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

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            Lead Product(s): rAAV2tYF-GRK1-RPGR

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 20, 2020

            Details:

            Administration of AGTC’s proprietary adeno-associated virus (AAV) vectors expressing variants of the RPGR gene corrected early markers of disease and provided evidence of a rescue effect in an animal model of XLRP, with a favorable safety profile.

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            Lead Product(s): AAV5-hFIX

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 18, 2020

            Details:

            A Phase 1/2 clinical trial of AMT-060 in 10 men with moderate to severe hemophilia B showed that the gene therapy was safe and effective, able to durably increase FIX levels for at least 3.5 years and reduce bleeds to near zero with the higher dose.

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 18, 2020

            Details:

            EMA’s Committee for Orphan Medicinal Products concluded that the currently proposed clinical development is sufficient to demonstrate significant benefit of Trappsol® Cyclo™ for Niemann-Pick Disease Type C1.

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            Lead Product(s): KB105

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 14, 2020

            Details:

            Clearly detectable TGM-1 expression in all treated areas following initial and repeat administration. KB105-expressed TGM1 was correctly localized in the epidermis, co-localizing with loricrin, and was functionally active.

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            Lead Product(s): AVR-RD-01

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 13, 2020

            Details:

            The new interim data continue to support potential first-line use of AVR-RD-01 for Fabry disease. Treatment with AVR-RD-01 resulted in 3% reduction in toxic metabolite plasma lyso-Gb3 one month.

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            Lead Product(s): DTX301

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 13, 2020

            Details:

            All three patients in Cohort 3 now confirmed responders. All three complete responders in the study remain clinically and metabolically stable after longer-term follow-up.

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            Lead Product(s): RP-L201

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 12, 2020

            Details:

            Two oral presentations highlight updates from the company’s Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 “Process A” for the treatment of FA.

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            Lead Product(s): SGT-001

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II 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:

            Solid Biosciences has received written communication from the U.S. FDA regarding the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. The program remains on clinical hold.

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            Lead Product(s): AVR-RD-04

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 05, 2020

            Details:

            Updated data will be presented on first patient dosed in Phase 1/2 clinical trial for cystinosis and first patient treated using plato™ gene therapy platform in Phase 2 Fabry disease clinical trial .

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            Lead Product(s): GTX-102

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 04, 2020

            Details:

            GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of Angelman syndrome.

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