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

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            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

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            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $650.0 million

            Deal Type: Agreement July 20, 2020

            Details:

            Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA).

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            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details:

            Orphazyme has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).

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            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $100.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering July 17, 2020

            Details:

            The company's lead candidate, FLT180a, an investigational gene therapy medicinal product candidate for the treatment of hemophilia B, is currently in a Phase 1/2 trial and has dosed 10 patients as of June 15, 2020.

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            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Novo Holdings

            Deal Size: $120.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing June 30, 2020

            Details:

            Freeline expects to use the proceeds from the financing to bring its lead program in Haemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical program for Fabry Disease and further progress its pipeline programs for Gaucher Disease and Haemophilia A.

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 30, 2020

            Details:

            Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS).

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            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

            Details:

            Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients.

            The Lundquist Institute

            • Development Update

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            Lead Product(s): Anakinra

            Therapeutic Area: Genetic Disease Product Name: Kineret

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 24, 2020

            Details:

            The trial, which began in May, involves 20 children with Sanfilippo syndrome, who will be given anakinra, which blocks the activity of interleukin-1 (IL-1), a protein in the body that causes joint damage, thereby reducing inflammation.

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            Lead Product(s): Sepofarsen

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2020

            Details:

            The presentation will highlight results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 05, 2020

            Details:

            Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A).

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