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rAAV Olig001 ASPA - Myrtelle

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Drug Profile

rAAV Olig001 ASPA - Myrtelle

Alternative Names: AVASPA; MYR-101; rAAV-Olig001-ASPA

Latest Information Update: 04 Apr 2024

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At a glance

  • Originator Pfizer
  • Developer Myrtelle
  • Class Gene therapies
  • Mechanism of Action Amidohydrolase replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Canavan disease
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Canavan disease

Most Recent Events

  • 02 Apr 2024 rAAV Olig001 ASPA - Myrtelle receives Regenerative Medicine Advanced Therapy (RMAT) status for Canavan disease in USA
  • 31 May 2023 Myrtelle completes enrolment in its phase I/II trial for Canavan disease in USA (NCT04833907)
  • 16 May 2023 Efficacy data from a phase I/II trial in Canavan disease released by Myrtelle

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