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A Multicenter, Nonrandomized, Open-label,Dose Escalation Clinical Trial to Assess the Safety and Efficacy of EXG001 307 After Intravenous Injection in Patients with Spinal Muscular Atrophy Type 1

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Trial Profile

A Multicenter, Nonrandomized, Open-label,Dose Escalation Clinical Trial to Assess the Safety and Efficacy of EXG001 307 After Intravenous Injection in Patients with Spinal Muscular Atrophy Type 1

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 27 Sep 2024

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At a glance

  • Drugs EXG001-307 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Sponsors Hangzhou Jiayin Biotech

Most Recent Events

  • 19 Sep 2024 Planned End Date changed from 1 Mar 2025 to 1 Aug 2025.
  • 19 Sep 2024 Planned primary completion date changed from 1 Mar 2025 to 1 Aug 2025.
  • 25 Apr 2024 According to an Exegenesis Bio media release, company expects to file our US IND for SMA Type 1 in 4Q 2024.

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