Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GS-100 · 1 trial · 1 indication
Incidence of adverse events (AEs) and serious AEs (SAEs)
Improvement in one or more domains of the 88-item Gross Motor Function Measure (GMFM-88) from Baseline to Week 52
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | GS-100 Low Dose Level: 4e14 total vector genomes (vg) for 6-18-year-olds (fully enrolled, 2 participants) |
| Cohort 2 | EXPERIMENTAL | GS-100 Mid Dose Level: 1e15 total vector genomes (vg) for 13-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 3 | EXPERIMENTAL | GS-100 High Dose Level: 3e15 total vector genomes (vg) for 6-18-year-olds, 2.6e15 vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 4 | EXPERIMENTAL | GS-100 Intermediate Dose Level: 2e15 total vector genomes (vg) for 6-18-year-olds, 1.75e15 total vg for 2-5-year-olds (fully enrolled, 1 participant) |
| Pivotal Cohort | EXPERIMENTAL | GS-100 Selected Dose Level: 1e15 total vector genomes (vg) for 6-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 3 participants) |
| Name | Type | Description |
|---|---|---|
| GS-100 | GENETIC | A single intracerebroventricular (ICV) dose of GS-100 |
Inclusion Criteria: * Patients must be 2 to 18 years of age, inclusive, at the time of signing the informed consent form (ICF) * Patients with a documented diagnosis of NGLY1 Deficiency based on detection of biallelic variants in the NGLY1 gene via molecular genetic sequencing * Elevated GNA levels...
GS-100 is an investigational gene therapy being developed for the treatment of NGLY1 deficiency, a rare genetic disorder. It is currently in Phase 3 clinical development and has not been approved by the FDA. The therapy is designed to address the underlying cause of the condition.
GS-100 is being developed by Grace Therapeutics, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of GS-100 in patients with NGLY1 deficiency. Grace Therapeutics is publicly traded under the ticker symbol GRCE.
GS-100 is currently in Phase 3 clinical development for NGLY1 deficiency. It is an investigational gene therapy that has not yet received regulatory approval. The ongoing Phase 3 trial is active but not recruiting participants, indicating that enrollment has been completed.
GS-100 is being evaluated in a Phase 3 clinical trial with the identifier NCT06199531. This trial is assessing the safety and efficacy of GS-100 gene therapy in patients with NGLY1 deficiency. The study is active but not recruiting, with an enrollment target of 10 participants in the United States.
Yes, GS-100 is a gene therapy. It is being developed as a treatment for NGLY1 deficiency, a rare genetic condition. The therapy is currently in Phase 3 clinical trials to evaluate its safety and efficacy in affected patients.