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
Chronic Hepatitis B, HBV, (NCT06680232)
Sponsors:
Precision BioSciences, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Transthyretin Amyloidosis with Polyneuropathy, ATTRv-PN, (NCT06672237)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Duchenne Muscular Dystrophy, DMD, (NCT06594094)
Sponsors:
HuidaGene Therapeutics Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT06598371)
Sponsors:
M.D. Anderson Cancer Center
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Hereditary Angioedema, HAE, (NCT06634420)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Neuromyelitis Optica Spectrum Disease, NMOSD, (NCT06633042)
Sponsors:
Bioray Laboratories
Indicator
IND Enabling
Phase I
Phase II
Phase III
Neovascular Age-Related Macular Degeneration, nAMD, (NCT06623279)
Sponsors:
HuidaGene Therapeutics Co., Ltd.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Alpha-1 Antitrypsin Deficiency, AATD, (NCT06622668)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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