In gene therapy, most companies obsess over the message. The code. The payload. Affinia Therapeutics was built around a harder truth that cuts through the noise in today’s startup news cycle. The message means nothing if the messenger never arrives. Founded in 2019 and headquartered in Waltham, Massachusetts, Affinia exists because delivery has been the quiet bottleneck holding gene therapy back from scale. The science traces to Luk Vandenberghe, Ph.D., co-inventor of AAV9, the vector behind Zolgensma, and to a founding belief that precision beats volume every time.
Affinia does not gamble with biology. It engineers it. Its ART platform, short for Affinia Rationally designed Therapeutics, rethinks the capsid, the payload, and the manufacturing process as a single integrated system. Heart. CNS. Muscle. Liver detargeting by design, not cleanup. This is gene therapy built with a destination in mind, not a hope that the body figures it out on the way. The platform is tissue agnostic, but the execution is exacting.
That philosophy sharpens around AFTX-201, targeting BAG3 dilated cardiomyopathy, a genetic heart disease affecting more than 70,000 patients across the US, Europe, and the UK. A 1-time intravenous administration delivering a fully human, full-length BAG3 transgene. Preclinical data showed restored cardiac function in animal models where conventional capsids failed at the same dose. Even with current standard of care, nearly 25% of BAG3 patients still require a heart transplant. That reality defines the urgency behind the science.
The leadership bench reads like lived experience, not theory. Rick Modi, CEO and board member, helped bring Zolgensma to market at AveXis before its acquisition by Novartis. Sean P. Nolan, former AveXis CEO, serves as Chairman. Charles Albright, Ph.D., CSO, led gene editing development at Editas after more than 12 years at Bristol Myers Squibb. Petra Kaufmann, M.D., CMO, oversaw clinical development for Zolgensma and held senior roles at the NIH. Paula Cobb serves as CBO. Rob Aboud co-founded the company and served as its former CLO. This is a team that has carried therapies from concept to clinic and beyond.
Capital has followed discipline. More than $210M raised across 3 rounds. A $110M Series B in 2021 led by EcoR1 Capital and Farallon Capital Management. A $40M Series C in October 2025 led by New Enterprise Associates, with Eli Lilly & Company joining as a new strategic investor. Add a multi-year research collaboration with Vertex Pharmaceuticals worth $80M upfront and up to $1.6B in milestones and royalties, and the signal is clear to anyone tracking serious startup news in biotech.
Affinia expects to submit its IND for AFTX-201 in Q4 2025, with the UPBEAT Phase 1/2 trial anticipated in early 2026, pending FDA acceptance. The company is moving from platform promise to clinical proof, and the window between those two states is where durable companies separate themselves from the rest of the startup news feed.
