Drug Profile

AXV 101

Alternative Names: AAV9-BBS1; AXV-101

Latest Information Update: 23 May 2025

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At a glance

Originator Axovia Therapeutics
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.

Yes - Bardet-Biedl Syndrome
New Molecular Entity No

Highest Development Phases

Preclinical Bardet-Biedl Syndrome

Most Recent Events

13 May 2025 Pharmacodynamics and adverse event data from a preclinical trial released by Axovia Therapeutics
13 May 2025 Axovia Therapeutics plans a first in human phase I/II trial for Bardet-Biedl Syndrome (Subretinal) in mid 2025 in United Kingdom
28 Apr 2025 AXV 101 is still in preclinical trials for Bardet-Biedl Syndrome in the United Kingdom

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