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

Advanced Primary or Secondary Peritoneal Tumors, (NCT07004647)
Sponsors:
Ruijin Hospital

IND Enabling

Phase I

Phase II

Phase III

Recurrent or Progressive High-grade Glioma, (NCT06737146)
Sponsors:
Suzhou Maximum Bio-tech Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Advanced Peritoneal Malignancies or Abdominal Metastatic Solid Tumors, (NCT06912152)
Sponsors:
Zhejiang University

IND Enabling

Phase I

Phase II

Phase III

Transfusion-dependent Beta- Thalassemia, (NCT06479616)
Sponsors:
Children's Hospital of Fudan University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or refractory B-cell non-Hodgkin's lymphoma, (NCT06838832)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Retinitis Pigmentosa, (NCT06952842)
Sponsors:
Chigenovo Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Snijders Blok-Campeau syndrome, (NCT06860672)
Sponsors:
Yongguo Yu

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, (NCT07000318)
Sponsors:
Children's Hospital of Fudan University

IND Enabling

Phase I

Phase II

Phase III

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