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Find Novel Drugs for Genetic Disease under Development

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            Lead Product(s): OTL-203

            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 15, 2020

            Details:

            MPS-I clinical trial interim results demonstrated- all patients, treated with OTL-203, achieving hematological recovery in less than 45 days – a primary outcome measure – as well as supraphysiological levels of IDUA enzyme in the blood.

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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): Gene Therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Sofinnova Partners

            Deal Size: $17.3 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing April 29, 2020

            Details:

            The funds will be used to advance Genespire’s leading-edge platform technologies towards the development of novel gene therapies in primary immunodeficiencies and metabolic genetic diseases.

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            Lead Product(s): OTL-203

            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 April 29, 2020

            Details:

            Accepted abstracts include data on investigational gene therapies for neurometabolic conditions, including updated interim data from proof-of-concept trial in OTL-203 for Mucopolysaccharidosis Type I.

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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).

            Genespire

            • Deals

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            Lead Product(s): Gene Therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Sofinnova Partners

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing April 20, 2020

            Details:

            This investment brings the total financing by Sofinnova Partners to €25 million over the past six months.

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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.

            University of North Carolina

            • Deals

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            Lead Product(s): Gene Therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Gene therapy

            Partner/Sponsor/Collaborator: AskBio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement March 18, 2020

            Details:

            Collaboration builds upon preclinical research that demonstrates potential for gene therapy to correct deficiency in UBE3A gene.

            PharmaCompass