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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: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 29 Apr 2025

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At a glance

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

Most Recent Events

  • 25 Mar 2025 Planned number of patients changed from 12 to 6.
  • 03 Jun 2024 According to a Grace Science media release, company that the 2nd NGLY1 Deficiency patient was successfully treated with GS-100 in May. A dose escalation is planned for the 3rd patient (second cohort) later this summer provided all safety signals remain positive in the first cohort.
  • 03 Jun 2024 According to a Grace Science media release, company announced that the IND for GS-100 for NGLY1 Deficiency, was accepted into the FDA's Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.

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