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            Lead Product(s): rAAVrh8-HexA/HexB

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 07, 2020

            Details:

            Presentations to showcase Axovant's investigational gene therapy programs, AXO-Lenti-PD and AXO-AAV-GM2.

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            Lead Product(s): AAV capsid-gene therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 27, 2020

            Details:

            Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.

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            Lead Product(s): Non-viral gene therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: CureDuchenne Ventures

            Deal Size: $1.0 million Upfront Cash: Undisclosed

            Deal Type: Financing April 22, 2020

            Details:

            Myosana will use this initial funding to advance development of its innovative non-viral gene therapy to deliver full length dystrophin for Duchenne muscular dystrophy (DMD).

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            Lead Product(s): Oligonucleotide linked antibody

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: CureDuchenne Ventures

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing March 31, 2020

            Details:

            The funding will support Dyne’s development of precision therapeutics designed to restore muscle health in people living with Duchenne muscular dystrophy (DMD).

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            Lead Product(s): mRNA-mediated protein replacement therapies

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: MPM Capital

            Deal Size: $80.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing March 26, 2020

            Details:

            ReCode will use the proceeds to continue the preclinical development of its lead programs in primary ciliary dyskinesia and cystic fibrosis.

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            Lead Product(s): Viral vector gene therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: ASC Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 14, 2020

            Details:

            Vigene will provide ASC with access to GMP mfg including viral vectors and plasmid DNA for its Hemophilia A gene therapy clinical program, as well as a manufacturing platform for future programs.