Diseases

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

ABCA4 Retinopathy

Disease

Acute Lymphoblastic Leukemia, ALL

Disease

Acute Myeloid Leukemia, AML

Disease

Age-related macular degeneration (AMD)

Disease

Alpha-1 Antitrypsin Deficiency

Disease

Alzheimer's disease

Disease

Amyotrophic Lateral Sclerosis (ALS)

Disease

Angelman Syndrome

Disease

Aortic (valve) stenosis

Disease

Atherosclerotic heart disease

Disease

Atopic dermatitis

Disease

Becker Muscular Dystrophy, BMD

Trials

Castration-Resistant Prostate Cancer, CRPC, and Salivary Gland Cancer, SGC, (NCT04249947)
Sponsors:
Poseida Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Metastatic Melanoma, (NCT06783270)
Sponsors:
Inge Marie Svane

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06752876)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Chronic Hepatitis B, HBV, (NCT06671093)
Sponsors:
Tune Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Glycogen Storage Disease Type Ia, GSDIa, (NCT06735755)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis, ATTR, (ChiCTR2400081216)
Sponsors:
AccurEdit Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Alpha 1-Antitrypsin Deficiency, AATD, (NCT06677307)
Sponsors:
Korro Bio

IND Enabling

Phase I

Phase II

Phase III

B-cell Non-Hodgkin Lymphoma, NHL, (NCT06321289)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

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