AAV9 viral vector containing HEXA and HEXB genes (TSHA-101) / Taysha Gene Therapies - LARVOL DELTA

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=3 | Active, not recruiting | Sponsor: Dr. Anupam Sehgal | Trial primary completion date: Mar 2023 ➔ Mar 2027

Gene therapy • Trial primary completion date • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=3 | Active, not recruiting | Sponsor: Dr. Anupam Sehgal | Suspended ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=6 | Suspended | Sponsor: Dr. Anupam Sehgal | Active, not recruiting ➔ Suspended

Trial suspension • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: Dr. Anupam Sehgal | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2; N=6; Recruiting; Sponsor: Dr. Anupam Sehgal; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Gene Therapies

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis (clinicaltrials.gov) - P1/2; N=6; Not yet recruiting; Sponsor: Dr. Anupam Sehgal

Clinical • New P1/2 trial • Gene Therapies