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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.
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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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