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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

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            Virtual BoothSanofi Active Ingredient Solutions cover both APIs sales & CDMO.

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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): Sepofarsen

            Therapeutic Area: Genetic Disease Product Name: QR-110

            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 January 07, 2021

            Details:

            Illuminate is a Phase 2/3 trial, which randomized 36 patients aged eight years or older to receive either sepofarsen at the target registration dose, a low dose, or sham treatment.

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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 December 27, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months.

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            Lead Product(s): REN001

            Therapeutic Area: Genetic Disease Product Name: REN001

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

            Partner/Sponsor/Collaborator: Novo Ventures

            Deal Size: $95.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing December 09, 2020

            Details:

            Funds will support developments of REN001 which is an oral, once-daily investigational drug known to control several genes involved in mitochondrial activity.

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

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

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering December 08, 2020

            Details:

            Funds to be used in the development of its lead program FT-4202 in sickle cell disease and completion of Phase 1 clinical trial, advancement of FT-7051 in metastatic castration-resistant prostate cancer, pre-approval activities for FT-2102 in AML.

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

            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 December 07, 2020

            Details:

            FT-4202 is a novel, investigational, selective red blood cell pyruvate kinase-R activator in development as a potential disease-modifying therapy for SCD. 6 of 7 (86%) patients on 300 mg of FT-4202 for 14 days achieved a hemoglobin increase > 1 g/dL from baseline.

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            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

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

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Licensing Agreement November 24, 2020

            Details:

            Under the terms of the agreement, Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the United States under a 50/50 profit-sharing structure.

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            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            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 November 09, 2020

            Details:

            Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world.

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            Lead Product(s): RTA 402

            Therapeutic Area: Genetic Disease Product Name: RTA 402

            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 November 09, 2020

            Details:

            Bardoxolone achieved the year 2 primary and key secondary endpoints with statistically significant improvements in EGFR as compared to placebo at week 100 and week 104 .

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

            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 November 04, 2020

            Details:

            The oral presentation will feature clinical data from the multiple ascending dose cohort of a randomized, multi-center, placebo-controlled Phase 1 trial of FT-4202 in people living with sickle cell disease (SCD).

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