April 28, 2025 — Frontera Therapeutics announced today that its recombinant adeno-associated virus (rAAV) gene therapy, FT-017, has received clinical trial clearance from both the U.S. Food and Drug Administration (FDA) and China's National Medical Products Administration (NMPA). This approval marks FT-017 as the first AAV-based gene therapy for hypertrophic cardiomyopathy (HCM) caused by MYBPC3 gene mutations to enter clinical trials in China and the fourth Frontera product to secure dual approvals in the U.S. and China.

Addressing a Critical Genetic Heart Condition

Hypertrophic cardiomyopathy (HCM) is a hereditary heart disease characterized by thickening of the ventricular walls, primarily affecting the left ventricle. With a global prevalence of 0.2%–0.5% (approximately 15–20 million patients) and 0.076% in China (about 1 million patients), HCM is linked to serious complications, including atrial fibrillation, stroke, heart failure, and sudden cardiac death. Mutations in the MYBPC3 gene, which encodes cardiac myosin-binding protein C, disrupt normal protein synthesis, leading to sarcomere dysfunction, myocyte hypertrophy, and cardiac remodeling.

Current treatments, such as symptomatic relief or myosin inhibitors, fail to:

    ✖    Correct underlying genetic defects

    ✖    Halt disease progression

    ✖    Reduce risks of arrhythmias or sudden cardiac death

This creates an urgent need for therapies that address the root cause of HCM and improve patient outcomes.

FT-017: A Transformative Gene Therapy

FT-017 leverages rAAV technology to deliver a codon-optimized human MYBPC3 gene directly to cardiomyocytes via intravenous infusion. This approach enables long-term, stable expression of functional cMyBP-C protein, restoring sarcomere structure and function. FT-017 offers the potential to:

    ✓   Treat HCM at its genetic source

    ✓   Slow, prevent, or reverse disease progression

    ✓   Reduce risks of atrial fibrillation, arrhythmias, and sudden  cardiac death

A Milestone for Frontera and Global Gene Therapy

"FT-017 represents a significant leap forward in gene therapy for cardiovascular diseases," said Dr. Xinyan Li, CEO of Frontera Therapeutics. "As our fourth product to gain dual US and China clearance, and the first AAV-based therapy for HCM in China, FT-017 underscores Frontera's leadership in innovative gene therapies. With only one comparable therapy, TN-201, in Phase Ib/II globally, we are committed to accelerating FT-017's clinical development to bring hope to HCM patients worldwide."