Gene-editing Clinical Trials
In 2023, therapeutic gene editing moved from promise to reality with the first approved CRISPR-based therapy, CASGEVY, which has since received regulatory clearance in multiple regions for the treatment of sickle cell disease and beta thalassemia.
Our manually curated CRISPR Medicine News Clinical Trials register tracks the advancement of gene-editing technologies in clinical development. The field now encompasses a diverse array of editing tools, including CRISPR-Cas, base editors, prime editors, zinc fingers, TALENs, meganucleases, CAS-CLOVER, RNA editors, and epigenetic-editing technology.
As of February 2025, CRISPR Medicine News monitors approximately 250 clinical trials involving gene-editing therapeutic candidates, with more than 150 trials currently active.
The clinical landscape spans multiple therapeutic areas, including:
- Blood cancers
- Haemoglobinopathies
- Solid cancers
- Viral diseases
- Metabolic disorders
- Autoimmune diseases
- Hereditary amyloidosis
- Inherited eye diseases
- Cardiovascular disease
- Bacterial diseases
- Immunodeficiencies
- Haemophilia
- Neurological conditions
- Muscular dystrophy
- Other rare inherited diseases
As of February 2025, gene editing for blood disorders continues to lead the field, with the majority of Phase 3 trials targeting sickle cell disease and/or beta thalassemia. Phase 3 trials are are also underway in hereditary amyloidosis and immunodeficiencies.
Our clinical trial register is routinely updated to reflect new developments in the field.
Stay informed with our CMN Weekly Newsletter highlighting the latest trial updates and breakthroughs in CRISPR medicine.
This page was last updated on: 28th February 2025
Autoimmune diseases
Bacterial diseases
Cardiovascular diseases
Sponsors:
CRISPR Therapeutics AG
Sponsors:
Verve Therapeutics, Inc.
Haematological malignancies (blood cancers)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.
Sponsors:
The First Affiliated Hospital of Nanjing Medical University
Sponsors:
Institut de Recherches Internationales Servier
Sponsors:
Chinese PLA General Hospital
Sponsors:
German Cancer Research Center
Sponsors:
Fate Therapeutics, Inc
Sponsors:
Caribou Biosciences, Inc.
Sponsors:
Great Ormond Street Hospital for Children NHS Trust
Sponsors:
Institut de Recherches Internationales Servier
Sponsors:
Cellectis S.A.
Sponsors:
Xuzhou Medical University
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
Sponsors:
Fate Therapeutics
Sponsors:
Bioray Laboratories - Shanghai Bangyao Biotechnology Co., Ltd.
Sponsors:
Third Military Medical University
Sponsors:
Xijing Hospital, The Fourth Military Medical University
Sponsors:
Caribou Biosciences, Inc.
Sponsors:
Bioray Laboratories
Sponsors:
Huazhong University of Science and Technology
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.
Sponsors:
Wugen inc.
Sponsors:
Xinqiao Hospital of Chongqing, Second Affiliated Hospital of the Army Medical University, Hematology Department
Sponsors:
Great Ormond Street Hospital
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
Sponsors:
Beam Therapeutics Inc.
Haemoglobinopathies
Sponsors:
Sangamo Therapeutics
Sponsors:
Vertex Pharmaceuticals Incorporated
Sponsors:
923rd Hospital of The People's Liberation Army
Sponsors:
The First Affiliated Hospital, Guangxi Medical University
Sponsors:
Institute of Hematology and Blood Diseases Hospital
Sponsors:
Institute of Hematology & Blood Diseases Hospital, China
Sponsors:
Children's Hospital of Fudan University
Sponsors:
CorrectSequence Therapeutics Co., Ltd
Haemophilia
Hereditary amyloidosis
Sponsors:
Intellia Therapeutics
Immunodeficiencies
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)
Inherited eye diseases
Sponsors:
HuidaGene Therapeutics Co., Ltd.
Sponsors:
HuidaGene Therapeutics Co., Ltd.
Metabolic disorders
Muscular dystrophies
Neurological disease
Sponsors:
Wave Life Sciences Ltd.
Sponsors:
Wave Life Sciences Ltd.
Solid cancers
Sponsors:
Yang Yang, MD, PhD, MSCR, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Sponsors:
Poseida Therapeutics, Inc.
Sponsors:
Huazhong University of Science and Technology
Sponsors:
First Affiliated Hospital, Sun Yat-Sen University
Sponsors:
Huazhong University of Science and Technology
Sponsors:
First Affiliated Hospital of Guangdong Pharmaceutical University
Sponsors:
The First Affiliated Hospital of Guangdong Pharmaceutical University
Viral diseases
Sponsors:
Affiliated Hospital to Academy of Military Medical Sciences
Sponsors:
Mahmoud Ramadan mohamed Elkazzaz
Other rare inherited blood disorders
Sponsors:
Shanghai Children's Medical Center
Other rare inherited disorders
Long-term follow-up (LTFU) studies
Sponsors:
Precision BioSciences, Inc.
Sponsors:
Editas Medicine, Inc.
Sponsors:
Vertex Pharmaceuticals
Sponsors:
Sangamo Therapeutics