Affinia Therapeutics has secured $40 million in Series C financing to advance its lead gene therapy program AFTX-201, a potential first-in-class treatment for BAG3 dilated cardiomyopathy (DCM). The round was led by New Enterprise Associates (NEA) with participation from new investor Eli Lilly & Company and existing investors including Alexandria Venture Investments, Atlas Venture, Avidity Partners, F-Prime, GV (formerly Google Ventures), Mass General Brigham Ventures, and Perceptive Advisors.
Novel Gene Therapy Approach for Rare Heart Disease
AFTX-201 represents a significant advancement in gene therapy for cardiovascular disease, utilizing Affinia's proprietary cardiotropic capsid technology. The therapy is designed to deliver a fully human, full-length BAG3 transgene using a novel capsid engineered for efficient and selective cardiac transduction at low doses. The treatment is intended as a simple one-time intravenous administration.
Preclinical studies in animal disease models demonstrated compelling efficacy data. AFTX-201 improved BAG3 protein levels in the heart and completely restored cardiac function, whereas the same gene construct using a conventional capsid at the same dose failed to achieve such benefits.
Addressing Significant Unmet Medical Need
BAG3 DCM is a serious, inherited heart condition with high mortality and represents a significant unmet medical need. The disease affects more than 70,000 patients in the U.S., Europe, and U.K. regions alone. The BAG3 gene encodes for a protein critical to the normal structure and function of heart cells. Patients with BAG3 DCM have mutations in the BAG3 gene and deficiency in functional BAG3 protein, resulting in early onset heart failure that progresses rapidly.
Despite current standard of care treatments, almost 25% of patients require heart transplants, highlighting the urgent need for effective therapeutic interventions.
Clinical Development Timeline
The financing will fund AFTX-201's progression toward clinical trials. Affinia expects to submit an Investigational New Drug (IND) application in the fourth quarter of 2025. If accepted by the FDA, the company plans to initiate the UPBEAT trial, a Phase 1/2 clinical trial for patients affected by BAG3 DCM, in the first quarter of 2026.
AFTX-201 is currently undergoing IND-enabling studies, which were initiated following Affinia's successful pre-IND meeting with the FDA. This regulatory milestone demonstrates the FDA's recognition of the therapy's potential and the company's development strategy.
Advanced Capsid Engineering Technology
Affinia has applied its generative AI discovery platform to engineer novel myotropic capsids that target the heart and skeletal muscles while avoiding the liver. This liver detargeting approach represents a significant advancement in gene therapy safety and efficacy, potentially reducing off-target effects that have challenged other AAV-based therapies.
"This financing recognizes the potential of our novel capsids and the value of our lead program AFTX-201, bringing it one step closer to potentially become the first-in-class and best-in-class genetic medicine to treat BAG3 DCM," said Rick Modi, Affinia's Chief Executive Officer.
Strategic Investment Validation
The participation of Eli Lilly & Company as a new strategic investor alongside leading venture capital firms validates Affinia's approach and pipeline potential. Ed Mathers, General Partner at NEA and Affinia Board Member, noted the syndicate's shared enthusiasm about "Affinia's science and robust pipeline to treat devastating cardiovascular and neurological diseases."
The financing will also support advancement of Affinia's broader gene therapy pipeline targeting additional devastating diseases in cardiovascular and neurological indications, leveraging the company's proprietary next-generation capsids, payloads, and manufacturing approaches.
