Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development

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            Lead Product(s): AAV gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Scottish Enterprise

            Deal Size: $2.5 million Upfront Cash: Undisclosed

            Deal Type: Funding September 02, 2020

            Details:

            This grant has been awarded to AskBio Europe to develop solutions to these industry-wide challenges. AskBio team will further enhance its Pro10™ platform, a simplified and cost-effective AAV manufacturing process that can be scaled and applied throughout the AskBio group.

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            Lead Product(s): MB-107,Busulfan

            Therapeutic Area: Genetic Disease Product Name: MB-107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 02, 2020

            Details:

            U.S. FDA has granted Orphan Drug Designation to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, in newly diagnosed infants under the age of two.

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            Lead Product(s): MB-207

            Therapeutic Area: Genetic Disease Product Name: MB-207

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 31, 2020

            Details:

            U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to MB-207, Mustang’s lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency.

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease Product Name: Trappsol Cyclo

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Novit LP

            Deal Size: $2.8 million Upfront Cash: Undisclosed

            Deal Type: Private Placement August 27, 2020

            Details:

            The company plans to begin a Phase III global pivotal clinical program later this year for treatment of NPC by intravenous administration of Trappsol® Cyclo™, the company’s proprietary formulation of hydroxypropyl beta cyclodextrin.

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            Lead Product(s): rAAV2tYF-GRK1-RPGR

            Therapeutic Area: Genetic Disease Product Name: AGTC 501

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

            Details:

            The studies, which evaluated the safety and efficacy of hRPGRco and another XLRP transgene in a canine model of XLRP, demonstrated stronger expression of hRPGRco than the other transgene at all dose levels evaluated.

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            Lead Product(s): AT132

            Therapeutic Area: Genetic Disease Product Name: AT132

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 20, 2020

            Details:

            The application was based on the Phase 3 study interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year data from the Phase 1/2 Study.

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            Lead Product(s): 4D-125

            Therapeutic Area: Genetic Disease Product Name: 4D-125

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 19, 2020

            Details:

            4D-125 is an AAV gene therapy with an optimized and proprietary vector designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina.

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            Lead Product(s): MB-107,Busulfan

            Therapeutic Area: Genetic Disease Product Name: MB-107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 17, 2020

            Details:

            U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease Designation to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, in newly-diagnosed infants.

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            Lead Product(s): ARO-ENaC

            Therapeutic Area: Genetic Disease Product Name: ARO-ENaC

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 11, 2020

            Details:

            Arrowhead has dosed the first subjects in AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF).

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            Lead Product(s): LB-001

            Therapeutic Area: Genetic Disease Product Name: LB-001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 10, 2020

            Details:

            The SUNRISE trial is a multi-center, open-label, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations.

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