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

Age-related macular degeneration (AMD)

Disease

Alzheimer's disease

Disease

Angelman Syndrome

Disease

Aortic (valve) stenosis

Disease

Atherosclerotic heart disease

Disease

Atopic dermatitis

Disease

Beta Thalassemia

Disease

Bordetella pertussis

Disease

Chronic Granulomatous Disease

Disease

Chronic Hepatitis B
Trials
Systemic Lupus Erythematosus, SLE, (NCT06255028)
Sponsors:
Century Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Chronic Granulomatous Disease, CGD, (NCT06325709)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06287099)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Transfusion-dependent Beta-thalassemia, TDT, (NCT06298630)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Autoimmune Diseases, (NCT05859997)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Systemic Lupus Erythematosus, SLE, (NCT06308978)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06300723)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Ornithine Transcarbamylase Deficiency, OTC, (NCT06255782)
Sponsors:
iECURE, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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