Development of First Curative Therapy for Myotonic Dystrophy
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
150,000
Program Name
Small Business Research Loan
Program Category
Business Financing
Principal Investigator
Joseph Ruiz
Fiscal Year
2019
Region
Research Triangle
Project Description:
Enzerna Biosciences is developing a patented adeno-associated virus (AAV) gene therapy called artificial site-specific RNA endonuclease (ASRE). ASRE is a protein-guided RNA-editing enzyme that can be designed to specifically bind RNA repeats and destroy toxic RNA molecules that cause nucleotide expansion disorders, such as myotonic dystrophy (DM1). Enzerna will use the SRL to fund IND-enabling activities to advance novel RNA-ablating gene therapies for DM1.