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The funding will help support creation of plasmid (a critical component of gene therapy drug production), as well as the required in-vivo efficacy study in the MPS IIIC mouse model.
Lead Product(s): AAV-TT
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Cure Sanfilippo Foundation
Deal Size: $0.4 million Upfront Cash: Undisclosed
Deal Type: Funding May 15, 2020