Lead Product(s): CRISPR/Cas9 genome
Therapeutic Area: Musculoskeletal Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Sarepta Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration January 13, 2020
As per collaboration agreement, the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein.