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Find Drugs for Genetic Disease in Phase I Clinical Development

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            Lead Product(s): PF1801

            Therapeutic Area: Genetic Disease Product Name: PF1801

            Highest Development Status: Phase I Product Type: Undisclosed

            Partner/Sponsor/Collaborator: PhaseBio Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 28, 2021

            Details:

            Immunoforge is developing agents for rare muscle related diseases, such as Duchenne muscular dystrophy, polymyositis, and inclusion body myositis, based on its patent with PF1801 from PhaseBio, which has completed phase 2 clinical trials.

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            Lead Product(s): Autologous CD34+ cells,TNS9.3.55

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Memorial Sloan Kettering Cancer Center

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 11, 2021

            Details:

            The EHA abstract, submitted by Simona Raso, reports that 2 out of 3 Thalassemia patients treated with EGT’s autologous CD34+ cells have sustained dramatic reduction in blood transfusions after 8 and 5 years, respectively.

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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 January 07, 2021

            Details:

            Intellia Therapeutics is continuing advancement of global Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR).

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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 January 07, 2021

            Details:

            INV-101 is a first-in-class, small molecule CB1 inverse agonist / antagonist being developed by Inversago for the treatment of PWS and non-alcoholic steatohepatitis (NASH).

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            Lead Product(s): ER-004

            Therapeutic Area: Genetic Disease Product Name: EDI200

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Pierre Fabre

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership December 21, 2020

            Details:

            AGC Biologics will manufacture GMP material for the next stage of clinical trial. ER-004 is an intra-amniotic drug that will pioneer the treatment of a rare and debilitating genetic disorder.

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            Lead Product(s): ER-004

            Therapeutic Area: Genetic Disease Product Name: EDI200

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Pierre Fabre

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration December 14, 2020

            Details:

            The Pierre Fabre group will be granted exclusive worldwide rights for the development, manufacturing and commercialization of ER-004. EspeRare and the Pierre Fabre Group will pool their respective expertise together in order to co-develop ER-004.