News

Warning: Spoilers for the movie are contained in this story.  Mention a rare disease milestone — and a new approach to treatment, a new medicine, or even a cure comes to mind. March 15, though, marks a milestone of a different sort for cystic fibrosis (CF) patients: the debut of…

Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), now can be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). The treatment also was approved for any mutation in the CFTR gene that responds to Symdeko based on lab…

Blocking the enzyme cathepsin S can alleviate symptoms and reduce lung damage in cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD), according to multinational teams led by researchers at Queen’s University Belfast, U.K. The findings on CF were reported in the study “Targeting of Cathepsin…

Like scores of runners everywhere, when Joshua Skampo gets home from work, he pulls on his running shoes and heads out into the dusk. But unlike most runners, Joshua runs for his life. He has cystic fibrosis (CF), a strength-sapping disease that makes it hard to breathe. Thick mucus…

ContraFect will receive up to $6.94 million in funding from CARB-X to support the development of their proprietary therapeutic peptides — amurins — against antibiotic-resistant bacterial infections caused by gram-negative ESKAPE pathogens. The company intends to develop these compounds as potential therapies for pulmonary exacerbations of cystic fibrosis and hospital-acquired…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Vertex Pharmaceuticals is planning to request regulatory approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) during the third and fourth quarters of 2019, respectively, for VX-659 or VX-445 triple combination regimens. If approved, these new combo therapies will represent an alternative…

An ongoing Phase 1/2 clinical trial exploring Translate Bio’s investigational therapy MRT5005 will start testing multiple ascending doses (MAD) in patients with cystic fibrosis (CF). This follows an assessment by a protocol review safety committee, which reviewed the study’s protocol and interim safety data. The committee approved…

MaRS Innovation recently announced the launch of two new projects targeting the development of new therapeutics for cystic fibrosis (CF) and respiratory syncytial virus (RSV). These projects are part of LAB150, MaRS Innovation’s joint venture with Evotec AG to support collaboration among scientists, investors, and pharmaceutical companies to…

Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients with cystic fibrosis (CF) caused by rare nonsense genetic mutations, in new trials integrated in the European HIT-CF project. “We are extremely excited to be participating in the HIT-CF research…