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

Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates (ESGCT 2024) - "During preclinical characterization of investigational gene therapies, biodistribution in NHPs of TSHA-101, TSHA-102, TSHA-105 and TSHA-120 was evaluated after administration by the IT (all products) or ICM (TSHA-102 only) routes. Overall, biodistribution analysis in Taysha’s NHP studies showed that both the IT and ICM routes led to comparable widespread AAV9 distribution throughout the CNS, achieving brain levels of ∼3 – 5 × 105 vg/µg at comparable doses and timing. These findings support the use of lumbar IT administration as an effective, procedurally simple approach for rAAV9 dosing of the CNS."

Gene therapy • Gene Therapies

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