Rare Disease

| CRISPR Medicine | This is an overview of articles and trials related to Rare Diseases.

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Rare Disease

| CRISPR Medicine | This is an overview of articles and trials related to Rare Diseases.

Sign-up to weekly Newsletter here.

Hereditary Angioedema, HAE (NCT05120830)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated
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
Beta-Thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
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, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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