BOSTON, June 13, 2013 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to National Tay-Sachs and Allied Diseases Association (NTSAD) for development of the first-ever treatment for Tay-Sachs and Sandhoff, rare diseases that are fatal in young children and extremely debilitating in their late-onset form. <\/p>\n
The gene therapy in development would correct an enzyme deficiency that causes the progressive neurodegeneration that marks these diseases. Both Tay Sachs and Sandhoff are lysosomal storage diseases, a group of more than 50 genetically inherited disorders characterized by deficiency of a vital enzyme that prevents the proper breakdown of undigested material inside cells. <\/p>\n
Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding of certain clinical studies, study-design assistance and several years of market exclusivity for the product upon regulatory approval. <\/p>\n
\"This orphan drug designation is a giant step forward in our efforts to bring hope to Tay-Sachs patients and their families, as today there are no treatments,\" said NTSAD President, Shari Ungerleider. \"Gene therapy has the potential to be a one-time transformative therapy for patients suffering from rare neurodegenerative genetic disorders such as Tay-Sachs. NTSAD, along with its funding partners, is committed to advancing the clinical and commercial development of our gene therapy platform because of the potential life-changing benefit it could have for patients and their families.\" <\/p>\n
Based on promising results of animal studies that have been ongoing since 2007, the Tay-Sachs Gene Therapy Consortium research team is completing pre-clinical studies in advance of a Phase I clinical trial. <\/p>\n
About the Tay-Sachs Gene Therapy Consortium <\/p>\n
The Tay-Sachs Gene Therapy (TSGT) Consortium was founded in 2007 to advance human clinical trials in the quest for a gene therapy treatment for Tay-Sachs and Sandhoff diseases. The multidisciplinary team, led by Miguel Sena Esteves, Ph.D., recipient of the 2011 Outstanding New Investigator Award from the American Society of Gene & Cell Therapy, includes scientists and clinicians from Auburn University, Boston College, Cambridge University-UK, Massachusetts General Hospital\/Harvard Medical School, University of Massachusetts Medical School, and New York University Medical School. <\/p>\n
About NTSAD <\/p>\n
The oldest rare disease advocacy organization in the nation, National Tay-Sachs and Allied Diseases Association (NTSAD) was founded in 1957 by concerned parents whose children were affected by Tay-Sachs disease or related rare genetic lysosomal storage diseases and leukodystrophies. Today NTSAD continues its multifaceted support of affected families and funds research seeking a treatment or cure. NTSAD also pioneered the development of community education about carrier screening programs for Tay-Sachs and related diseases, which became models for all genetic diseases. More information is available at http:\/\/www.ntsad.org<\/a>. <\/p>\n<\/p>\n <\/p>\n See the rest here:<\/p>\n National Tay-Sachs & Allied Diseases Association Receives U.S. Orphan Drug Designation for Novel Gene Therapy<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" BOSTON, June 13, 2013 (GLOBE NEWSWIRE) -- The U.S. Continue reading