Top 25 Gene delivery platform companies based in the United States

“AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…”

“Forge, a member of Ajinomoto Bio-Pharma Services, is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea…”

“REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.”

“Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.”

“Tessera Therapeutics is a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim…”

“Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.”

“ArsenalBiosciences, Inc, is a clinical stage programmable cell therapy company focused on the realization of solid tumor cell therapy to defeat cancer.”

“Andelyn Biosciences is a gene therapy CDMO dedicated to Turning Hope Into Reality™.”

“For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases.”

“Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.”

“Founded in 2014 in Indianapolis, IN, Genezen is a best-in-class gene therapy CDMO with over a decade of experience supporting the cell and gene therapy manufacturing market…”

“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…”

“Voyager Therapeutics, Inc.(Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure –…”

“Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using our proprietary, comprehensive metagenomics-derived genome…”

“Atara Biotherapeutics Inc, is a leading allogeneic T-cell immunotherapy company pioneering the development of transformative off-the-shelf therapies for patients with serious…”

“Verve Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, is developing a new class of genetic medicines for cardiovascular disease with the potential to transform…”

“Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop long-term curative therapies, as well as other…”

“Poseida was acquired by Roche in early 2025 and is now part of the Roche Group.”

“Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…”

“Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…”

“Century Therapeutics is an innovative biotechnology company that is building an industry-leading, allogeneic, iPSC-derived cell therapy platform that integrates cutting-edge gene…”