Cystic Fibrosis – PharmaLive

Flagship Pioneering is All Hands on Deck for Cystic Fibrosis

Business, Collaborations, Cystic Fibrosis, Partnerships, R&D, Venture Capital

Prominent life sciences venture capital firm Flagship Pioneering announced a strategic partnership with the Cystic Fibrosis Foundation on November 3.

November 3, 2021/by BioSpace

JHL Co-founders Convicted of Trade Secret Theft

Blockbusters, Business, Cancer, Convictions, Cystic Fibrosis, Indictments, Money Laundering, Roche, San Francisco, Trade Secrets, Wire Fraud

Racho Jordanov and Rose Lin, the septuagenarian co-founders of Taiwanese biotech company JHL Biotech, were convicted Aug. 26 of conspiring to steal trade secrets related to four Genentech (Roche) blockbuster cancer and cystic-fibrosis drugs. The pair were also found guilty of wire fraud.

August 30, 2021/by Andrew Humphreys

Vertex cystic fibrosis treatment gets U.S. nod for use in children aged 6-11

Approvals, Business, Children, Cystic Fibrosis, Drug Cocktails, Drug Pricing, Expanded Use, FDA, FDA/Regulatory, Shares, Therapeutics

The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals Inc.’s Trikafta to treat cystic fibrosis in children aged 6 to 11 years old, making the cocktail therapy the first approved treatment for CF in that age group.

June 9, 2021/by Andrew Humphreys

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

Vertex Looks to Expand Asset Portfolio

Beta Thalassemia, Blockbusters, Business, Clinical Trials, Collaborations, CRISPR, Cystic Fibrosis, J.P. Morgan Healthcare Conference, M&A, Mutations, Product Pipeline, R&D, Sickle Cell Disease, Therapeutics

At the 2021 virtual JP Morgan Healthcare Conference, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”

January 12, 2021/by Andrew Humphreys

Skyhawk and Vertex Team Up on RNA Splicing-Based Therapies

Alpha-1 Antitrypsin Deficiency (AATD), Business, Clinical Trials, Collaborations, Cystic Fibrosis, Deals, Licensing, Neurodegenerative Diseases, Oncology, Pain, R&D, RNA Splicing, Small Molecules

Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing.

December 22, 2020/by BioSpace

FDA Approves Trikafta, Symdeko and Kalydeco for CF Patients With Certain Rare Mutations

Approvals, Blockbusters, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), Expanded Approval, FDA, Genetic Diseases, in vitro data, New Indications, Rare Mutations, Therapeutics

The U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for use in people with cystic fibrosis with certain rare mutations.

December 21, 2020/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

Corbus Pharma’s Lenabasum Fails Again, This Time in Cystic Fibrosis

Business, Clinical Trials, Cystic Fibrosis, Fibrosis, Inflammation, R&D, Shares, Small Molecules, Topline Data

Topline results from a 28-week Phase IIb trial show that lenabasum, a novel oral small molecule developed by Massachusetts-based Corbus Pharmaceuticals, did not reduce the rate of new pulmonary exacerbations compared with placebo in patients with cystic fibrosis (CF).

October 6, 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