The news:
CAMBRIDGE, Mass., and PHILADELPHIA, Feb. 19 /PRNewswire/ -- Ariad Pharmaceuticals Inc , through its
subsidiary, ARIAD Gene Therapeutics, Inc., and Genovo, Inc. today announced the formation of a joint venture to
develop and commercialize gene therapy products for the estimated $11 billion therapeutic protein market. ARIAD
and Genovo will share equally in all costs and profits resulting from the joint venture's activities.
In addition to bringing together two of the leading companies in the gene therapy field, the joint venture will
combine Genovo's gene transfer technologies and ARIAD's gene expression regulation system. The companies
believe that their combined technologies can form a novel platform for the delivery of genes into human tissue and
the subsequent control of the amount of therapeutic protein produced by those genes.
The goal of the ARIAD-Genovo joint venture is to convert current protein therapy, which requires frequent
injections, into therapy based on orally administered drugs. The joint venture's products may offer potential
improvements in the efficacy of protein therapy as well. The products envisioned by the joint venture involve the
injection of genetic material directly into a patient's muscle or skin. This genetic material would equip the patient's
cells to produce a therapeutic protein of choice in response to a specific small-molecule drug. Following
administration of the genetic material, the patient would take the orally available drug to achieve stable, long-term
production of the protein at optimum therapeutic levels.
The joint venture's products are being designed to address specifically the key issues facing the practical clinical
application of gene therapy: efficiently and safely delivering genetic material directly to the patient's cells, causing
those cells to produce adequate and sustainable levels of therapeutic protein, controlling production levels in a
manner consistent with conventional pharmaceutical dosing, and providing the ability to cease treatment should it
become unnecessary or unwanted.
While the joint venture's products may incorporate genes that encode for any of a large number of therapeutic
proteins, the joint venture will focus initially on developing alternative gene therapy-based products for currently
marketed injectable proteins, such as growth hormone.
ARIAD's technology for precisely controlling the dose of therapeutic proteins delivered by gene therapy is based
on transcription factors, intracellular signaling molecules that activate protein productiin. ARIAD has engineered a
specific, two-part transcription factor that activates protein production only when its two halves are brought
together by a small-molecule drug. By equipping cells with a therapeutic gene and the genes required to produce
ARIAD's two-part transcription factor, it is possible to activate and control the level of therapeutic protein
production in vivo using an orally administered drug.
Genovo is a leader in the development and manufacture of gene transfer technologies, including both viral- and
nonviral-based vectors. These vectors are used in gene therapy to equip patient's cells with the genes necessary to
produce a therapeutic protein of choice. They may also be used to equip cells to produce ARIAD's two-part
transcription factor. Genovo's leadership position in gene therapy stems from its exclusive license of patents and
access to the pioneering work of James Wilson, M.D., Ph.D., and his laboratory at the University of Pennsylvania
School of Medicine. Dr. Wilson is director of the Institute for Human Gene Therapy at the University of
Pennsylvania School of Medicine and scientific founder of Genovo.
"By combining ARIAD and Genovo's expertise and technology in gene therapy, we intend to develop safer, more
effective and less costly ways to deliver protein drugs," said Harvey J. Berger, M.D., ARIAD's chairman and chief
executive officer. "We anticipate that human clinical trials of the first joint venture product will commence next
year."
"In addition to developing gene therapy products itself, the ARIAD-Genovo joint venture will seek partnerships
with companies that have a strong strategic interest in protein therapeutics and the markets they address," said Peter
Neff, chairman and chief executive officer of Genovo.
The current worldwide market for injected therapeutic proteins is estimated at over $11 billion annually. As the
number of new protein products increases and as the indications for such products expand, this market is expected
to experience significant growth. This growth will likely accelerate if improvements can be achieved in the patient
compliance, safety and efficacy of protein-based therapy.
ARIAD Pharmaceuticals is engaged in the discovery and development of novel pharmaceuticals based on
intracellular signaling technology. The company has established highly integrated capabilities in functional
genomics, molecular cell biology, structure-based drug design, combinatorial chemistry and pharmacology.
ARIAD is developing small-molecule drugs that block signal transduction pathways in cells responsible for
osteoporosis, immune and inflammatory diseases. In addition, the company has developed a system to control signal
transduction pathways for the regulation of therapeutic protein production in gene therapy.
Genovo is a gene therapy company focusing primarily on the development of viral and nonviral vector systems.
Genovo is engaged in the development of core technologies for gene transfer, and in the application of these
technologies in selected disease. Through its relationships with the University of Pennsylvania and other academic
institutions, Genovo has the capacity to rapidly move its research from conception through preclinical testing and
into clinical trials.
Some of the matters discussed in this news release are forward-looking statements that involve risks and
uncertainties, including but not limited to economic, competitive, governmental and technological factors affecting
ARIAD's operations, markets, products, services and prices, and other factors discussed under the heading
"Cautionary Statement Regarding Forward-Looking Statements" in ARIAD's Annual Report on Form 10-K filed
with the Securities and Exchange Commission. SOURCE ARIAD Pharmaceuticals, Inc. |
