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.

Duchenne Muscular Dystrophy, DMD, (NCT05514249)
Sponsors:
Cure Rare Disease, Inc
Indicator
IND Enabling
Phase I
Phase II
Phase III
Retinitis Pigmentosa, RP, (NCT05805007)
Sponsors:
Peking University Third Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle cell disease, SCD, (NCT05951205)
Sponsors:
Vertex Pharmaceuticals Incorporated
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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
IndicatorIndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04774536)
Sponsors:
Mark Walters, MD 
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
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