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            University of Massachusetts Medical School

            • Deals

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            Lead Product(s): AAV-based gene

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Foundation to Fight H-ABC

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement October 13, 2020

            Details:

            A team of researchers from University of Massachusetts Medical School and Yale School of Medicine will combine extensive expertise in the fields of Adeno-associated virus, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Genentech

            Deal Size: $375.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration September 21, 2020

            Details:

            Under the terms of the agreement, Scenic will utilize its Cell-Seq platform and its data warehouse of genetic modifiers to identify drug targets in multiple therapeutic areas.

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            Lead Product(s): LNP based mRNA Therapeutics

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: $455.0 million Upfront Cash: $75.0 million

            Deal Type: Collaboration September 16, 2020

            Details:

            The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator protein to be produced.

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            Lead Product(s): Induced pluripotent stem cell

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Centogene

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Collaboration August 20, 2020

            Details:

            The collaboration brings together Evotec’s leading induced pluripotent stem cell platform and broad drug discovery and development capabilities with CENTOGENE’s global proprietary rare disease platform, including iPSC lines, to address the needs of this orphan drug indication.

            Gero

            • Deals

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Bulba Ventures

            Deal Size: $2.2 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing August 05, 2020

            Details:

            The financing brought the total capital raised by the firm to more than $7.5M which will be invested in Gero's proprietary AI/ML platforms for new drugs development for aging and other complicated disorders.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: MyoKardia

            Deal Size: $465.0 million Upfront Cash: $12.5 million

            Deal Type: Collaboration July 21, 2020

            Details:

            Under the agreement, MyoKardia will access Fulcrum’s unique, proprietary target discovery engine to identify therapeutics that control the expression of genes that are known to be underlying drivers of genetic cardiomyopathies.

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            Lead Product(s): Bispecific antibodies

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration February 13, 2020

            Details:

            The milestone was achieved upon the successful completion of the discovery phase and designation by Takeda of undisclosed bispecific antibodies with approval to advance to IND-enabling studies.