Date: 2016-04-27
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 19th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
Company: Audentes Therapeutics (USA - CA) Genethon (France)
Product: AT132
Action
mechanism:
- gene therapy. X-Linked Myotubular Myopathy (XLMTM) is caused by mutations in the MTM1 gene, which encodes a protein called myotubularin. Myotubularin plays an important role in the development, maintenance and function of muscle cells. AT132 is a recombinant serotype 8 adeno-associated virus (AAV8) vector expressing murine myotubularin. Audentes is developing AT132 for the treatment of XLMTM in collaboration with Genethon.
Disease: X-linked myotubular myopathy (XLMTM)
Therapeutic
area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country:
Trial
details:
Latest
news:
- • On April 27, 2016, Audentes Therapeutics announced that data related to its product candidate AT132 will be presented at the 19th Annual Meeting of the American Society of Gene and Cell Therapy to be held in Washington, D.C. on May 4-7th, 2016. The promising results from a long-term pre-clinical study of AT132 in a dog model of X-linked Myotubular Myopathy (XLMTM) support the advancement of this novel therapeutic into clinical development. Data from an ongoing study in the naturally occurring dog model of XLMTM confirm earlier observations demonstrating the durable effects of treatment with AT132. At more than three years following a single IV administration of AT132, the dogs in the study showed prolonged survival and near-normal levels of neurological function, strength, gait and respiratory function, as well as normal muscle pathology as compared with age-matched untreated XLMTM and unaffected controls. These data have been presented by Matthew Elverman, M.D. of the University of Washington School of Rehabilitation Medicine.
Is
general: Yes
