CFTR protein Archives

SpliSense Grabs $28.5 Million for Restorative Cystic Fibrosis Treatment

CFTR protein, Cystic Fibrosis, Financing, Genetic pulmonary diseases, Liver Disease, Lungs, Mutations, Series B

Jerusalem-based SpliSense, which focuses on cystic fibrosis and other genetic pulmonary diseases, closed on a $28.5 million Series B financing round. Participating in the round were OrbiMed, Israel Biotech Fund, Integra Holdings and the Cystic Fibrosis Foundation.

May 13, 2021/by Andrew Humphreys

Roche Carves Way into CF Space with Portfolio Acquisition

Acquisitions, Business, CFTR Gene, CFTR protein, Cystic Fibrosis, Lung Damage, Lung Infections

Roche acquired a portfolio of experimental cystic fibrosis therapies, including a candidate that has the potential to treat all patients with the disease.

October 7, 2020/by Andrew Humphreys

Moderna and Vertex Establish New Collaboration

Business, CFTR protein, Collaborations, Cystic Fibrosis, Gene Editing, Investments, Lipid Nanoparticles (LNPs), Lung Cells, Messenger RNA (mRNA) Therapeutics

Moderna Inc. and Vertex Pharmaceuticals Incorporated announced a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF).

September 16, 2020/by Andrew Humphreys

FDA Approves Symdeko to Treat Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in CFTR Gene

CFTR protein, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), FDA, FDA/Regulatory, Genetic Diseases, New Indications

The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.

June 21, 2019/by Business Wire