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

Angelman Syndrome

Disease

Aortic (valve) stenosis

Disease

Atherosclerotic heart disease

Disease

Atopic dermatitis

Disease

Becker Muscular Dystrophy, BMD

Disease

Beta Thalassemia

Trials

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

Relapsed/Refractory Hematologic Malignancies (NCT06492304)
Sponsors:
CRISPR Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Haemophilia B, (NCT06611436)
Sponsors:
Be Biopharma

IND Enabling

Phase I

Phase II

Phase III

Lupus Nephritis, (NCT06681337)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Chronic Hepatitis B, HBV, (NCT06680232)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis with Polyneuropathy, ATTRv-PN, (NCT06672237)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

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