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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.
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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.
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