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A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients with NGLY1 Deficiency

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Trial Profile

A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients with NGLY1 Deficiency

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 07 May 2026

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At a glance

  • Drugs GS 100 (Primary)
  • Indications Inborn genetic disorders
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Sponsors Grace Science

Most Recent Events

  • 04 May 2026 Protocol has been amended to include two new arms (Experimental: Cohort 4 and Experimental: Pivotal Cohort) , it has changed from 3 to 5.
  • 04 May 2026 Planned End Date changed from 31 Jan 2028 to 1 Mar 2031.
  • 04 May 2026 Planned primary completion date changed from 31 Jan 2028 to 1 Mar 2027.

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