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AXV-102

Development stage: Preclinical
Lead developer: Axovia Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intravenous, Subretinal, Intravitreal

Overview

AXV-102 is an investigational gene therapy being developed by Axovia Therapeutics, a subsidiary of Jaguar Gene Therapy, for the treatment of Bardet-Biedl Syndrome 1 (BBS1), a rare genetic disorder classified as a ciliopathy. The therapy utilizes an adeno-associated virus (AAV) vector to deliver corrective genetic material targeting the underlying cause of BBS1. As of April 2025, AXV-102 is in preclinical development and has not yet entered clinical trials. The mechanism involves gene replacement or supplementation via AAV vectors targeting the Bardet-Biedl syndrome 1 protein.

Targets

BBS1 (Bardet-Biedl syndrome 1 protein)

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