In Vivo Efficacy Studies of Novel Therapeutics for Rare Disease
NCBiotech Grants & Loans
See the full listing of all NCBiotech grants and loans awarded since July 2012.
Organization
Enzerna Biosciences, Inc
Award Date
Award Amount
250,000
Program Name
Small Business Research Loan
Program Category
Business Financing
Principal Investigator
Pete Sazani
Fiscal Year
2023
Region
Research Triangle
Project Description:
Enzerna uses a proprietary gene editing platform, termed Artificial Site-specific RNA Endonucleases (ASRE) to recognize and repair specific nucleotide repeat defects.These defects are the cause of over 40 known rare diseases including Huntington's Disease (HD) and Myotonic Dystrophy (DM; for dystrophia myotonia). SRL funding will support in vivo validation of two ASRE-based lead therapeutic candidates for HD and DM.