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Cell and gene therapy developers

163 articles

Borealis Biosciences

Borealis Biosciences is a Vancouver, Canada-based biotechnology company developing xRNA medicines for kidney diseases, launched by Versant Ventures and Novartis in August 2024 with $150 million in funding.

BridgeBio

BridgeBio Pharma, Inc. is a commercial-stage biopharmaceutical company in Palo Alto, California, founded in 2015, known for its hub-and-subsidiary structure and acoramidis, approved in 2024 for transthyretin amyloid cardiomyopathy.

CAMP4 Therapeutics

CAMP4 Therapeutics Corporation, formerly Marauder Therapeutics, is a clinical-stage biopharmaceutical company in Cambridge, Massachusetts, founded in 2015, whose antisense oligonucleotide drugs upregulate disease-linked genes; its lead candidate CMP-002 targets SYNGAP1-related disorder.

CARsgen Therapeutics

CARsgen Therapeutics (科济药业) is a Shanghai-based biopharmaceutical company, founded in 2014 and listed in Hong Kong, that develops CAR T-cell therapies for blood cancers and solid tumors.

Cartesian Therapeutics

Cartesian Therapeutics is a clinical-stage biotechnology company in Frederick, Maryland, developing mRNA-engineered CAR-T therapies for autoimmune diseases; its lead candidate, Descartes-08, is in Phase 3 for myasthenia gravis.

Castle Creek Biosciences

Castle Creek Biosciences is a private cell and gene therapy company in Exton, Pennsylvania, developing D-Fi, a Phase 3 gene therapy for dystrophic epidermolysis bullosa.

CellChina (西比曼)

CellChina (西比曼), also known as Cellular Biomedicine Group (CBMG), is a Shanghai-based clinical-stage company developing CAR-T, TCR-T, TIL, and stem-cell therapies for cancer and degenerative disease.

Cellular Biomedicine Group

Cellular Biomedicine Group was a clinical-stage biopharmaceutical company developing CAR-T immune-cell therapies for cancer, founded in 2009, Nasdaq-listed until its 2021 privatization, and renamed AbelZeta Pharma in 2023.

Celularity

Celularity Inc. is a cellular and regenerative medicine company in Florham Park, New Jersey, founded by Robert Hariri and Peter Diamandis in 2018 to develop placenta-derived therapies.

Century Therapeutics

Century Therapeutics is a Philadelphia-based, clinical-stage biotechnology company created by Versant Ventures in 2018 that develops allogeneic, off-the-shelf cell therapies for cancer and autoimmune disease from induced pluripotent stem cells.

Chroma Medicine

Chroma Medicine was a Boston-based biotechnology company, founded in 2020, that developed single-dose epigenetic editing therapies that regulate genes without cutting DNA; it merged into nChroma Bio in December 2024.

Complement Therapeutics

Complement Therapeutics GmbH is a clinical-stage biotechnology company, founded in 2021 as a University of Manchester spinout, developing CTx001, a gene therapy for geographic atrophy secondary to age-related macular degeneration.

CoRegen, Inc.

CoRegen, Inc. is a Houston biotechnology startup developing CRISPR-edited regulatory T-cell therapies for solid tumors, which received FDA clearance for its first trial in 2026.

Cure Genetics (苏州克睿基因生物科技有限公司) (Suzhou Kerui Gene Biotechnology)

Cure Genetics (苏州克睿基因生物科技有限公司, Suzhou Kerui Gene Biotechnology) is a clinical-stage cell and gene therapy biotechnology company founded in Suzhou, China, in 2016, known for its VELP AAV vector-engineering and AIMS CAR-NKT platforms.

CUTISS

CUTISS AG is a Zurich-based biotechnology company spun off from the University of Zurich in 2017 that develops personalized skin grafts, most notably denovoSkin for severe burns, now in Phase 3 trials.

Dicerna Pharmaceuticals

Dicerna Pharmaceuticals was a Lexington, Massachusetts biopharmaceutical company that developed RNA interference (RNAi) therapeutics on its GalXC platform, with lead candidate nedosiran for primary hyperoxaluria.

DTx Pharma

DTx Pharma was a San Diego biotechnology company, founded in 2017 by Artie Suckow, that developed siRNA therapeutics using its FALCON platform and was acquired by Novartis in 2023.

Dyne Therapeutics

Dyne Therapeutics is a US clinical-stage biotechnology company founded in 2019 in Massachusetts that develops oligonucleotide therapies for neuromuscular diseases using its FORCE muscle-delivery platform.

EdiGene (博雅辑因)

EdiGene (博雅辑因) is a Chinese gene-editing therapeutics company founded in 2015 and headquartered in Beijing, with subsidiaries in Guangzhou and Cambridge, Massachusetts, developing CRISPR-based therapies including ET-01 for beta-thalassemia.

eGenesis

eGenesis is a clinical-stage biotechnology company in Cambridge, Massachusetts, founded in 2016 from George Church's Harvard research, that develops gene-edited pig kidneys for transplanting into kidney-failure patients.

Encoded Therapeutics

Encoded Therapeutics is a US precision gene therapy company in South San Francisco, California, founded in 2014 by Kartik Ramamoorthi and Stephanie Tagliatela, developing ETX101 for Dravet syndrome.

Ensoma

Ensoma is a Boston-based genomic medicines company founded in 2019 that develops one-time, in vivo engineered hematopoietic cell therapies on its Engenious virus-like-particle platform for genetic diseases, immune disorders, and cancer.

Fangtuo Bio (Fangtuo Biopharma)

Fangtuo Bio, known in Chinese as 芳拓生物 and internationally as Frontera Therapeutics, is a clinical-stage Chinese gene therapy company founded in 2019, developing rAAV therapies for ophthalmic and cardiovascular diseases.

FerGene

FerGene was a gene-therapy company created in 2019 as a Ferring Pharmaceuticals subsidiary, backed by Blackstone with over $570 million, to commercialize nadofaragene firadenovec for bladder cancer in the United States.

Funove (复诺健生物)

Funove, formally 复诺健生物科技有限公司 and known internationally as Virogin Biotech Ltd., is a Chinese-Canadian biotechnology company founded in 2015 that develops oncolytic-virus and mRNA cancer therapies.

Gameto, Inc.

Gameto, Inc. is a clinical-stage biotechnology company founded in 2020 by Dina Radenkovic and Martin Varsavsky, known for Fertilo, a stem-cell therapy that matures eggs outside the body to reduce hormone injections in IVF.

Generation Bio

Generation Bio Co., formerly Torus Therapeutics, was a Cambridge, Massachusetts biotechnology company that developed non-viral, redosable genetic medicines and was acquired by XOMA Royalty in February 2026.

Genespire

Genespire is a Milan-based biotechnology company founded in 2020 as a spin-out of the San Raffaele Telethon Institute, developing single-dose gene therapies for rare pediatric diseases.

Gracell Bio

Gracell Bio, formally Gracell Biotechnologies Inc., was a Chinese clinical-stage biotech in Suzhou developing CAR-T cell therapies for cancer and autoimmune disease, acquired by AstraZeneca in 2024.

Gracell Biotechnologies (亘喜生物)

Gracell Biotechnologies (亘喜生物) was a Suzhou, China biotech founded in 2017 that developed CAR-T therapies for cancer and autoimmune disease and was acquired by AstraZeneca in 2024.