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Zertomibgene cazparvovec - Tenaya Therapeutics

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Drug Profile

Zertomibgene cazparvovec - Tenaya Therapeutics

Alternative Names: TN-201

Latest Information Update: 12 Jun 2026

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At a glance

  • Originator Tenaya Therapeutics
  • Class Cardiovascular therapies; Gene therapies
  • Mechanism of Action Gene transference; Myosin-binding protein C replacements
  • Orphan Drug Status

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

    Yes - Hypertrophic cardiomyopathy
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Hypertrophic cardiomyopathy

Most Recent Events

  • 03 Jun 2026 US FDA accepts Rare Disease Evidence Principles (RDEP) process for Zertomibgene cazparvovec for Hypertrophic cardiomyopathy (In children) in USA
  • 03 Jun 2026 Zertomibgene cazparvovec - Tenaya Therapeutics receives Priority Medicine (PRIME) status for Hypertrophic cardiomyopathy in European Union
  • 03 Jun 2026 Efficacy, pharmacodynamics and adverse events data from a phase I/II MyPEAK-1 trial in hypertrophic cardiomyopathy released by Tenaya Therapeutics

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