SCGE Shared 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 |
Publications | Documents |
|---|---|---|---|---|---|---|---|---|
| Phenylketonuria (PKU) |
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PKU Program Publications
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PKU Program Documents |
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| Carbamoyl Phosphate Synthetase I (CPS1) Deficiency |
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CPS1 Program Publications |
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| Urea Cycle Disorders (UCDs) |
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UCD Program Publications |
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UCD Program Documents |
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| Spinal Muscular Atrophy (SMA) |
Coming Soon..
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| Angelman Syndrome |
Coming Soon..
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| Prion Disease | ||||||||
Prion Disease Program Documents |
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| Leber Congenital Amaurosis (KCNJ13) |
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| Best Vitelliform Macular Dystrophy (BVMD, Best's Disease) |
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