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AXV 101

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Drug Profile

AXV 101

Alternative Names: AAV9-BBS1; AXV-101

Latest Information Update: 09 Jan 2026

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At a glance

  • Originator Axovia Therapeutics
  • Class Gene therapies
  • Mechanism of Action Bbs1 protein replacements; 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

  • 06 Jan 2026 Axovia Therapeutics plans a first in human trial for Bardet-Biedl Syndrome in early 2026
  • 04 Sep 2025 The MHRA cleared CTA for AXV 101 in Bardet-Biedl Syndrome
  • 13 May 2025 Pharmacodynamics and adverse event data from a preclinical trial released by Axovia Therapeutics

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