call for questions.

As we begin, I’d like to remind everyone that the projections and forward-looking statements that we discuss during this conference call are based upon the information that we currently have available. This information will likely change over time. By discussing our current perception of the market and our future performance of Sangamo with you today, we are not undertaking an obligation to provide updates in the future.

Actual results may differ substantially from what we discuss today and no one should assume at a later date that our comments from today are still valid. We alert you to be aware of risks that are detailed in documents that the company files with the Securities and Exchange Commission, specifically our quarterly reports on Form 10-Q and our Annual Report on Form 10-K. These documents include important factors that could cause the actual results of the company’s operation to differ materially from those contained in our projections or forward-looking statements.

Now, I’d like to turn the call over to Ed.

Edward Lanphier – President and Chief Executive Officer

Thank you, Liz and thank you all for joining us for our conference call to discuss the data that we disclosed in a press release this morning from our placebo-controlled double-blind Phase 2b clinical trial SB-509-901 which was designed to evaluate SB-509 in subjects with moderate severity diabetic neuropathy.

In summary, SB-509 treatment did not show greater improvements from baseline compared with placebo at a 180 days in key endpoints of sural nerve conduction velocity or NCV, neuropathy impairment score in the lower limb or NIS-LL, quantitative sensory testing or QST, or intraepidermal nerve fiber density or IENFD.

We did see trends towards improvement in an exploratory endpoint for lower extremity neurologic sensory exam or LENSE. This is a standardized exam which provides the clinical measure of sensory function in subjects with neuropathy. I have asked Dale to provide more detailed description in the study and findings later in the call.

We are obviously disappointed that the SB-509-901 trial did not produce a better outcome in the pre-specified primary and secondary endpoints. We designed the 901 study using clinical data from our previous Phase 1 and Phase 2 clinical trials and believe that we conducted a thoroughly designed and well-controlled trial. Therefore, based upon these results we will discontinue all further clinical development of SB-509.

Moving forward, we will focus our attention and resources on our rich pipeline of ZFP Therapeutic programs, particularly in HIV/AIDS and in monogenic diseases. These are unmet medical needs for which our Zinc Finger Nuclease genome editing technology is uniquely suited to provide a therapeutic solution.

Before I say anything more about our future plans, let me turn the call over to Dale to give you some more details on the data that we collected in the 901 trial. Dale?

Dale Ando – Vice President, Therapeutic Development and Chief Medical Officer

Thanks Edward. The challenge in any clinical development program is to design trials that provide the best chance of seeing a significant difference between the placebo group and the treated group around clinically meaningful and potentially approvable endpoints over the period of the study.

As you know, we have previously carried out several Phase 2 trials in population with varying severities of diabetic neuropathy, which is the progressive degeneration of the nerves. Using the data from these studies, we undertook a rigorous accrual process designed to exclude both the very mild and very severe populations and thereby subjects that we believe would be the most responsive to treatment. This selection involved assessing baseline measurements of all major endpoints and accruing only subjects that sell into a pre-specified range.

In addition, we instituted rigorous training of site personnel with regard to drug administration and technical and clinical measurements. The trial enrolled 170 subjects in which only three did not complete the trial. So, just from randomized one-to-one and treated by intramuscular injections at day 0, 60, and 120 with either SB-509 or a saline placebo. We monitored the progression of the diabetic neuropathy symptoms by measuring sural, NCV, NIS-LL, QST, and IENFD and LENSE at baseline in several points throughout the 100 days study period. We also assess the quality of life using questionnaires and monitored their underlying diabetes.

The placebo treatment groups were well-matched in terms of their overall demographics and diabetes control over the course of the study period. As we had observed in prior trials, SB-509 was generally well-tolerated. There were three serious adverse events in the SB-509 treated group compared to three serious adverse events in the placebo group. The remaining adverse events were mild and reversible and generally equivalent in both groups. The primary endpoint of the study was sural NCV. We saw no statistical difference in this measurement at 180 days between the SB-509 treated and placebo group.

As in past trials, the SB-509 treated group showed improvement in sural NCV measurement. However, this study the placebo group unexpectedly also showed improvement. Compared to placebo treatment, SB-509 treatment did result in a greater improvement in nerve fiber density in the skin as we have seen in the 601 study, but it was not statistically significant. While we did not see any differences between the SB-509 treated groups in the overall neurologic exam as quantified by NIS-LL, in the pre-specified analysis, we did observe a clinically relevant improvement from baseline in the mean total LENSE score in SB-509 treated subjects compare to placebo treated subjects with a p-value of 0.11.

This trend affected pinprick and touch pressure sensation more than vibration and was primarily due to improvement seen in subjects with a baseline IENFD score of less than 9 fibers per millimeter or subjects with more severe DN. In this group, the effect on total LENSE score was prominent at 90 days with the mean improvement from baseline in treated subjects compared with the worsening in placebo subjects which gave a p-value of 0.008.

The LENSE uses assessments routinely applied in clinical neurologic examination and provides a measure of patient-reported sensory function. These positive outcomes in LENSE which mean more directly reflect patient benefit by therefore scientifically intriguing. However, in the end, we did not meet our key clinical endpoints and accordingly will no longer pursue development of SB-509.

Let me now hand the call back to Edward.

Edward Lanphier – President and Chief Executive Officer

Thanks, Dale. So, first and foremost, I would like to take this opportunity to thank the patients, investigators, and the Juvenile Diabetes Research Foundation for their support and participation in this trial. I would also like to thank and acknowledge my colleagues here at Sangamo who did a very professional job conducting this study.

While it’s always disappointing to have to closedown a clinical program, we have a broadly applicable technology platform which provides us with multiple opportunities for the development of new therapeutics. Our ZFN mediated genome editing platform enables us to modify any gene that we choose and as such is ideally suited to address diseases for which the gene target is well-defined such as our CCR-5 HIV programs and in monogenic diseases such as hemophilia.

As most of you know, we recently presented very encouraging data from our studies of SB-728-T at ICAAC demonstrating “significant progress towards a functional cure for HIV/AIDS.” Based upon these data, we planned to continue to expand this clinical program and begin two new clinical trials in the first half of 2012 designed to maximize the engraftment of CD4 T-cells with both CCR-5 alleles modified.

One study will be in subjects for natural heterozygote for CCR-5, delta-32 mutation in whom biallelic modification is maximized and a second study in non-heterozygotes which will explore mechanisms to enhance engraftment of SB-728-T and maximize the impact of HIV-resistant cells on Viral Load Reduction and the overall improvement of the immune system of HIV patients.

We also have several programs at the late-stage of large animals, model studies including our ZFP Therapeutic for Parkinson’s disease, which is being tested in non-human primates. And our ZFM program for the correction of the Factor IX gene and hemophilia, which is being studied in a dog model of the disease. In addition, we have ongoing pre-clinical and research stage programs in Huntington’s disease, sickle-cell anemia, X-linked SKID, and other monogenic diseases.

Finally and very importantly, we have a very strong financial position and remain on track to this end to end the year with at least $85 million in cash and cash equivalents. I look forward to updating you on our progress, on our third quarter call later this month. This completes our prepared comments.

I would now like to open up the call for your questions.

Question-and-Answer Session

Operator

(Operator Instructions) Our first question comes from Charles Duncan with JMP Securities. You may begin.

Charles Duncan – JMP Securities

Hi, guys. Just a couple of questions, first involve Edward, in terms of the diabetic neuropathy program, what do you think was the issue, was this the lack of biologic activity or perhaps multiple factors involved in measuring clinical activity and diabetic neuropathy?

Edward Lanphier

Good, Charles, let me start and then perhaps Geoff and Dale would like to say further. There is no question this is a complex disease. There is no question that there is a lot of underlying biology. I’ll say two things. One, I think the design of this trial and the successful efforts to accrue precisely the population that we went to accrue gave us a very good study in terms of what we have proposed to do. With that said, we did not meet our primary or secondary endpoints, which are measures of changes in neurological health and as such our plan is to no longer continue to invest in this program. Dale or Geoff, anything in addition?

Dale Ando

No, I think that says it all Edward. Charles, diabetic neuropathy has always been a difficult study to examine. Clinically, the endpoints are the best that we can come up with, but I think if you ask around the community, folks are not delighted with nerve conduction velocity as being a primary endpoint. So, and certainly a lot of the measures that we use have a fair amount of statistical variation. So, you are right, it is a tough disease to approach. We did not see as much and as clear an increase or a difference in the nerve fiber density as we had expected to see based on the prior studies, but we did see some difference between the active and treated group, which might be a suggestion that the underlying biological activity, which is pretty clear in pre-clinical studies was nevertheless operating, but simply not enough to be able to detect sufficient differences in nerve conduction velocity and NIS-LL to lead to releasing of in change here.

Charles Duncan – JMP Securities

Okay. And if I could…

Edward Lanphier

I think Charles…

Charles Duncan – JMP Securities

Excuse me.

Edward Lanphier

Charles, by contrast some of those complications and diversity of endpoints and even subjectivity of endpoints with the kinds of endpoints that we are able to look at in specific gene targets both in monogenic diseases as well as in CCR-5 and changes in viral load. So, moving forward, I think we are looking at indications where the endpoints are more well-established and are much more clear to see a signal in early stage clinical programs.

Charles Duncan – JMP Securities

It makes sense to me perhaps if I could ask a question about the HIV program, you know, that the diabetic neuropathy program has been terminated. It seems to me that both operationally and financially you may be able to focus more resources on that program. Can you tell us, what you can do for that program? Is that possible that you could catalyze timelines or perhaps even point to the next value added milestone for that program?

Edward Lanphier

Well, I think we have been gearing up for, I’ll take last 18 months and you heard me several times say that both our enthusiasts for this program, but it’s a science and database development plan and as of ICAAC of this year with the clarity around the correlation between biallelic modification and reduction in viral load, we are expeditiously and then by that I mean in as quick way as possible and not in a resource constraint way towards the two clinical trials.

I won’t repeat them here in now that I outlined earlier and we discussed just a few minutes ago on the call. But there is no question that has been in the plans all long and we continue to move forward that expeditiously and we continued and have been investing aggressively in our preclinical program and highlighting the Parkinson’s program, the hemophilia program, the hemoglobinopathy programs and others.

Charles Duncan – JMP Securities

We have noticed in clin trials that you have been able to expand the HIV program with more patient and longer treatment interruptions. Are there any takeaways from the diabetic neuropathy program in terms of say the FDA or your capabilities and comfort with the technology platform, which is alone you have to expand the HIV program?

Edward Lanphier

Well, obviously these are different programs, different drugs, different diseases indications. I’ll say and as Dale discussed the safety profile of SB-509 in this patient population is good, outstanding. The drug was very well tolerated and safe in this group and essentially equivalent at these in both the placebo and the treated population. And I would also speak to the relationship that Dale and the team here have and the quality of that relationship with the both of our investigators as well as regulatory agencies. But moving forward, I think there is a growing safety base both in SB-509, but also moving forward we have a growing safety database in the SB-728-T.

Charles Duncan – JMP Securities

Thanks Edward. I will hop back in the queue.

Edward Lanphier

Thanks Charles.

Operator

Thank you. Our next question comes from Liana Moussatos with Wedbush. You may begin.

Liana Moussatos – Wedbush

Thanks for taking my question. When do you think you will have monogenic diseases in the clinic like hemophilia and you mentioned Parkinson’s you think you will have something in the clinic next year?

Edward Lanphier

Thanks, Liana. I think I’m going to give you an answer that may not be completely satisfying, but its one that you heard from me many times. The decision to move things forward will be based upon science and based upon data. So, we are moving multiple programs forward. Actually I think if you look at the breadth of our preclinical pipeline and even the maturity of our preclinical pipeline. I think it’s very robust relative to many, many, many biotech companies. So, we are moving those forward.

To say at this point, here is the one that we are going to take forward in terms of an IND and the timing on that is just premature. But I can assure you that I’m is interested and anxious to provide that guidance as anyone, maybe more so. And that as we have the large animal data confirming what we have seen in earlier stage animal studies with our existing programs and this moving forward, our monogenic disease programs. We will certainly continue to keep you informed of that and as soon as we have clarity around from a science-driven perspective we will guide to the timing of INDs.

Liana Moussatos – Wedbush

Okay. And the status of glioblastoma and ALS programs, what is that?

Edward Lanphier

The ALS is part of the SB-509 package and as such we do not intend to continue our further investment in that. That certainly a program that where there is initially data it was a signal-seeking study in ALS and there are some signals there. But we will not be continuing to move that forward ourselves. As it relates to glioblastoma the guidance is the same as before upon having a sufficient amount of data to discuss. We will be presenting that in appropriate scientific meeting.

Liana Moussatos – Wedbush

Thank you.

Operator

Thank you. I show no further questions at this time. I would now like to turn the call back over to Edward Lanphier.

Edward Lanphier

Actually, I’m showing this time one more question. Operator?

Operator

We have a question from Chad Messer with Piper Jaffray. You may begin.

Chad Messer – Piper Jaffray

Hi, thanks, thanks guys. You have mentioned in the release in briefly in Dale’s remarks that you had a more robust placebo response then you had anticipated. How much was that a contributing factor relative to some of the other factors you discussed and do you have any series is to what might have caused the placebo that will be more robust or is it just look like kind of way in the bad luck to you?.

Edward Lanphier

I’ll start this is certainly something Dale is start deeply about and is commented on. Chad, your overall observation is exactly right. We saw and this is not scientific. But at a high level, we saw essentially the same improvement in the moderate severity population in this 901 trial that we saw in that disease segment population 601 trial. So, a fairly robust improvement and in the subset in the 601 trial that placebo population eroded and so it created a delta and a chronically meaningful delta and that’s what we intended to recapitulate. What we did see in this trial across those endpoints largely was that same sort of robust positive response in the treated population. But we saw a similar improvement in placebo population. Why that is I mean is obviously a question of debate, I know Dale has some strong feelings about the improvement in diabetes control. Dale is there anything you want to comment further about either the placebo effect here or after that.

Dale Ando

Yeah, I don’t think we exactly, but the treatment in diabetes has improved significantly with the addition of DPP-4 inhibitors, GLP-1, and PPAR-gamma mechanism of drug. So, possible that there may be non-glucose related improvements in the diabetic neuropathy and that is difference from the 601 study when those drugs were not that prevalent use, so there is that potential.

Chad Messer – Piper Jaffray

All right, well, thanks guys. I guess this highlights the complexity of the disease you’re going after and I do comment you for it at least one in the right study to trying to get these answers.

Dale Ando

Thanks Chad. I appreciate that.

Operator

Thank you. I’ll show no further questions. I’d like to turn the call back over to Edward Lanphier.

Edward Lanphier – President and Chief Executive Officer

Thank you. We’d like to thank you for joining us. We look forward to speaking with you again when we release our third quarter financial information. We’ll be available later today if you have any follow-up questions. Thank you.

Operator

Ladies and gentlemen, this concludes today’s conference. Thank you for your participation. Have a wonderful day.

[sim1] Sangamo BioSciences Announces Phase 2b Trial of SB-509 in Diabetic Neuropathy Di...

sim1 — 10/3/2011 8:09:01 AM

Sangamo BioSciences Announces Phase 2b Trial of SB-509 in Diabetic Neuropathy Did Not Meet Key Study Endpoints

October 3, 2011

Company to Discontinue SB-509 Development to Focus on Pipeline of ZFP® Therapeutics for HIV and Monogenic Diseases
Company to Hold Conference Call at 8:30 am ET Today to Discuss Data
RICHMOND, Calif., Oct. 3, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) today announced that its Phase 2b study (SB-509-901) did not meet its primary or secondary clinical endpoints in subjects with moderate severity diabetic neuropathy (DN) as compared to placebo.

"We are disappointed that this trial did not produce a better outcome in the pre-specified primary and secondary endpoints," said Edward Lanphier, Sangamo's president and CEO. "Based on these results, we will discontinue further development of SB-509 and will focus our attention and resources on our pipeline of ZFP Therapeutics for HIV and monogenic diseases for which our genome editing technology is uniquely well positioned. We would like to thank the patients, investigators and the Juvenile Diabetes Research Foundation (JDRF) for their support and participation in the trial."

Phase 2b Trial Results

SB-509 treatment did not show statistically significant improvements from baseline compared with placebo at 180 days in the primary endpoint, sural nerve conduction velocity (sNCV), the secondary endpoint, neuropathy impairment score in the lower limb (NIS-LL), or intraepidermal nerve fiber density (IENFD).

In a pre-specified analysis, a clinically relevant improvement was observed in the mean total LENSE score of 3.4 points in SB-509-treated subjects compared to a 1.9 point improvement in placebo treated subjects from baseline (p=0.11). This trend affected pinprick and touch pressure sensation more than vibration, and was primarily due to improvement seen in subjects with a baseline IENFD score of <9 fibers/mm. In this group the effect on total LENSE score was more prominent at 90 days (mean improvement of 4.3 points in treated subjects compared with a worsening of 0.8 points in placebo subjects; p=0.008) than at 180 days (mean improvement of 4.8 points in treated subjects compared with an improvement of 1.8 points in placebo subjects; p=0.08).

SB-509 was generally well-tolerated. There were three serious adverse events in the SB-509 treated group compared with three serious adverse events in the placebo group. The remaining adverse events were mild and reversible and were generally equivalent in both groups.

The full clinical data set will be published or presented at an appropriate medical meeting.

"While positive effects on major endpoints in SB-509-treated subjects in this trial were of a similar magnitude to those observed in our earlier Phase 2 trial (SB-509-601), the placebo group in the SB-509-901 study showed an unexpected improvement in these measures over the 180 day test period," stated Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "Although LENSE was only an exploratory endpoint in the trial, the positive outcomes that we observed were scientifically intriguing. LENSE uses assessments routinely applied in clinical neurological examination and provides a measure of patient-reported sensory function, in contrast to current endpoints such as NCV whose clinical relevance is less clear."

Phase 2b Trial Design (SB-509-901)

The double blind, repeat-dosing, placebo controlled Phase 2b study, SB-509-901, was designed to finalize dose, schedule, and primary and secondary endpoints for pivotal Phase 3 trials. The trial accumulated data on endpoints including nerve conduction velocity in the sural nerve (sNCV, the primary endpoint), Neurological Impairment Score in the Lower Limb (NIS-LL, the secondary endpoint), as well as exploratory endpoints of Lower Extremity Neurological Sensory Examination (LENSE), quality of life assessments (QOL) and intraepidermal nerve fiber density (IENFD). The study involved a total of 170 subjects who were randomized 1:1 between placebo and treatment groups.

About SB-509

SB-509 is an injectable plasmid encoding a zinc finger DNA-binding protein (ZFP) transcription factor (ZFP TF) designed to upregulate the endogenous expression of the gene encoding vascular endothelial growth factor (VEGF-A). In pre-clinical studies, VEGF-A has been demonstrated to have direct angiogenic, neurotrophic and neuroprotective properties.

About Diabetic Neuropathy

The Centers for Disease Control estimate that from 1980 through 2010, the number of Americans with diabetes has more than quadrupled from 5.6 million to 25.8 million and that of those about 60 to 70 percent have mild to severe forms of neuropathy. Diabetic neuropathy is a progressive degenerative disease that is one of the most frequent complications of diabetes, affecting between 15.8 and 18 million Americans in 2010. High blood glucose levels lead to nerve damage over time, primarily affecting peripheral nerves. Symptoms include numbness, tingling sensations and pain particularly in the toes or feet, which gradually evolve to loss of sensation and motor function as nerve damage progresses. Ulcers and sores may appear on numb areas of the foot as pressure wounds or injuries go unnoticed. Despite palliative treatment, these areas of trauma frequently become infected and this infection may spread to the bone, necessitating amputation of the leg or foot. More than 60 percent of non-traumatic lower-limb amputations in the United States occur among people with diabetes.

Conference Call

Sangamo will host a conference call to discuss these results today, October 3, 2011 at 8:30 a.m. ET, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo BioSciences website in the Investor Relations section under "Events and Presentations" http://investor.sangamo.com/events.cfm. The webcast replay will also be available for two weeks after the call.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The passcode for the call is 15507481. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 11:30 am ET on October 3, 2011 to midnight ET on October 10, 2011. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406 respectively. The conference ID number for the replay is 15507481.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is SB-509, which recently reported Phase 2b clinical trial data in patients with diabetic neuropathy. Sangamo also has a Phase 1/2 clinical trial and two ongoing Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS as well as a Phase 1 trial of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneration. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins called zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence, Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at www.sangamo.com.

ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform for the treatment of diabetic neuropathy, HIV/AIDS and monogenic diseases. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.

SOURCE Sangamo BioSciences, Inc.

[sim1] Sangamo BioSciences to Host Conference Call to Discuss Results From Phase 2b Tri...

sim1 — 9/30/2011 10:02:35 PM

Sangamo BioSciences to Host Conference Call to Discuss Results From Phase 2b Trial of SB-509 in Diabetic Neuropathy Teleconference and Webcast to be held at 8:30 am ET, Monday, October 3, 2011

Press Release Source: Sangamo BioSciences, Inc. On Friday September 30, 2011, 5:40 pm EDT

RICHMOND, Calif., Sept. 30, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) today announced that the company will hold a teleconference and webcast at 8:30 am ET, on Monday, October 3, 2011, to discuss results of the Phase 2b clinical trial (SB-509-901) from its ZFP Therapeutic® program to develop SB-509 as a treatment for diabetic neuropathy. A press release summarizing the data from the trial will be issued at 7:00 am ET on Monday prior to this call.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The passcode for the call is 15507481. Participants may access the live webcast via a link on the Sangamo BioSciences website in the Investors section under "Events and Presentations http://investor.sangamo.com/events.cfm. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 11:30 am ET on October 3, 2011 to midnight ET on October 10, 2011. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406 respectively. The conference ID number for the replay is 15507481.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy. Sangamo also has a Phase 1/2 clinical trial and two ongoing Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS as well as a Phase 1 trial of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneration. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins called zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence, Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at www.sangamo.com.

ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

[nigel bates] “HAART Treatment Interruption Following Adoptive Transfer of Zinc Finger Nucleas...

nigel bates — 9/2/2011 8:44:19 AM

“HAART Treatment Interruption Following Adoptive Transfer of Zinc Finger Nuclease (ZFN) Modified Autologous CD4 T- Cells (SB-728-T) to HIV-Infected Subjects Demonstrates Durable Engraftment and Suppression of Viral Load”

seekingalpha.com

[tnsaf] When Zinc Fingers Miss the Mark Two new techniques identify how often zin...

tnsaf — 8/9/2011 1:20:50 PM

When Zinc Fingers Miss the Mark
Two new techniques identify how often zinc fingers nucleases cleave off-target sites.

By Tia Ghose | August 7, 2011

the-scientist.com

Zinc finger nucleases are designed to be like heat-seeking missiles, precisely targeted to find and cut specific sequences of DNA. Occasionally, however, they may snip the wrong spot, causing unintended breaks. Two papers published today (August 7) in Nature journals describe ways to systematically find such off-target action, which could one day help design gene therapies that avoid collateral damage.

“Until this time there hasn’t been a really comprehensive way of defining zinc finger nuclease specificity,” said Carlos Barbas, a chemical biologist at the Scripps Research Institute in La Jolla, Calif., who was not involved in the study. “As we begin to treat patients with zinc finger nucleases and modify genomes, we need to know where those modifications are being made.”

Zinc fingers, so named because their structure resembles a hand with a pointed finger, bind to different three-letter nucleotide sequences. By stringing together several zinc fingers and adding a DNA-cleaving nuclease, researchers can precisely target specific genes to be cut. Such specificity raises the possibility of developing zinc finger nuclease (ZFN) gene therapies. Indeed, Sangamo Biosciences, a pharmaceutical company, is testing an HIV-treatment candidate in human safety trials which uses a ZFN to modify the CCR-5 T-cell receptor that HIV uses to enter a cell.

But it’s not a perfect system: sometimes the molecule may bind and clip a different, nearly identical DNA sequence, potentially killing cells.

To systematically characterize those off-target cleavage sites, Harvard chemical biologist David Liu and his colleagues tested two ZFNs against a library of 100 billion snippets of DNA, some of which appear in the human genome. Most often, the nucleases cleaved the target site. But they occasionally cut other similar sequences as well—including one gene associated with a cancer signaling pathway.

“A superficial interpretation of our paper might lead one to be pessimistic about zinc finger nucleases, but actually I’m optimistic,” Liu said. In addition to confirming that the fraction of off-target breaks decreased with lower concentrations of ZFNs, the researchers found that using ZFNs that bind less avidly to the target sequence seemed to have fewer unintentional breaks, he said. That suggests it may be possible to design ZFN therapies in a way that minimizes those off-target effects. The researchers published their results in Nature Methods.

In the second study, published in Nature Biotechnology, researchers dosed human leukemia cells with a ZFN which cuts the CCR-5 receptor. They identified the cut locations by transfecting the cells with tagged virus particles that bound to the broken ends of the DNA, and found that by and large, the ZFN bound to the target CCR-5 DNA. About 1 in 20,000 times, however, it cleaved a second receptor gene nearly identical in sequence, as well as a few other similar sequences even more rarely. But the researchers used an extremely high concentration of ZFN, and used a cell line that is very permissive of ZFN action, to see what the worst case scenario would be, said coauthor Phillip Gregory, chief scientific officer of Sangamo BioSciences. The low rate of off-target cutting even under these conditions “validates our expectations that the proteins would be tremendously specific,” and suggests that much lower medical doses applied in the clinic would almost never be expected to cause off-target cuts, he said.

The methods might one day be used in early drug development to pick candidates with the best specificity, Barbas said. It’s unclear, however, just how comprehensive the new ZFN tests are, he said. The tagged virus particle method, for example, may miss some off-target cleavage, because the viral tags may not bind to every single break. Furthermore, Barbas added, unlike DNA in a test tube, cellular DNA is tightly wound into chromatin, so many of the binding sites found by the test tube method might be shielded from ZFNs and never be cut in living cells.

So while the new tests may be key tools for early drug development, a complete picture will only come once a person’s entire genome sequence can be had for $1000, he said, when researchers can test people who receive ZFN treatments for every off-target break.

R. Gabriel, et. al, “An unbiased genome-wide analysis of zinc-finger nuclease specificity,” Nature Biotechnology, doi:10.1038/nbt.1948, 2011.

V. Pattanayak, et. al, “Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection,” Nature Methods, doi:10.1038/nmeth.1670, 2011.

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micro

>>So while the new tests may be key tools for early drug development, a complete picture will only come once a person’s entire genome sequence can be had for $1000, he said, when researchers can test people who receive ZFN treatments for every off-target break.<<

In every cell??? I don't think so.