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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): 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).

            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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            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 June 01, 2020

            Details:

            Orphazyme has initiated the submission of its New Drug Application (NDA) for a rolling review by the US Food and Drug Administration (FDA) for arimoclomol, based on the promising results reported from clinical trials. Trial conducted in collaboration with CytRx Corporation.

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            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Palvella Therapeutics

            Deal Size: $3.0 milion Upfront Cash: $2.0 milion

            Deal Type: Agreement May 28, 2020

            Details:

            The milestone payment comes as a result of a development funding and royalties agreement to advance their lead product candidate PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel) for the treatment of pachyonychia congenita.

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            Lead Product(s): OTL-101

            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 May 07, 2020

            Details:

            Orchard Therapeutics to establish OTL-200, OTL-103, OTL-203 and OTL-201 programs as top near-term priorities and reduce investment in OTL-101and OTL-300.

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            Lead Product(s): Livoletide

            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 April 06, 2020

            Details:

            Livoletide did not achieve statistically significant improvement in primary endpoint of change in hyperphagia and food-related behaviors relative to placebo.

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            Lead Product(s): ZYN002

            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 February 26, 2020

            Details:

            The trial is evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 210 children ages three through 17 with full mutation FXS.

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