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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

Approved

Sickle Cell Disease

Casgevy (exa-cel)|🇨🇭 CRISPR Therapeutics / Vertex

CRISPR-Cas9Ages: 12+ years3 countries
Approved

Transfusion-Dependent Beta-Thalassemia

Casgevy (exa-cel)|🇨🇭 CRISPR Therapeutics / Vertex

CRISPR-Cas9Ages: 12+ years3 countries
Recruiting

B-cell Non-Hodgkin Lymphoma, B-cell Malignancies

CTX110|🇨🇭 CRISPR Therapeutics

CRISPR-Cas9Ages: 18+ years1 country
Recruiting

Hyperlipidemia, Cardiovascular Disease

CTX310|🇨🇭 CRISPR Therapeutics

CRISPR-Cas9Ages: 18-75 years2 countries
IND Preparation

Alpha-1 Antitrypsin Deficiency

CTX460|🇨🇭 CRISPR Therapeutics

SyNTase (novel editor)Ages: 18-65 years1 country
Recruiting

Autoimmune Diseases

CTX611|🇨🇭 CRISPR Therapeutics

CRISPR-Cas9Ages: 18-65 years1 country
Planned

Cardiovascular Disease

CTX340|🇨🇭 CRISPR Therapeutics

CRISPR-Cas9Ages: 18-75 years1 country
Recruiting

Type 1 Diabetes

VCTX210|🇨🇭 CRISPR Therapeutics / ViaCyte

CRISPR-Cas9Ages: 18-65 years2 countries
Recruiting

Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

Nexiguran Ziclumeran (NTLA-2001)|🇺🇸 Intellia Therapeutics

CRISPR-Cas9Ages: 18-80 years4 countries
Recruiting

Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)

Nexiguran Ziclumeran (NTLA-2001)|🇺🇸 Intellia Therapeutics

CRISPR-Cas9Ages: 18-80 years3 countries
Active (not recruiting)

Hereditary Angioedema (HAE)

Lonvoguran Ziclumeran (NTLA-2002)|🇺🇸 Intellia Therapeutics

CRISPR-Cas9Ages: 18+ years4 countries
Recruiting

Alpha-1 Antitrypsin Deficiency (AATD)

NTLA-3001|🇺🇸 Intellia Therapeutics

CRISPR-Cas9 (gene insertion)Ages: 18-65 years1 country
Recruiting

Sickle Cell Disease

OTQ923 (with Novartis)|🇺🇸 Intellia Therapeutics / Novartis

CRISPR-Cas9Ages: 18-40 years1 country
Recruiting

Sickle Cell Disease

BEAM-101|🇺🇸 Beam Therapeutics

Base Editing (adenine)Ages: 18-40 years1 country
Recruiting

T-cell Acute Lymphoblastic Leukemia

BEAM-201|🇺🇸 Beam Therapeutics

Base EditingAges: 18+ years1 country
Recruiting

Alpha-1 Antitrypsin Deficiency (AATD), Hereditary ATTR Amyloidosis

BEAM-302|🇺🇸 Beam Therapeutics

Base EditingAges: 18-65 years5 countries
Recruiting

Chronic Granulomatous Disease (CGD)

PM359|🇺🇸 Prime Medicine

Prime EditingAges: 2-40 years1 country
IND Preparation

Alpha-1 Antitrypsin Deficiency

PM-AATD|🇺🇸 Prime Medicine

Prime EditingAges: 18-65 years1 country
Recruiting

Heterozygous Familial Hypercholesterolemia (HeFH)

VERVE-101|🇺🇸 Verve Therapeutics

Base Editing (adenine)Ages: 18-75 years2 countries
Recruiting

Heterozygous Familial Hypercholesterolemia (HeFH)

VERVE-102|🇺🇸 Verve Therapeutics

Base Editing (adenine)Ages: 18-75 years1 country
Recruiting

Sickle Cell Disease, Transfusion-Dependent Beta-Thalassemia

Reni-cel (EDIT-301)|🇺🇸 Editas Medicine

CRISPR-Cas12aAges: 12-35 years1 country
Recruiting

Relapsed/Refractory B-cell Non-Hodgkin Lymphoma

CB-010|🇺🇸 Caribou Biosciences

CRISPR (chRDNA)Ages: 18+ years1 country
Recruiting

Relapsed/Refractory Multiple Myeloma

CB-011|🇺🇸 Caribou Biosciences

CRISPR (chRDNA)Ages: 18+ years1 country
Recruiting

HIV-1 Infection

EBT-101|🇺🇸 Excision BioTherapeutics

CRISPR-Cas9 (multiplex)Ages: 18-65 years1 country
Recruiting

Urinary Tract Infections (E. coli)

LBP-EC01|🇺🇸 Locus Biosciences

CRISPR-Cas3Ages: 18+ years1 country
Recruiting

Systemic Lupus Erythematosus (SLE)

ET-01 (CAR-T)|🇨🇳 EdiGene Inc.

CRISPR-Cas9Ages: 18-65 years1 country
Recruiting

Relapsed/Refractory B-cell Malignancies

ET-02 (CAR-T)|🇨🇳 EdiGene Inc.

CRISPR-Cas9Ages: 18-70 years1 country
Recruiting

Duchenne Muscular Dystrophy (DMD)

HG-DMD|🇨🇳 HuidaGene Therapeutics

CRISPR-Cas12Ages: 6-18 years1 country
Recruiting

Neovascular Age-related Macular Degeneration (nAMD)

HG-nAMD|🇨🇳 HuidaGene Therapeutics

CRISPR-Cas12Ages: 50+ years1 country
Recruiting

Transfusion-Dependent Beta-Thalassemia

CS-101|🇨🇳 CorrectSequence Therapeutics

Base Editing (proprietary tBE)Ages: 12-35 years1 country
Recruiting

Familial Chylomicronemia Syndrome (FCS)

CS-APOC3|🇨🇳 CorrectSequence Therapeutics

Base EditingAges: 18-65 years1 country
Recruiting

Primary Hyperoxaluria Type 1 (PH1)

YOLT-203|🇨🇳 YolTech Therapeutics

CRISPR-Cas12Ages: 12+ years2 countries
Recruiting

Refractory Systemic Lupus Erythematosus

BT-SLE|🇨🇳 Base Therapeutics (Shanghai)

Base EditingAges: 18-65 years1 country
IND Preparation

Atherosclerotic Cardiovascular Disease (ASCVD)

GEB-200|🇭🇰 GenEditBio

CRISPR-Cas9Ages: 18-75 years2 countries
Recruiting

Age-related Macular Degeneration (AMD)

TGT-001|🇰🇷 ToolGen

CRISPR-Cas9Ages: 50+ years1 country
Recruiting

HPV-associated Cervical Neoplasia

TGT-HPV|🇰🇷 ToolGen

CRISPR-Cas9Ages: 25-65 years1 country
Recruiting

Multiple Solid Tumors, Melanoma, NSCLC

BNT-Oncology Pipeline|🇩🇪 BioNTech

CRISPR-Cas9 / mRNAAges: 18+ years2 countries
Active

Blood Disorders, Blindness, Congenital Heart Disease

Bayer-CRISPR JV|🇩🇪 Bayer / CRISPR Therapeutics

CRISPR-Cas9Ages: TBD2 countries
Recruiting

Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

UCART19|🇫🇷 Cellectis

TALENAges: 1-70 years2 countries
Recruiting

Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Neoplasm

UCART123|🇫🇷 Cellectis

TALENAges: 18-70 years2 countries
Active (not recruiting)

Hemophilia A

Giroctocogene Fitelparvovec (SB-525)|🇺🇸 Sangamo Therapeutics / Pfizer

Zinc Finger Nuclease (ZFN)Ages: 18-65 years4 countries
Recruiting

Testicular Cancer, Ovarian Cancer, Endometrial Cancer, NSCLC

BNT211|🇩🇪 BioNTech

CRISPR-Cas9Ages: 18+ years3 countries

Not finding what you need?

New gene editing clinical trials are registered regularly. Here are more ways to find trials:

Data last updated: 2026-03-29. We verify trial information against official sources including ClinicalTrials.gov, company press releases, and FDA filings.