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Find Drugs for Genetic Disease in Phase I Clinical Development

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            Lead Product(s): ASP0367

            Therapeutic Area: Genetic Disease Product Name: MA-0211

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 20, 2020

            Details:

            Preclinical data and results from Astellas’ Phase I healthy volunteer study collectively suggest that ASP0367 could improve exercise intolerance and fatigue in PMM patients by increasing the number and enhancing the function of mitochondria in patient’s cells.

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            Lead Product(s): KVD824

            Therapeutic Area: Genetic Disease Product Name: KVD824

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 15, 2020

            Details:

            KVD824 is a twice-daily oral plasma kallikrein inhibitor for prevention of HAE attacks. KalVista initially evaluated KVD824 in a three-part first-in-human study in which 84 subjects received at least one dose of KVD824.

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            Lead Product(s): CB-280

            Therapeutic Area: Genetic Disease Product Name: CB-280

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 07, 2020

            Details:

            The randomized, double-blind, placebo-controlled, multiple ascending dose-escalation study is exploring CB-280 versus placebo in adults with cystic fibrosis and chronic infection with Pseudomonas aeruginosa who are stable on cystic fibrosis medications.

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            Lead Product(s): ARCT-810

            Therapeutic Area: Genetic Disease Product Name: LUNAR-OTC

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 05, 2020

            Details:

            All subjects in cohorts up to 0.3 mg/kg have completed dosing and all study visits. Based on the available preliminary study data, ARCT-810 has been well tolerated at these doses, which are all within the anticipated therapeutic range.

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            Lead Product(s): INV-101

            Therapeutic Area: Genetic Disease Product Name: INV-101

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 29, 2020

            Details:

            The trial will evaluate the safety, tolerability and pharmacokinetics of INV-101. Inversago will enroll healthy participants in Canada, from 18 to 55 years old. Subjects will initially be randomized to receive a single dose of placebo or one of 5 dose levels of INV-101.

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            Lead Product(s): Posiphen

            Therapeutic Area: Genetic Disease Product Name: ANVS401

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 29, 2020

            Details:

            The study compares in both patient populations how nerve cells die by measuring all the steps in the toxic cascade leading to nerve cell death and how ANVS401 might reverse the toxic cascade and recover normal brain function.

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            Lead Product(s): Gavorestat

            Therapeutic Area: Genetic Disease Product Name: AT-007

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 24, 2020

            Details:

            AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG.

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            Lead Product(s): Tolcapone

            Therapeutic Area: Genetic Disease Product Name: CRX-1008

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 15, 2020

            Details:

            The results confirm that CRX-1008 is a potent TTR stabilizer in both plasma and CSF using established ex vivo assays. CRX-1008 normalized TTR concentration in plasma with an overall mean increase in plasma TTR tetramer concentration of 55%.

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            Lead Product(s): REC-994

            Therapeutic Area: Genetic Disease Product Name: REC-994

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Bayer HealthCare

            Deal Size: $239.0 million Upfront Cash: Undisclosed

            Deal Type: Series D Financing September 09, 2020

            Details:

            Proceeds from the financing will support the clinical development of Recursion’s therapeutic pipeline which is the broadest and deepest pipeline to date of any company operating within the space of digitally-powered drug discovery.

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            Lead Product(s): RP-A501

            Therapeutic Area: Genetic Disease Product Name: RP-A501

            Highest Development Status: Phase I 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:

            Rocket Pharmaceuticals has treated the first patient in the higher dose cohort in its open-label, Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon Disease.

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