Cure
Overview
A look at 13 startups and public companies advancing genetic medicines, cell-based therapies, and next-generation treatment platforms.
Gene and cell therapies are becoming core tools for treating cancer, rare diseases, autoimmune conditions, and degenerative disorders, with a growing number of products now reaching patients. The main question facing the field is no longer whether these approaches can work, but how to scale them, manufacture them reliably, and deploy them in ways that are both clinically and economically viable.
“What excites me is the breadth of tools now in play, from traditional AAV and lentiviral vectors to ex vivo CD34-positive stem cell gene therapies and increasingly sophisticated gene-modified cell therapies like CAR-T,” said Johnathon Anderson, PhD, a research scientist, CEO of Peptide Systems, and associate professor at the UC Davis School of Medicine. “We’re seeing these products move beyond hematologic malignancies into solid tumors and autoimmune disease.”
Plus, the steady number of FDA approvals for cell and gene therapies over the last few years demonstrates that this field of study is no longer a boutique experiment, Anderson notes. Instead, it is an evolving and maturing field of study that is touching real people with cancer, rare diseases, autoimmune diseases, and more. “Overall, gene therapies let researchers intervene at the level of the causal pathway. And the lessons we are learning will spill over into RNA medicines, protein engineering, and even small molecule drug discovery. So gene therapy…is accelerating our understanding of human biology in ways that benefit the whole therapeutic ecosystem.”
For this list, we are grouping gene therapies and gene-modified cell therapies together, reflecting how closely these fields now intersect across discovery, manufacturing, and clinical development. Advances in vector engineering, cell processing, automation, and regulatory pathways are increasingly shared, even as the underlying modalities differ.
While there are hundreds of companies working in this space, we selected 14 that stand out for their scientific ambition, clinical progress, or platform potential. Together, they offer a snapshot of where gene and cell therapy is headed in 2026, and how the next generation of genetic medicines is taking shape.
Genetix Biotherapeutics (formerly Bluebird Bio)
Sector: Gene therapy
HQ: Somerville, MA
Year Founded: 1992
Key Leaders: David Meek, CEO; Brian Riley, President, Chief Technical Officer; Joanne Lager, MD, Chief Medical Officer; Tom Klima, Chief Commercial and Operating Officer; Wendy DiCicco, Chief Financial Officer; Adrian Chaisson, Chief of Staff
Number of Employees: 450
Stage: Privately-held, commercial stage company
Financial Snapshot: $1.12 billion raised over the years
Key Products: Gene therapies for genetic diseases including FDA-approved treatments for sickle cell β-thalassemia, and cerebral adrenoleukodystrophy (Zynteglo, Skysona, Lyfgenia) Recent Highlights: In late 2025, Bluebird Bio was purchased and rebranded as a privately-held, commercial stage company Genetix Therapeutics. Since then, the company has presented data to the American Society of Hematology on two of its therapies and the impact on patients. The company describes this data as the most robust long-term dataset in ex vivo gene therapy for hemoglobinopathies.
Kite Pharma
Sector: Oncology cell therapy
HQ: Santa Monica, CA
Year Founded: 2009 (acquired by Gilead Sciences in 2017)
Key Leaders: Cindy Perettie, EVP and Global Head; Laura Alquist, VP and Global Head of Technical Operations; Kevin Cognetti, VP of Corporate Strategy and Planning
Number of Employees: ~ 4,000
Stage: Public (GILD)
Financial Snapshot: Acquired by Gilead Sciences for $11.9 billion in 2017
Key Products: CAR-T therapies for oncology including FDA-approved drugs Yescarta and Tecartus
Recent Highlights: Kite Pharma, a Gilead company, has presented a number of recent updates on their CAR T-cell therapies to the American Society of Hematology. It specifically showcased the progress of Yescarta, an immunotherapy used to treat certain types of blood cancers.
Sarepta Therapeutics
Sector: Gene therapy
HQ: Cambridge, MA
Year Founded: 1980
Key Leaders: Douglas Ingram, CEO; Ian M. Estepan, President and COO; Louise R. Rodino-Klapac, PhD, President, R&D and Technical Operations; Diane Berry, PhD, EVP, Chief Global Policy & Advocacy Officer; Patrick E. Moss, PharmD, EVP, Chief Commercial Officer
Number of Employees: ~ 1,400
Stage: Public (SRPT)
Financial Snapshot: $2.29 billion market cap
Key Products: Elevidys is the company’s FDA-approved drug in gene therapy. The company also has RNA-based therapies on the market.
Recent Highlights: In November 2025, Sarepta announced that the U.S. Food and Drug Administration approved dosing in Cohort 8 of the ENDEAVOR study, which will evaluate an enhanced immunosuppressive regimen alongside Elevidys in people with Duchenne muscular dystrophy. The update reflects ongoing collaboration with regulators as the company studies approaches to manage safety risks while advancing its gene therapy program.
Krystal Biotech
Sector: Gene therapy
HQ: Pittsburgh, PA
Year Founded: 2016
Key Leaders: Krish Krishnan, Chairman and CEO; Suma Krishnan, President, Research & Development; Kathryn Romano, EVP & Chief Accounting Officer
Number of Employees: ~ 300
Stage: Public (KRYS)
Financial Snapshot: A publicly traded company (KRYS) with a multibillion-dollar market cap; supported by growing commercial revenue from FDA-approved gene therapy, Vyjuvek
Key Products: Gene therapy for DEB (dystrophic epidermolysis bullosa) with the drug, Vyjuvek, approved by the FDA
Recent Highlights: In September 2025, the FDA approved a label update for Vyjuvek that expands eligible patients to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibility with respect to its application and management of wound dressings. The FDA granted the company an End of Phase 2 meeting to discuss potential development pathways to support the registration of inhaled treatment for non-small cell lung cancer.
Elevate Bio
Sector: Cell and gene therapy platform
HQ: Waltham, MA
Year Founded: 2017
Key Leaders: Ger Brophy, PhD, Interim CEO; Larry Lockwood, PhD, Chief Commercial Officer; Michael Paglia, MS, CTO; Amy Pooler, PhD, Chief Scientific Officer
Number of Employees: ~ 400
Stage: Private ($401M series D)
Financial Snapshot: $1.3 billion raised
Notable Investors: Lee Family Office, Novo Nordisk, Woodline Partners, Fidelity Management & Research, GG 1978, Vertex Ventures, Invus, Surveyor Capital
Key Products: The company offers a set of platform technologies such as gene editing. They also provide expertise in design, development, and manufacturing of both ex vivo and in vivo cell and gene therapies.
Recent Highlights: In August, 2025, the company received comprehensive certification from the Initiative for Certification of Manufacturing Capabilities (ICMC) program for its viral gene delivery, non-viral gene delivery, and cell therapy manufacturing capabilities. In 2019, AlloVir, formerly known as ViraCyte, joined the company’s growing portfolio. AlloVir, a late-clinical stage allogeneic cell therapy company.
Inceptor Bio
Sector: Cell therapy
HQ: Morrisville, NC
Year Founded: 2020
Key Leaders: Shailesh Maingi, Founder & Executive Chair; Matthias Schroff, PhD, CEO; Abe Maingi, Chief Business Officer
Number of Employees: 24
Stage: Series A funding, clinical stage company
Financial Snapshot: Most recent deal raised $21 million
Notable Investors: Kineticos Life Sciences Management (Kineticos Ventures, Kineticos Disruptor Fund), Next Coast Ventures
Key Products: The company’s pipeline is focused on next-gen cell therapies (CAR-T, CAR-M, CAR-NK) for solid tumors, using their proprietary OUTLAST platform. Their lead candidate IB-T101 (CD70-targeted CAR-T) is in clinical trials for clear cell Renal Cell Carcinoma (ccRCC).
Recent Highlights: In February 2025, the company announced that the first patient has been dosed in the clinical trial of IB-T101. This trial furthers the company's efforts to address the unmet medical needs of people with clear cell Renal Cell Carcinoma (ccRCC). That same month, the company closed a $21 million series A2 financing, which was led by a syndicate of insiders including Kineticos Life Sciences.
Chimeron Bio
Sector: Gene therapy
HQ: Philadelphia, PA
Year Founded: 2015
Key Leaders: Kevin Heyeck, CEO and Board Member; Monica Coleman, CFO; Antonin de Fougerolles, PhD, Board Member; Afshin Safavi, PhD, Chairman of the Board
Number of Employees: 14
Stage: Series A
Financial Snapshot: Private, Series A-stage company that has raised funding in the low-teen millions from a mix of institutional, strategic, and public-sector investors
Notable Investors: Monfort, Dreavent, Pennsylvania Department of Community and Economic Development, Alexandria LaunchLabs
Key Products: The company is developing multiplexed in vivo CAR-T cell therapies for cancer, with a focus on reducing the complexity of traditional CAR-T manufacturing. The company is also advancing immuno-oncology programs, including therapeutic approaches for genetically defined cancers.
Recent Highlights: In 2024, the company received the National Institutes of Health’s Small Business Innovation Research Innovative Concept Award through the National Cancer Institute. This funding will support the development of a cancer vaccine against an inherited form of colorectal cancer.
Forte Biosciences
Sector: Clinical-stage biopharmaceuticals (autoimmune and inflammatory diseases)
HQ: Dallas, TX
Year founded: 2007
Key leaders: Paul A. Wagner, PhD, CEO and Chairperson of the Board; Antony A. Riley, CFO; Christopher Roenfeldt, COO; Barbara Finck, MD, Senior Medical Clinician
Number of Employees: 16
Stage: Public company (FBRX)
Financial Snapshot: Publicly traded, small-cap biotechnology company (FBRX) with a lean operating footprint
Key Products: Forte Biosciences is developing protein-based immunomodulatory therapies targeting the IL-2 pathway for autoimmune and autoimmune-related diseases. Its lead candidate, FB-102, is being evaluated for conditions including celiac disease.
Recent Highlights: In 2025, Forte completed a public offering to support continued development of its pipeline and presented Phase 1b clinical data for FB-102, reporting favorable safety and early biological activity signals in patients with celiac disease.
Genascence
Sector: Gene therapy for musculoskeletal and degenerative diseases
HQ: Palo Alto, California
Year Founded: 2017
Key Leaders: Thomas W. Chalberg, PhD, Founder, Chairman, and CEO; Annahita Keravala, PhD, Founder, Chief Scientific Officer; Jeymi Tambia, MBChB, Chief Medical Officer; Christopher H. Evans, PhD, Founder; Steven C. Ghivizzani, PhD, Founder; Paul Robbins, PhD, Founder
Number of Employees: <10
Stage: Series A
Financial Snapshot: A private, early-stage company backed by a mix of public-sector and strategic funding
Notable investors: California Institute for Regenerative Medicine, U.S. Department of Defence, Deepwork Capital, Pacira BioSciences, University of Florida Research Foundation
Key Products: Gene therapy for osteoarthritis including its IL-1Ra gene therapy
Recent Highlights: In May 2025, the company announced positive 12-month safety and biomarker results from the Phase 1b DONATELLO clinical trial evaluating GNSC-001, a gene therapy blocking interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). Then, in July, the U.S. Food and Drug Administration (FDA) granted the company the Regenerative Medicine Advanced Therapy (RMAT) designation to GNSC-001.
American Gene Technologies
Sector: Gene therapy
HQ: Rockville, MD
Year Founded: 2008
Key Leaders: Jeff Galvin, Founder and CEO; Drew Palin, MD, President; Marcus Conant, MD, Chief Medical Advisor; Barry H. Wells, MD, VP Investor Relations
Number of Employees: 25
Stage: Private, clinical-stage
Financial Snapshot: Raised a total of $52.8 million over eight funding rounds
Notable Investors: Carat Venture Partners, MCMA Ventures, Opus8, Ossian Capital, Ride Wave Ventures
Key Products: The company has a gene delivery platform that accelerates the development of a wide variety of drug candidates and its AGT103-T, an HIV therapeutic, is currently in Phase 1 clinical trials. It also has a growing patent portfolio.
Recent Highlights: In 2025, American Gene Technologies shared updated data from its completed Phase 1 clinical study of AGT103-T, an autologous cell and gene therapy being evaluated as a functional cure strategy for HIV. The company reported encouraging safety results and evidence of sustained reductions in intact HIV proviral DNA in evaluable participants, supporting continued regulatory discussions around next development steps for the program.
Arsenal Bio
Sector: Cell therapy, immuno-oncology
HQ: San Francisco, CA
Year Founded: 2018
Key leaders: Ken Drazan, MD, Chairman, CEO, and Co-Founder; Gift Ngarmchamnanrith, MD, Chief Medical Officer; W. Nicholas Haining, BM, BCh, Chief Scientific Officer; Tim Sirichoke, Chief Technical Operations Officer
Number of employees: ~ 300
Stage: Series C
Financial Snapshot: Closed an oversubscribed $325 million Series C financing round in 2024
Notable Investors: ARCH Venture Partners, Milky Way Investments Group, Regeneron Ventures, NVentures (NVIDIA’s venture capital arm), Luma Group, T. Rowe Price Associates, Inc., Rock Springs Capital, Parker Institute for Cancer Immunotherapy (PICI), SoftBank Vision Fund 2, Bristol-Myers Squibb Company, Westlake Village BioPartners, Kleiner Perkins, Byers Capital, and Hitachi Ventures
Key Products: The company’s pipeline includes cell therapies programmed with multiple coordinated synthetic drug functions. One key product includes AB-3028 for prostate cancer.
Recent Highlights: In January 2025, the company announced that Bristol-Myers Squibb exercised its exclusive license option for ArsenalBio’s AB-4000 series, the lead collaboration program under its multi-program agreement. This collaboration advances next-generation T cell therapies for the treatment of solid tumors. In September of 2025, the company went through a restructuring to position itself as a clinical stage company.
Klotho Neurosciences
Sector: Gene and cell therapy for neurodegenerative and age-related disorders
HQ: Omaha, NE
Year Founded: 2004
Key leaders: Joseph Sinkule, PharmD, Founder, CEO, Chairman of the Board; Jeffrey LeBlanc, CFO; Miguel Chillón Rodriguez, PhD, Chief Scientific Officer and Consultant; Shalom Hirshman, MD, Medical Advisor and Director
Number of Employees: <5
Stage: Public company (KLTO)
Financial Snapshot: Market cap around $28 million–$36 million as of late 2025
Key Products: Klotho Neurosciences is advancing early-stage gene and cell therapy programs centered on the Klotho pathway, with preclinical candidates targeting conditions such as Alzheimer’s disease and amyotrophic lateral sclerosis. Its approach leverages gene delivery technologies to modulate production of the Klotho protein, which is implicated in aging and cognitive function.
Recent Highlights: In 2025, Klotho Neurosciences won the BioTech Breakthrough “Cell Therapy Innovation of the Year” award, recognizing progress in its Klotho-based therapeutic platform. The company also reported ongoing work on manufacturing and development of its gene therapy candidates and expanded its research portfolio through collaborations aimed at broadening potential indications.
Ambulero
Sector: Vascular gene therapy
HQ: Miami, FL
Year Founded: 2019
Key Leaders: Robert L. Buchanan, CEO; Carlton Anderson, COO; Omaida Caridad Velazquez, MD, Chief Medical Officer; Zhao-Jun Liu, MD, PhD, Chief Scientific Officer
Number of Employees: 5
Stage: Seed
Financial Snapshot: Raised $5.5 million in two rounds
Notable Investors: Orphinic Scientific, Endless Frontier Labs, Ventac Partners, University of Miami
Key Products: The company is developing two treatment modalities. One product is the AMB-301, which is a gene therapy for treating a rare form of vascular disease. The other product is AMB-201, which is a gene therapy that uses a clinically tested AAV vector to deliver and over-express E-selectin in ischemic tissues.
Recent Highlights: In June 2025, the company announced the start of Investigational New Drug (IND)-enabling studies for its AMB-301, gene therapy product designed to treat a rare vascular disorder called Buerger’s Disease. (AMB-301 received US FDA Orphan Drug Status in 2021.)
