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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Bill & Melinda Gates Foundation

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration February 17, 2021

            Details:

            As part of the agreement, the Bill & Melinda Gates Foundation will provide Novartis funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD).

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            Lead Product(s): AAV9-based gene therapy

            Therapeutic Area: Genetic Disease Product Name: TSHA-105

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 19, 2021

            Details:

            TSHA-105 joins a portfolio of rare pediatric disease and orphan drug designations obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1, TSHA-102 for Rett syndrome, TSHA-104 for Leigh syndrome and TSHA-103 for SLC6A1-epilepsy.

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            Lead Product(s): TSHA-112

            Therapeutic Area: Genetic Disease Product Name: TSHA-112

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

            Partner/Sponsor/Collaborator: Taysha Gene Therapies

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Collaboration January 13, 2021

            Details:

            The Partnership has produced over 18 novel product candidates, including TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1, which are currently in clinical development.

            University of Massachusetts Medical School

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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.