GS1191 / Gritgen Therap - LARVOL DELTA

Long-Term Efficacy and Safety of Lower Dose AAV8-FVIII Gene Therapy in Chinese Patients with Severe Hemophilia A (ISTH 2026) - P=N/A, P1 | "GS1191-0445 is an investigational gene therapy vector based on adeno-associated virus serotype 8 (AAV8)...No significant safety concerns were observed. DOI*10.1016/j.rpth.2026.103564"

Clinical • First-in-human • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=7 | Active, not recruiting | Sponsor: Gritgen Therapeutics Co., Ltd.

New P1/2 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Low dose AAV8-FVIII gene therapy on Chinese severe Hemophilia A patients with sustainable efficacy and safety (ASH 2025) - P=N/A | "An investigator-initiated trial (IIT) (NCT04728841) and a phase Ⅰ/Ⅱ trial (CTR20231730) of GS1191-0445 have beenconducted in Chinese patients with severe Hemophilia A to evaluate long-term efficacy and safety.Nineteen adults with severe Hemophilia A (factor Ⅷ level ≤1%) have been recruited and injected with asingle treatment of AAV8-hFⅧ infusion...Twoimmunosuppressive regimens, prophylactic prednisone or prednisone plus tacrolimus, can be selectedto control the potential AAV capsid immune response from the 7-day prior to study drug infusion to atleast 12 weeks post study drug administration...One of themuses Nadroparin calcium injection and Aspirin enteric-coated tablets to prevent blood clots...Gene therapy with AAV8-hFVIII vector in Chinese patients with Hemophilia A demonstrated a good safetyprofile and sustained, clinically relevant benefit, as measured by a substantial reduction in annualizedrates of bleeding events and complete cessation of prophylactic..."

Clinical • Gene therapy • Cardiovascular • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A (clinicaltrials.gov) - P3 | N=50 | Recruiting | Sponsor: Gritgen Therapeutics Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

The first in China! Huayi Lejian GS1191-0445 injection officially entered the multi-center phase III clinical trial stage (bydrug.pharmcube.com) - "...Suzhou Huayi Lejian Biotechnology Co., Ltd. (hereinafter referred to as "Huayi Lejian')...reached an agreement with the Center for Drug Evaluation of the National Medical Products Administration (CDE, NMPA) on the Phase III clinical trial plan for its independently developed GS1191-0445 injection, marking that Huayi Lejian GS1191-0445 injection has officially entered the Phase III clinical trial stage...GS1191-0445 injection is a gene therapy drug based on an adeno-associated virus (AAV) vector that targets the core cause of hemophilia A - coagulation factor VIII (FVIII) gene deficiency."

Trial status • Hemophilia A

To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia a (clinicaltrials.gov) - P3 | N=50 | Not yet recruiting | Sponsor: Gritgen Therapeutics Co., Ltd.

New P3 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases