Top 84 Gene Therapy startups

Jan 27, 2025 | By Jason Kwon

These startups develop therapies that manipulate gene expression or altere the biological properties of living cells.
1
Country: China | Funding: CN¥200M
Qihe Biotech develops novel genome editing technologies with an emphasis on making them autonomous and programmable.
2
Country: USA | Funding: $543M
Taysha Gene Therapies is a developer of treatments to eradicate severe & life-threatening monogenic diseases of the central nervous system.
3
Country: USA | Funding: $496M
Maze Therapeutics is a operator of a biotechnology firm intended to focused on translating genetic insights into new medicines.
4
Country: UK | Funding: £125M
Purespring Therapeutics is an AAV gene therapy company focused on the kidney globally.
5
Country: USA | Funding: $630.8M
ArsenalBio is focused on integrating technologies such as CRISPR-based genome engineering, scaled and high throughput target identification, synthetic biology, and machine learning to advance a new paradigm to discover and develop in immune cell therapies.
6
Country: USA | Funding: $100M
Judo Bio is a biotechnology company that develops precision therapeutics targeting specific cell populations to address genetic diseases.
7
Country: USA | Funding: $3.4B
Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.
8
Country: USA | Funding: $1.5B
Intellia is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics utilizing a recently developed biological tool known as the CRISPR/Cas9 system. We believe that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing diseased genes in the human body through a single treatment course. We intend to leverage our leading scientific expertise, clinical development experience and intellectual property position to unlock broad therapeutic applications of CRISPR/Cas9 genome editing and develop a potential new drug class.
9
Country: USA | Funding: $1.2B
ElevateBio is a biotechnology company that specializes in cell and gene-based therapies.
10
Country: UK | Funding: $1.1B
Autolus utilises advanced cell programming CAR-T and manufacturing technologies and we have established a development-stage pipeline of products for the treatment of haematological malignancies and solid tumours.
11
Country: USA | Funding: $931.6M
Editas Medicine's mission is to translate its genome editing technology into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level.
12
Country: USA | Funding: $865M
Sana Biotechnology is a developer of engineered cells intended to be used as medicine for patients. It's deploying a platform that can repair and control genes in cells or replace any cell in the body.
13
Country: Netherlands | Funding: $780.7M
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients
 with severe disorders. We believe our multiple partnerships and the regulatory approval of our lead product Glybera in the European Union for a subset of patients with LPLD provides validation for our approach.
14
Country: USA | Funding: $763.8M
Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing potential one-time, life-altering treatments for debilitating genetic diseases. We initially are applying our integrated platform to treat rare diseases where no, or only palliative, therapies exist.
15
Country: USA | Funding: $692.3M
Kriya Therapeutics is a next-generation gene therapy company focused on designing and developing transformative new treatments.
16
Country: USA | Funding: $595M
4D Molecular Therapeutics is unlocking the full potential of the gene therapy field. Our customized AAV vectors can deliver genes to any cell in the body to eradicate disease.
17
Country: USA | Funding: $546.1M
Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered zinc finger DNA-binding proteins (ZFPs) as novel ZFP Therapeutics targeting various monogenic and infectious diseases with unmet medical needs.
18
Country: USA | Funding: $536.4M
Generation Bio is a biotechnology company developing a breakthrough class of genetic medicines to enable a new generation of people unaffected by inherited disease. The company’s therapies are based on its proprietary GeneWave technology, which delivers durable, high levels of gene expression and can be re-dosed to titrate to effect and to sustain impact over a lifetime.
19
Country: USA | Funding: $531.8M
Tessera Therapeutics is pioneering Gene Writing - a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source.
20
Country: USA | Funding: $460.5M
Poseida Therapeutics utilizes best-in-class genome engineering capabilities to develop targeted lifesaving therapeutics.
Editor: Jason Kwon
Jason Kwon is a senior editor for MedicalStartups. He has previously covered the pharmaceutical and medical research industries for FDAnews and worked as a head of marketing for medical startup Sonic Therapeutics. Before that, he co-founded a startup consulting business for emerging entrepreneurial hubs in Asia. Jason graduated from St. Bonaventure University’s journalism school. In his free time, Jason enjoys yoga, watching movie trailers, traveling to places where he can't get cell service. You can contact Jason at jaskwon(at)medicalstartups(dot)com