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Find Clinical Drug Pipelines for Genetic Disease

A collaboration with SCORR Marketing

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

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            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease

            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): Lonafarnib

            Therapeutic Area: Genetic Disease

            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 March 23, 2020

            Details:

            Lonafarnib is a first-in-class, oral farnesyltransferase inhibitor which has demonstrated extended survival in children and young adults with Progeria.

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 23, 2020

            Details:

            The Committee evaluated all safety data in a blinded fashion from the Phase I trial in Patients With Niemann-Pick Disease Type C and the Effects of Dosing Upon Biomarkers of NPC Disease.

            FORMA Therapeutics

            • Deals

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: NewCo

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Divestment March 19, 2020

            Details:

            Transaction supports FORMA’s business model transition from early research focus to integrated biopharma company with research, development and commercial capacity.

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            Lead Product(s): RP-A501

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 19, 2020

            Details:

            Researchers evaluated the efficacy of a single intravenous (IV) infusion of recombinant adeno-associated serotype nine capsid containing the human LAMP2B transgene in LAMP2-deficient mice.

            University of North Carolina

            • Deals

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: AskBio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement March 18, 2020

            Details:

            Collaboration builds upon preclinical research that demonstrates potential for gene therapy to correct deficiency in UBE3A gene .

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            Lead Product(s): Setmelanotide

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 18, 2020

            Details:

            Rhythm is currently evaluating setmelanotide’s ability to reduce hunger and affect weight loss in an ongoing pivotal Phase 3 trial in patients living with Alström and Bardet-Biedl syndromes.

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            Lead Product(s): Baclofen,Naltrexone Hydrochloride,Sorbitol API

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 18, 2020

            Details:

            All existing data indicate that PXT3003 is a safe and well tolerated drug combination.

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            Lead Product(s): SIG-001

            Therapeutic Area: Genetic Disease

            Highest Development Status: IND Enabling Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Canada Pension Plan Investment Board

            Deal Size: $80.3 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing March 17, 2020

            Details:

            The funding will support the first-in-human clinical trial of Sigilon’s novel encapsulated cell therapy for hemophilia A.

            PharmaCompass