Cystic Fibrosis Weekly Update

Contents:

Zikani Raises $7.5M to Support Work into Therapies for Nonsense Mutations

Apr 22, 2020 07:00 am | Joana Carvalho, PhD

Zikani Therapeutics has raised $7.5 million in Series A funding to advance work, using its TURBO-ZM technology platform, into potential therapies for rare disorders caused by nonsense mutations, including cystic fibrosis (CF). TURBO-ZM is a proprietary platform that enables Zikani to rapidly produce ribosome modulating agents (RMAs). These compounds work by targeting ribosomes — small structures […]
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I Wish I Was Sheltered in Place with Anyone but Me

Apr 21, 2020 03:00 pm | Bailey Vincent

pre-existing condition, perspective, slowing down

I am gravely inflexible. This might seem shocking to those who know me, since I write openly about being a professional dancer. A lot of my career depends on how willingly I stretch, and how openly I hide imperfections in plain sight. I am stuck at home these days (like everyone else sheltering in place), […]
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New Capsules Being Readied for Trial of Digestive Enzyme in CF Patients with EPI

Apr 21, 2020 07:00 am | Ines Martins, PhD

Delpharm has agreed to manufacture AzurRx BioPharma‘s investigational therapy MS1819 — a potential treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF) in clinical testing— using enteric capsules, an approach that increases the delivery of lipases (digestive enzymes). Delpharm produced a first batch of MS1819 with enteric capsules, and is now working to make […]
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