Drug Profile

AAV8 RK BBS10

Alternative Names: AAV-BBS10; AAV8-RK-BBS10

Latest Information Update: 06 Feb 2025

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At a glance

Originator MeiraGTx
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Preclinical Bardet-Biedl Syndrome

Most Recent Events

21 Nov 2024 Pharmacodynamic and adverse events data from a preclinical studies in Bardet-Biedl syndrome released by MeiraGTx
14 Nov 2024 Preclinical trials in Bardet-Biedl Syndrome in USA (Subretinal)
14 Nov 2024 MieraGTX receives Rare Pediatric Disease Designations from the FDA for AAV8 RK BBS10 in Bardet Biedl Syndrome

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