TKT STRENGTHENS INTELLECTUAL PROPERTY POSITION FOR RARE GENETIC DISEASES WITH ISSUANCE OF PATENT FOR THE TREATMENT OF SANFILIPPO SYNDROME TYPE A (MPS IIIA)
Cambridge, MA, March 13, 2001
Transkaryotic Therapies, Inc. today announced that the U.S. Patent and Trademark Office has issued U.S. Patent No. 6,200,563 entitled, "Synthetic Mammalian Sulphamidase and Genetic Sequences Encoding Same." The patent is based on work performed by Dr. John Hopwood and colleagues, who cloned the gene and cDNA for sulphamidase and characterized the protein. The patent claims are related to purified sulphamidase made from cells transfected with DNA encoding the protein. The protein sulphamidase is useful in the investigation, diagnosis and treatment of Sanfilippo syndrome type A, also known as mucopolysaccharidosis IIIA (MPS IIIA). The patent was issued to Women and Children's Hospital in North Adelaide, Australia and licensed to TKT through an exclusive worldwide license agreement.
"Based on a license agreement with Women and Children's Hospital, three patents relating to lysosomal enzymes have issued recently relating to treatments for MPS I, II, and IIIA. The issuance of these patents further enhances TKT's ability to develop protein and gene therapy products for these serious diseases," said Richard F Selden, M.D., Ph.D., President and Chief Executive Officer of TKT. "These diseases generally result in a diminished quality of life as well as numerous hardships. We remain committed to our goal of providing effective therapies to patients in need."
Sanfilippo syndrome type A is a rare inherited genetic disorder caused by deficient activity of the protein sulphamidase. Sulphamidase is present in the lysosome of cells where it helps break down large biomolecules called glycosaminoglycan catabolisms. Patients affected by Sanfilippo syndrome type A suffer from a variety of symptoms including skeletal deformities and progressive mental retardation.
A copy of the patent described above is available on the worldwide web at www.uspto.gov.
Transkaryotic Therapies, Inc. (TKT) is a biopharmaceutical company dedicated to the development and commercialization of products based on its three proprietary development platforms: Gene-Activated® proteins, Niche Protein™ products, and Gene Therapy. The Company's gene activation technology is a proprietary approach to the large-scale production of therapeutic proteins, which does not require the cloning of genes and their subsequent insertion into non-human cell lines. TKT's Niche Protein product platform is based on protein replacement for the treatment of rare genetic diseases, a group of disorders characterized by the absence of certain metabolic enzymes. The Company's Gene Therapy technology, known as Transkaryotic Therapy™, is focused on the commercialization of non-viral, ex vivo gene therapy products for the long-term treatment of chronic protein deficiency states.
This press release contains forward-looking statements that involve a number of risks and uncertainties. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, the words, "believes," "anticipates," "plans," "expects," "intends," and similar expressions are intended to identify forward-looking statements. Important factors that could cause actual results to differ materially from the expectations described in these forward-looking statements are set forth under the caption "Certain Factors That May Affect Future Results" in the Company's Annual Report on Form 10-K for the year ended December 31, 1999 and the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2000 which are on file with the Securities and Exchange Commission and incorporated herein by reference. These important factors include risks as to whether TKT's products will advance in the clinical trials process, the timing of such clinical trials, whether the clinical trial results will warrant continued product development, and whether the Company's products will receive approval from the U.S. Food and Drug Administration or equivalent regulatory agencies, and, if such products receive approval, whether they will be successfully marketed; the results of any patent litigation in which the Company is involved or may become involved; competition; and the Company's dependence on collaborators.
Gene-Activated® is a registered trademark and Niche Protein™, TKT™, and Transkaryotic Therapy™ are trademarks of Transkaryotic Therapies, Inc.
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