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AAVB 039

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Drug Profile

AAVB 039

Alternative Names: AAV.ABCA4.intein; AAV8.ABCA4; AAVB-039; Dual adeno-associated virus based Stargardt disease gene therapy - AAVantgarde

Latest Information Update: 08 Oct 2025

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At a glance

  • Originator AAVantgarde Bio
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action ATP binding cassette transporter subfamily A member 4 replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Stargardt disease
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Stargardt disease

Most Recent Events

  • 02 Oct 2025 AAVB 039 receives Orphan Drug status for Stargardt disease in USA
  • 02 Oct 2025 Medicines and Healthcare products Regulatory Agency (MHRA) approves Clinical Trial Authorisation (CTA) for AAVB 039 in Stargardt disease in United Kingdom
  • 02 Oct 2025 AAVantgarde Bio plans clinical trial for Stargardt disease in United Kingdom

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