Find Gene Editing Clinical Trials
Search 42 active gene editing and gene therapy clinical trials worldwide. Find trials by condition, location, phase, and technology.
Important: This tool helps you discover gene editing clinical trials. It does NOT determine your eligibility. Contact the trial site directly or speak with your doctor. Trial availability and eligibility criteria change frequently.
Showing 42 of 42 trials
Transfusion-Dependent Beta-Thalassemia
Casgevy (exa-cel)|🇨🇭 CRISPR Therapeutics / Vertex
B-cell Non-Hodgkin Lymphoma, B-cell Malignancies
CTX110|🇨🇭 CRISPR Therapeutics
Hyperlipidemia, Cardiovascular Disease
CTX310|🇨🇭 CRISPR Therapeutics
Alpha-1 Antitrypsin Deficiency
CTX460|🇨🇭 CRISPR Therapeutics
Autoimmune Diseases
CTX611|🇨🇭 CRISPR Therapeutics
Cardiovascular Disease
CTX340|🇨🇭 CRISPR Therapeutics
Type 1 Diabetes
VCTX210|🇨🇭 CRISPR Therapeutics / ViaCyte
Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
Nexiguran Ziclumeran (NTLA-2001)|🇺🇸 Intellia Therapeutics
Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)
Nexiguran Ziclumeran (NTLA-2001)|🇺🇸 Intellia Therapeutics
Hereditary Angioedema (HAE)
Lonvoguran Ziclumeran (NTLA-2002)|🇺🇸 Intellia Therapeutics
Alpha-1 Antitrypsin Deficiency (AATD)
NTLA-3001|🇺🇸 Intellia Therapeutics
Sickle Cell Disease
OTQ923 (with Novartis)|🇺🇸 Intellia Therapeutics / Novartis
Sickle Cell Disease
BEAM-101|🇺🇸 Beam Therapeutics
T-cell Acute Lymphoblastic Leukemia
BEAM-201|🇺🇸 Beam Therapeutics
Alpha-1 Antitrypsin Deficiency (AATD), Hereditary ATTR Amyloidosis
BEAM-302|🇺🇸 Beam Therapeutics
Chronic Granulomatous Disease (CGD)
PM359|🇺🇸 Prime Medicine
Alpha-1 Antitrypsin Deficiency
PM-AATD|🇺🇸 Prime Medicine
Heterozygous Familial Hypercholesterolemia (HeFH)
VERVE-101|🇺🇸 Verve Therapeutics
Heterozygous Familial Hypercholesterolemia (HeFH)
VERVE-102|🇺🇸 Verve Therapeutics
Sickle Cell Disease, Transfusion-Dependent Beta-Thalassemia
Reni-cel (EDIT-301)|🇺🇸 Editas Medicine
Relapsed/Refractory B-cell Non-Hodgkin Lymphoma
CB-010|🇺🇸 Caribou Biosciences
Relapsed/Refractory Multiple Myeloma
CB-011|🇺🇸 Caribou Biosciences
HIV-1 Infection
EBT-101|🇺🇸 Excision BioTherapeutics
Urinary Tract Infections (E. coli)
LBP-EC01|🇺🇸 Locus Biosciences
Systemic Lupus Erythematosus (SLE)
ET-01 (CAR-T)|🇨🇳 EdiGene Inc.
Relapsed/Refractory B-cell Malignancies
ET-02 (CAR-T)|🇨🇳 EdiGene Inc.
Duchenne Muscular Dystrophy (DMD)
HG-DMD|🇨🇳 HuidaGene Therapeutics
Neovascular Age-related Macular Degeneration (nAMD)
HG-nAMD|🇨🇳 HuidaGene Therapeutics
Transfusion-Dependent Beta-Thalassemia
CS-101|🇨🇳 CorrectSequence Therapeutics
Familial Chylomicronemia Syndrome (FCS)
CS-APOC3|🇨🇳 CorrectSequence Therapeutics
Primary Hyperoxaluria Type 1 (PH1)
YOLT-203|🇨🇳 YolTech Therapeutics
Refractory Systemic Lupus Erythematosus
BT-SLE|🇨🇳 Base Therapeutics (Shanghai)
Atherosclerotic Cardiovascular Disease (ASCVD)
GEB-200|🇭🇰 GenEditBio
Age-related Macular Degeneration (AMD)
TGT-001|🇰🇷 ToolGen
HPV-associated Cervical Neoplasia
TGT-HPV|🇰🇷 ToolGen
Multiple Solid Tumors, Melanoma, NSCLC
BNT-Oncology Pipeline|🇩🇪 BioNTech
Blood Disorders, Blindness, Congenital Heart Disease
Bayer-CRISPR JV|🇩🇪 Bayer / CRISPR Therapeutics
Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia
UCART19|🇫🇷 Cellectis
Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm
UCART123|🇫🇷 Cellectis
Hemophilia A
Giroctocogene Fitelparvovec (SB-525)|🇺🇸 Sangamo Therapeutics / Pfizer
Testicular Cancer, Ovarian Cancer, Endometrial Cancer, NSCLC
BNT211|🇩🇪 BioNTech
Not finding what you need?
New gene editing clinical trials are registered regularly. Here are more ways to find trials:
- Search ClinicalTrials.gov directly — the official U.S. database of all registered clinical trials worldwide.
- View our full Pipeline Tracker — a curated list of all gene editing programs in development, including preclinical programs.
Data last updated: 2026-03-29. We verify trial information against official sources including ClinicalTrials.gov, company press releases, and FDA filings.