SCGE Regulatory Documents
The Somatic Cell Genome Editing (SCGE) Consortium is an NIH Common Fund program created to accelerate translational research in genome editing. A primary goal is to disseminate documents generated through regulatory interactions between the U.S. Food and Drug Administration (FDA) and consortium projects to help the rare disease communities and the providers and researchers that serve them.
| Disease Area | Program | Lead Optimization |
INTERACT Meeting |
Pre-IND Meeting |
IND Enabling Studies |
Clinical Trial Initiated |
|---|---|---|---|---|---|---|
Inborn
Errors of Metabolism |
Phenylketonuria (PKU) |
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| Carbamoyl Phosphate Synthetase I (CPS1) Deficiency |
Coming Soon..
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| Urea Cycle Disorders (UCDs) |
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| Spinal Muscular Atrophy (SMA) |
Coming Soon..
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| Angelman Syndrome |
Coming Soon..
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| Prion Disease |
Coming Soon..
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| Leber Congenital Amaurosis (KCNJ13) |
Coming Soon..
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