4D-310 / 4D Molecular Therap - LARVOL DELTA

Low Pre-existing AAV-Neutralizing Titers Detected Using a Cell-Based Assay Did Not Impact 4D-310 Safety or Efficacy in Fabry Cardiomyopathy Patients (ASGCT 2025) - "Our data underscore that cell-based assays to detect AAV capsid neutralizing factors, in particular assays without a confirmatory Protein A/G/L depletion step, may over-estimate the prevalence of pre-existing AAV neutralizing antibodies. Implementation of ligand-binding total ADA assays for patient screening can help minimize the risk of excluding patients who may otherwise benefit from AAV gene therapy. Disease Focus of Abstract:Inborn Errors in Metabolism"

Clinical • Cardiomyopathy • Cardiovascular • Fabry Disease • Gene Therapies • Genetic Disorders

4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: 4D Molecular Therapeutics | Active, not recruiting ➔ Recruiting | Trial completion date: Jan 2030 ➔ Jun 2030 | Trial primary completion date: Jan 2026 ➔ Jun 2026

Enrollment open • Trial completion date • Trial primary completion date • Fabry Disease • Gene Therapies • Genetic Disorders

4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: 4D Molecular Therapeutics | Trial completion date: May 2028 ➔ Jan 2030 | Trial primary completion date: May 2024 ➔ Jan 2026

Gene therapy • Trial completion date • Trial primary completion date • Fabry Disease • Gene Therapies • Genetic Disorders

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: 4D Molecular Therapeutics | Trial completion date: Jun 2027 ➔ Jun 2030 | Trial primary completion date: Jun 2023 ➔ Jan 2026

Gene therapy • Trial completion date • Trial primary completion date • Fabry Disease • Gene Therapies • Genetic Disorders

4DMT to Present Interim Clinical Data from INGLAXA Phase 1/2 Trials of 4D-310 for Fabry Disease Cardiomyopathy at 19th Annual WORLDSymposium (GlobeNewswire) - "4D Molecular Therapeutics...today announced that the Company will host a live webcast on February 22 to present interim clinical data from its 4D-310 INGLAXA Phase 1/2 clinical trials for the treatment of Fabry disease cardiomyopathy and will give an update on clinical development plans....The Company also announced cardiac efficacy data from the 4D-310 INGLAXA Phase 1/2 clinical trials will be presented in a platform presentation at the 19th annual WORLDSymposium™ in Orlando, Florida."

P1/2 data • Fabry Disease

4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: 4D Molecular Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Fabry Disease • Gene Therapies • Genetic Disorders

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: 4D Molecular Therapeutics | Recruiting ➔ Active, not recruiting | Trial primary completion date: Feb 2024 ➔ Jun 2023

Enrollment closed • Gene therapy • Trial primary completion date • Fabry Disease • Gene Therapies • Genetic Disorders

4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: 4D Molecular Therapeutics

New P1/2 trial • Fabry Disease • Gene Therapies • Genetic Disorders

[VIRTUAL] Development of Novel AAV-Based Gene Therapy for Cardiac Disease (ATS 2021) - "After completing all preclinical and IND-enabling studies in normal and Fabry mice, we performed a dose-ranging study in NHPs using C102 carrying GLA (4D-310)...The tropism of this novel AAV variant, C102, represents a significant advance over conventional AAVs and is well suited for the development of gene therapies for diseases where cardiac involvement is primary or syndromic, such as FD. Since the therapeutic transgene can readily be switched (modularity), the C102 capsid could be the gene therapy vehicle to treat any number of cardiovascular diseases."

Cardiomyopathy • Fabry Disease • Gene Therapies • Heart Failure

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: 4D Molecular Therapeutics; Trial primary completion date: Feb 2027 ➔ Feb 2024

Clinical • Trial primary completion date • Fabry Disease • Gene Therapies • Genetic Disorders

[VIRTUAL] A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression and Safety in Non-Human Primates (ASGCT 2021) - "4D-310 demonstrated safety and secreted high-level and stable AGA activity in plasma and throughout the heart and blood vessel walls. These data in a primate model demonstrate the potential of 4D-310 for a dual mechanism-of-action in Fabry disease by leveraging cell-autonomous production of AGA within the heart plus high plasma enzyme levels. 4D-310 has the potential to address the unmet medical need in FD patients, including AGA expression within cardiovascular tissues."

Clinical • Cardiovascular • Fabry Disease • Gene Therapies • Genetic Disorders • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Classic Fabry Disease (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: 4D Molecular Therapeutics; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Fabry Disease • Gene Therapies

US Fabry disease gene therapy rapid screening [Google translation] (Medical News) - "In the US, 4D-310, the 4DMT's Fabry disease gene therapy, has been designated as a subject for rapid FDA review....A clinical trial in the second half of this year is ahead, and the results of preclinical trials show that it is better delivered to target tissues related to diseases such as the heart, kidneys, and blood vessels, 4DMT introduced."

Review • Fabry Disease • Genetic Disorders

4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease (Businesswire) - "4D Molecular Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to 4D-310 for Fabry disease, a debilitating lysosomal storage disorder caused by a deficiency of alpha-galactosidase A (AGA) enzyme activity."

Fast track designation • Fabry Disease • Genetic Disorders

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Classic Fabry Disease (clinicaltrials.gov) - P1/2; N=18; Not yet recruiting; Sponsor: 4D Molecular Therapeutics

Clinical • New P1/2 trial • Fabry Disease • Gene Therapies