Market Place
Company
Pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme. 
X
Details:
These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 years and older.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Small molecule
Partner/Sponsor/Collaborator: Global Blood Therapeutics
Deal Size: $353.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement March 16, 2021
DDF Summit 2022 US
Not Confirmed
Contact SupplierDetails:
Through this collaboration, BridgeBio will work with Baylor College of Medicine to identify and translate promising innovations into potential therapies for patients with genetically driven diseases.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Undisclosed
Partner/Sponsor/Collaborator: Baylor College of Medicine
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration July 21, 2022
Details:
Under the terms of the Agreement, Precision will develop an ARCUS nuclease and conduct in vitro characterization, with Novartis then assuming responsibility for all subsequent research, development, manufacturing and commercialization activities of PBGENE-HbE.
Lead Product(s): PBGENE-HbE
Therapeutic Area: Genetic Disease Product Name: PBGENE-HbE
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation
Deal Size: $1,475.0 million Upfront Cash: $75.0 million
Deal Type: Collaboration June 21, 2022
Details:
These precisely reprogrammed cell types are generated with the company’s precision cellular reprogramming technology opti-oxTM that enables unlimited batches of any human cell to be manufactured consistently at scale.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 June 16, 2022
Details:
LUCA Science will use its proprietary platform technology to manufacture, store and deliver innovative mitochondrial drugs to research and development of therapeutics for unmet medical needs in a wide range of diseases for which there have been no effective treatments.
Lead Product(s): Mitochondrial-based Therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Kyowa Kirin
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration May 18, 2022
Details:
First neurodegenerative disease model launched from bit.bio's new ioDisease Model portfolio. Accurately replicates Huntington's disease genotype in human cells, setting a new standard in cell models for drug discovery.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 May 09, 2022
Details:
Two poster to be presentated highlighting three of the Company’s gene therapy programs using directed evolution to potentially improve treatment options for Hemophilia A and lysosomal storage diseases.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 May 02, 2022
Details:
Proceeds from the financing will be used to advance OMass’s portfolio towards clinical trials. This includes the development of an insurmountable antagonist of the MC2 receptor for congenital adrenal hyperplasia.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Small molecule
Partner/Sponsor/Collaborator: GV
Deal Size: $100.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing April 28, 2022
Details:
Both companies, aim to identify small molecule drugs to treat Niemann Pick Type C (NP-C), a rare lipid storage disorder that affects lipid metabolism, a second undisclosed program to treat a severe heritable metabolic syndrome, and Barth syndrome program.
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Small molecule
Partner/Sponsor/Collaborator: Barth Syndrome Foundation
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership March 30, 2022
Details:
This collaboration will build on Tessera’s innovations in genome engineering technologies and apply Serotiny’s tMDP mining and design expertise to further optimize Tessera’s programmable Gene Writer proteins, capable of making diverse and directed alterations to the genome.
Lead Product(s): Genetic Medicine
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Tessera Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration February 28, 2022
