Product Description
AAVB-081 is an intra-retinal AAV8-based dual hybrid product targeting MYO7A-associated Usher syndrome (USH1B). AAVantgarde’s dual hybrid platform uses two AAV8 vectors, each containing one half of an expression cassette encoding for the Myo7A gene and works at the cell nucleus level, recombining the two halves of the transgene back into a single one within the cell. This technology translates into an efficient recombination that generates therapeutically meaningful protein levels in animal models. (Sourced from: https://www.aavantgarde.com/en/news/aavantgarde-announces-first-patient-dosed-with-aavb-081-in-fih-phase-12-luce-1-study-for-retinitis-pigmentosa-related-to-ush1b/)
Mechanisms of Action: Gene Therapy,MYO7A
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: AAVantgarde Bio Srl
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
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