Proteostasis Therapeutics (PTI) has entered into a definitive merger agreement with Yumanity Therapeutics to leverage their common expertise about protein misfolding to advance the development of disease-modifying therapies for several disorders. The merger should be complete before the end of the year. The combined company will operate under the…
The Cystic Fibrosis Foundation (CFF) has awarded nearly $15 million to 33 organizations that are researching how to improve outcomes for cystic fibrosis (CF) patients who are battling infections. The awards fall under the CFF’s Infection Research Initiative, a large-scale effort to provide $100 million in funding…
Poorer oxygen uptake and transport may underlie skeletal muscle differences that affect exercise tolerance in children and adolescents with cystic fibrosis (CF) relative to healthy peers, a recent study suggests. The study, “Tissue oxygenation in peripheral muscles and functional capacity in cystic fibrosis: A cross‐sectional study,”…
The European Commission (EC) approved Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene. The decision follows a positive opinion,…
Note: This story was updated Aug. 25, 2020, to clarify that, according to NuvoAir, the regulatory entity in question is the European Medical Device Directive. The NuvoAir Home platform, NuvoAir’s respiratory support system for patients with lung diseases such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF),…
The U.S. Food and Drug Administration (FDA) has granted the designation of orphan drug to KB407, Krystal Biotech’s potential inhaled gene therapy for the treatment of cystic fibrosis (CF). “We are pleased to receive Orphan Drug Designation for KB407 to treat cystic fibrosis as…
The nonprofit Claire’s Place Foundation has launched a pilot program to help 15 adults with cystic fibrosis (CF) or caregivers of patients acquire flexible employment, enabling them to continue working while dealing with the disease’s daily needs. The Work Proudly Program aids people in choosing careers and gaining the…
AzurRx BioPharma has enrolled the first three patients in its Phase 2b trial investigating MS1819 as a treatment for exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients. The trial, dubbed OPTION 2 (NCT04375878), aims to enroll 30 CF patients ages 18 and older. Top-line…
MS1819, AzurRx BioPharma’s investigational treatment for exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF), safely increased fat absorption in the first five patients receiving the therapy alongside pancreatic enzyme replacement therapy (PERT) in a Phase 2 clinical trial. The interim analysis also showed the…
A Phase 1/2 clinical trial assessing Arrowhead Pharmaceuticals‘ investigational inhaled therapy, ARO-ENaC, for the treatment of cystic fibrosis (CF), has dosed its first participants, the company announced. The AROENaC1001 trial (NCT04375514) is designed to investigate ARO-ENaC in 24 healthy volunteers and 30 CF patients, and is…
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