Clinical Trials - Rare Diseases

View a full list of all ongoing trials here.

 

 

Beta - thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
Haemophilia B Factor IX, (2017-004805-42)
Sponsors:
Sangamo Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Haemophilia B, (NCT02695160)
Sponsors:
Sangamo Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Bioverativ Therapeutics Inc. (a Sanofi company)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals
IndicatorIndicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Graphite Bio Inc
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04853576)
Sponsors:
Editas Medicine, Inc. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Transthyretin amyloidosis, ATTR, (NCT04601051)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
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