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

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

            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 December 07, 2020

            Details:

            In a cohort of nine patients, six of seven patients (86%) who received FT-4202 achieved a hemoglobin increase of greater than 1 g/dL from baseline with once-daily dosing at 300 mg for 14 days.

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            Lead Product(s): PTC518

            Therapeutic Area: Genetic Disease Product Name: PTC518

            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 November 17, 2020

            Details:

            PTC518 is an orally bioavailable molecule with broad central nervous system and systemic distribution and has been designed to target Huntingtin protein expression with high selectivity and specificity.

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            Lead Product(s): RP-L401

            Therapeutic Area: Genetic Disease Product Name: RP-L401

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

            Partner/Sponsor/Collaborator: California Institute for Regenerative Medicine

            Deal Size: $3.7 million Upfront Cash: Undisclosed

            Deal Type: Funding November 13, 2020

            Details:

            Clinical Grant of $3.7 M to Fund US Phase 1 Study of RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis. This will be Rocket’s second CIRM grant after receiving one in 2019 for the development of the company’s gene therapy for Leukocyte Adhesion Deficiency-I.

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            Lead Product(s): EDG-5506

            Therapeutic Area: Genetic Disease Product Name: EDG-5506

            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 November 11, 2020

            Details:

            The Phase 1 trial is designed to assess the safety, tolerability and pharmacokinetics (PK) of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

            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 November 10, 2020

            Details:

            European Commission granted Orphan Drug designation to Forma’s FT-4202 for the treatment of sickle cell disease (SCD), based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).

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            Lead Product(s): NTLA-2001

            Therapeutic Area: Genetic Disease Product Name: NTLA-2001

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

            Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 09, 2020

            Details:

            Intellia’s global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

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            Lead Product(s): FTX-6058

            Therapeutic Area: Genetic Disease Product Name: FTX-6058

            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 November 04, 2020

            Details:

            Multiple posters on the company’s IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and β-thalassemia will be presented at the virtual 62nd ASH Annual Meeting and Exposition.

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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: Cystic Fibrosis Foundation

            Deal Size: $2.4 million Upfront Cash: Undisclosed

            Deal Type: Funding November 02, 2020

            Details:

            The award will support clinical development of CB-280, Calithera’s investigational first-in-class arginase inhibitor, which promotes higher tissue levels of nitric oxide (NO) to reduce the risk of infections in people with cystic fibrosis (CF).

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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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