Hengyu Bio Congratulates Frontera Therapeutics on FT-017 Receiving Dual Clinical Trial Approvals from NMPA and FDA, Marking a New Milestone in Cardiovascular Gene Therapy

Apr 28, 2025 13:23 CST Updated 13:23

Frontera Therapeutics

Innovative Gene Therapy Drug R&D and Producer

April 28——Frontera TherapeuticsAnnouncement`, its independently developed`Recombinant Adeno-Associated Virus (rAAV) Gene Therapy Product FT-017At the same time, obtainClinical Trial Permits from China's National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA)`, for the treatment of`MYBPC3Gene Mutation-Associated Hypertrophic Cardiomyopathy (HCM). This is the fourth innovative gene therapy product of Frontera Therapeutics to achieve dual filing and approval in both China and the United States, and alsoChina's First Clinical Trial of AAV Gene Therapy for Cardiovascular System Diseases。

Hengyu BioProvided international submission compliance for this projectVirus Clearance Validation (VC)Biological safety testing services in ChinaInternational compliance quality and technology system, rapid delivery and expertiseProject Management in the Industry, Safeguarding the Launch and Global Expansion of Innovative Drugs.

AboutMYBPC3HCM Associated with Gene Mutations

Hypertrophic Cardiomyopathy (HCM) is a primary cardiomyopathy characterized by ventricular wall thickening (mainly left ventricular hypertrophy). It is the most common hereditary heart disease in clinical practice and is closely associated with atrial fibrillation, stroke, heart failure, and sudden cardiac death. The global prevalence of HCM is 0.2%-0.5%, with approximately 15 to 20 million patients. In China, the prevalence of HCM is about 0.076%, with approximately 1 million patients. The main cause of HCM is pathogenic variants in genes encoding myocardial sarcomere-related proteins (such as the cardiac myosin-binding protein C gene, MYBPC3), which result in insufficient synthesis of normal proteins, leading to structural or functional abnormalities in sarcomeres or sarcomere-related proteins. This induces histological and morphological changes in cardiomyocytes, causing cellular hypertrophy, disarrayed arrangement, interstitial fibrosis, and myocardial remodeling.

Current treatment options (including symptomatic treatment and inhibition of myosin's contractile function) can only provide temporary relief of symptoms.
✖ Unable to repair pathogenic gene defects
✖ Unable to delay or halt disease progression
✖ Unable to prevent the risks of atrial fibrillation, arrhythmia, and sudden cardiac death

Therefore, there is a significant unmet clinical need to delay disease progression, improve quality of life, and reduce the risk of sudden death in patients with hereditary HCM.

AboutFT-017

Based on the main pathogenesis of HCM, targetingMYBPC3Gene mutations causing cMyBP-C deficiency or dysfunction are addressed by FT-017 through gene therapy: intravenous infusion of a codon-optimized humanMYBPC3Gene transfer into cardiomyocytes enables specific, long-term, and stable expression of normal human cMyBP-C, restoring the normal structure and function of myocardial sarcomeres.

In terms of therapeutic advantages, compared with traditional therapies, FT-017 has the potential to:

✓ Treat the root causeMYBPC3HCM Caused by Gene Mutations

✓ Delay, halt, or even reverse disease progression

✓ Prevent the occurrence of atrial fibrillation, arrhythmia, or sudden cardiac death.

Dr. Xinyan Li, Co-founder & CEO of Frontera TherapeuticsSaid: "FT-017It is the fourth independently developed innovative product by Frontera Therapeutics to gain approval from both Chinese and U.S. regulatory authorities for clinical trials. The expansion into the cardiovascular disease field not only demonstrates Frontera Therapeutics' outstanding innovation capabilities in gene therapy but also represents a significant attempt for China’s AAV gene therapy products in cardiovascular system diseases. Currently, internationally, there is only one similar drug, TN-201, undergoing Phase Ib/II clinical trials. We will accelerate the clinical trials of FT-017 to bring new treatment options to patients with hereditary hypertrophic cardiomyopathy worldwide as soon as possible.”

*Source: Frontera Therapeutics official website, click to read the original article for more information.

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According to incomplete statistics, Hengyu Bio has successfully contributed with its internationally compliant biosafety testing services.80+Project AcquisitionClinical and Market LaunchTest Batch Document (Including40+Overseas Registration Project), for20+BLA and Commercialization ItemsProviding critical support;The declared projects cover a full range of categories (ADC drugs, mono/di/multi-antibodies, recombinant proteins, CAR-T/CAR-NK, iPSC/MSC, gene editing, AAV gene therapy, etc.).

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About Frontera Therapeutics

Frontera Therapeutics is a clinical-stage international gene therapy drug development company dedicated to providing innovative, high-quality, and affordable rAAV gene therapy drugs for patients with eye diseases, cardiovascular diseases, and other conditions worldwide. The company has established research and development centers, GMP production bases, and clinical operation centers. The founding team and core technology team possess extensive experience in innovative drug development and efficient clinical translation capabilities, and have formed a world-class scientific advisory board and clinical expert advisory committee.

The company's R&D center has established an AAV capsid and gene expression cassette engineering transformation technology platform. Through innovative rational design and in vitro/in vivo optimization screening, the AAV products achieve better tissue targeting, higher and more sustained gene expression, and lower immunogenicity, thereby ensuring excellent efficacy and safety of the products. The company's GMP production base has established a complete Sf9 insect cell-baculovirus expression vector system (Sf9 BEVS) for AAV production and quality control and has successfully achieved 500L scale production with product quality meeting the requirements for clinical trial medications.

The company has developed several pipeline products with "first-in-class" or "best-in-class" potential advantages, covering genetic diseases and major chronic diseases. These products have now entered Phase II clinical trials, with patient recruitment ongoing simultaneously at multiple research centers across China. For more information, please visit the company's website:cn.fronteratherapeutics.com

About Hengyu Bio

HengyuBiologicalRelying on international compliance standards, with outstanding quality and technical strength, we have built a one-stop Biosafety service platform focused on macromolecular drugs and CGT drugs. It has passed audits and certifications such as EU QP, CNAS, and ISO9001, meeting the stringent requirements of global regulatory authorities like NMPA, FDA, and EMA, providing reliable assurance for innovative pharmaceutical companies.Hengyu Bio has cumulatively served nearly 300 biopharmaceutical companies, with rich experience in project and submission practices, and has already provided submission cases for 80+ biologics.20+ BLA and Commercialization ProjectsProvide Critical Support(Including 40+ overseas filingsCase），Including services such as viral clearance validation, cell bank characterization, commercial batch release, detection of replication-competent viruses (RCL, rcAAV, RCR, etc.), and HCD and HCP kits and assays.

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