Clinical Trials - Rare Diseases

View a full list of all ongoing trials here.

Beta - thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Haemophilia B Factor IX, (2017-004805-42)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Haemophilia B, (NCT02695160)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Herpes Simplex Virus Refractory Keratitis, (NCT04560790)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis type II, MPS II, (2018-000192-33)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Bioverativ Therapeutics Inc. (a Sanofi company)

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04774536)
Sponsors:
Principal Investigator: Mark Walters, MD UCSF Benioff Children's Hospital Oakland

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Graphite Bio Inc

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04853576)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassemia, TDT, (NCT03432364)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Transthyretin amyloidosis, ATTR, (NCT04601051)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

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