Date: 2016-04-27
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the American Society of Gene and Cell Therapy Annual Meeting
Company: Audentes Therapeutics (USA - CA)
Product: AT132
Action
mechanism:
- gene therapy. X-linked Myotubular Myopathy (XLMTM) is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age. XLMTM is caused by mutations in the MTM1 gene, which encodes myotubularin, a protein that plays an important role in the development, maintenance and function of skeletal muscle cells. AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene. Audentes is developing AT132 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. 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.
Is
general: Yes
