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GS 100

Drug Profile

GS 100

Alternative Names: AAV9-based NGLY1 gene therapy - Grace Science; GS-100

Latest Information Update: 07 Jun 2024

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At a glance

  • Originator Grace Science
  • Class Gene therapies; Recombinant proteins
  • Mechanism of Action Aminohydrolase- eplacements; Gene transference
  • Orphan Drug Status

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

    Yes - Inborn genetic disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Congenital disorders of glycosylation

Most Recent Events

  • 03 Jun 2024 US FDA selects GS-100 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program
  • 13 Feb 2024 Phase-I/II clinical trials in Congenital disorders of glycosylation (In children, In adolescents) in USA (Intraventricular, infusion) (NCT06199531)
  • 31 Dec 2023 GS 100 receives Fast Track designation for congenital disorders of glycosylation in USA
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