Date: 2018-02-06
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 14th Annual WORLDSymposium 2018
Company: Audentes Therapeutics (USA - CA)
Product: AT982
Action
mechanism:
- gene therapy. Pompe disease is caused by mutations in the gene encoding the lysosomal enzyme alpha-glucosidase, or GAA, which results in a deficiency of GAA protein and leads to the accumulation of glycogen. AT982 consists of an AAV8 vector that delivers a GAA gene expression cassette containing a novel hybrid promoter designed to increase GAA activity in targeted tissues, including skeletal and cardiac muscle, the nervous system and the liver. Audentes holds exclusive global rights to both AAV8 and AAV9 in Pompe disease from RegenXBio.
Disease: Pompe disease
Therapeutic
area: Rare diseases - Genetic diseases
Country:
Trial
details:
Latest
news:
- • On February 6, 2018, Audentes Therapeutics announced it has selected an optimized clinical development candidate for its Pompe disease program. Data supporting the selection were presented during a general session at the 14th Annual WORLDSymposium 2018 in Poster #123, titled: "A Novel Hybrid Promoter Directing AAV-mediated Expression of Acid Alpha-Glucosidase to Liver, Muscle, and CNS Yields Optimized Outcomes in a Mouse Model of Pompe Disease."
- This poster describes the results of an AAV gene therapy construct selection study conducted in a well-characterized mouse model of Pompe disease. The study evaluated both AAV8 and AAV9 capsid serotypes, paired with novel promoter elements designed to target GAA expression in a range of tissues, including skeletal and cardiac muscle, the nervous system and the liver. The study included a range of doses and utilized a broad battery of biochemical and immunological assays. Of the six vectors evaluated, an AAV8 vector with a novel hybrid promoter was selected as the clinical development candidate, AT982. This vector was confirmed to provide GAA expression and activity in target tissues, improved muscle pathology and a favorable immunogenicity profile.
- Audentes plans to file an IND for AT982 in the second quarter of 2018 and plans to initiate a Phase 1 / 2 clinical study in the fourth quarter of 2018.
Is
general: Yes
