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Research programme: AAV-based gene therapy - Rocket Pharmaceuticals/Temple University

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Drug Profile

Research programme: AAV-based gene therapy - Rocket Pharmaceuticals/Temple University

Alternative Names: AAV9-BAG3; BAG3 gene therapy; Recombinant AAV-based gene therapy - Rocket Pharmaceuticals

Latest Information Update: 25 Aug 2025

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At a glance

  • Originator Temple University
  • Developer Rocket Pharmaceuticals; Temple University
  • Class Cardiovascular therapies; Gene therapies; Heart failure therapies
  • Mechanism of Action BAG3 protein replacements; Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical CNS disorders; Dilated cardiomyopathy
  • No development reported Heart failure

Most Recent Events

  • 30 Jun 2025 IND submission anticipate in first half of 2025
  • 12 Mar 2025 Preclinical development is ongoing in USA for Dilated cardiomyopathy (Rocket Pharmaceuticals pipeline, March 2025)
  • 28 Sep 2023 No recent reports of development identified for preclinical development in Dilated cardiomyopathy in USA (Parenteral)

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