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

Transfusion-dependent Beta-Thalassemia, TDT, (NCT06291961)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT06328764)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT06565026)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Transfusion-dependent Beta-Thalassemia, TDT, (NCT05015920)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Primary Open Angle Glaucoma, POAG, (NCT06465537)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Herpes Simplex Virus Type I Stromal Keratitis, (NCT06474442)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Herpes Simplex Virus Type I Stromal Keratitis, (NCT06474416)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06373991)
Sponsors:
EdiGene Inc.

IND Enabling

Phase I

Phase II

Phase III

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