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Find Drugs for Genetic Disease in Phase II Clinical Development

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            Lead Product(s): IMR-687

            Therapeutic Area: Genetic Disease Product Name: IMR-687

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 30, 2020

            Details:

            Imara recently initiated a Phase 2b clinical trial of IMR-687 in adult patients with beta-thalassemia and expects to dose the first patient in the near-term.

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            Lead Product(s): HST5040

            Therapeutic Area: Genetic Disease Product Name: HST5040

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 28, 2020

            Details:

            HemoShear received Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration for HST5040, a once-daily oral small molecule drug being developed to treat methylmalonic acidemia (MMA) and propionic acidemia (PA).

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            Lead Product(s): Allogeneic human retinal progenitor cells

            Therapeutic Area: Genetic Disease Product Name: jCell

            Highest Development Status: 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 July 27, 2020

            Details:

            Phase 2b results show that treatment with jCell was well-tolerated and demonstrated encouraging evidence of potential benefit in patients as an investigational treatment for retinitis pigmentosa agnostic to genetic subtype.

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            Lead Product(s): RZ358

            Therapeutic Area: Genetic Disease Product Name: RZ358

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 27, 2020

            Details:

            In the Company’s quarterly report filed on Form 10-Q with the US Securities and Exchange Commission in May 2020 (“10-Q”), the Company reported that, as a result of the Pandemic, it paused the RIZE study after enrollment of the first patient.

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            Lead Product(s): AAVrh74.MHCK7.micro-dystrophin

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: 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 July 24, 2020

            Details:

            Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 in Study 101 were recently published in JAMA Neurology, and Study 102, a randomized, double-blind, placebo-controlled study of SRP-9001, is ongoing.

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            Lead Product(s): Trehalose API

            Therapeutic Area: Genetic Disease Product Name: SLS-005

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2020

            Details:

            Seelos Therapeutics has received a positive opinion on European Orphan Drug Designation for SLS-005 in Sanfilippo syndrome from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

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            Lead Product(s): OTL-103

            Therapeutic Area: Genetic Disease Product Name: OTL-103

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

            Partner/Sponsor/Collaborator: Orchard Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement July 15, 2020

            Details:

            Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.

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            Lead Product(s): Infigratinib

            Therapeutic Area: Genetic Disease Product Name: BBP-831

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 15, 2020

            Details:

            First child with achondroplasia has been dosed with the investigational medicine infigratinib, an orally available small molecule, that targets the overactivity of fibroblast growth factor receptor 3 (FGFR3) in the PROPEL 2 Phase 2 clinical trial.

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            Lead Product(s): Marzeptacog alfa

            Therapeutic Area: Genetic Disease Product Name: MarzAA

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 13, 2020

            Details:

            Subcutaneous marzeptacog alfa (activated) (MarzAA) rapidly achieves and maintains therapeutic levels. Data confirm Phase 3 study design to treat acute bleeding events in hemophilia.

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            Lead Product(s): Allogeneic human retinal progenitor cells

            Therapeutic Area: Genetic Disease Product Name: jCell

            Highest Development Status: 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 July 07, 2020

            Details:

            Results from the Ph 2b trial of jCell in retinitis pigmentosa will be presented at the American Society of Retina Specialists. jCell is an investigational treatment for retinitis pigmentosa which has received FDA Regenerative Medicine Advanced Therapy designation.

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