Diseases

Overview diseases where gene editing tools like CRISPR, Prime Editors, Base Editors, ZFNs, TALENs, CAS-CLOVER, MegaTAL and MegaNucleases are applied.

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Disease

Acute Lymphoblastic Leukemia, ALL

Disease

Acute Myeloid Leukemia, AML

Disease

Alzheimer's disease

Disease

Angelman Syndrome

Disease

Atopic dermatitis

Disease

Beta Thalassemia

Disease

Bordetella pertussis

Disease

Chronic Hepatitis B

Disease

Disease

Cystic Fibrosis

Disease

Diabetes Type 1

Disease

Duchenne Muscular Dystrophy, DMD

Trials

Epithelial Ovarian Cancer, (EOC), NCT05617755
Sponsors:
Arsenal Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Hemophilia B & Mucopolysaccharidosis, MPS, (NCT04628871)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Duchenne Muscular Dystrophy, DMD, (NCT05514249)
Sponsors:
Cure Rare Disease, Inc

IND Enabling

Phase I

Phase II

Phase III

Retinitis Pigmentosa, RP, (NCT05805007)
Sponsors:
Peking University Third Hospital

IND Enabling

Phase I

Phase II

Phase III

Human immunodeficiency virus, HIV, (NCT03164135)
Sponsors:
Affiliated Hospital to Academy of Military Medical Sciences

IND Enabling

Phase I

Phase II

Phase III

T-Cell Acute Lymphoblastic Lymphoma, T-ALL, (NCT05885464)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Phenylketonuria, PKU, (NCT05222178)
Sponsors:
Homology Medicines

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion Dependent Beta-Thalassaemia, TDT, (NCT05477563)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

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