News

A pancreatic enzyme replacement product that is not derived from pigs — Sollpura (liprotamase) — was as good at maintaining the height and weight of cystic fibrosis (CF) patients as a pig-derived product, according to a clinical trial. The information came from the extension period of Anthera’s Phase 3…

The naturally occurring molecule thymosin α1 (Tα1) reversed tissue defects in mice with cystic fibrosis (CF) and in human cells that contained a mutation associated with the disease, according to a study. The findings mean that Tα1 could be a promising treatment for CF patients carrying the F508del mutation in…

Enterprise Therapeutics has received a £95,000 grant from Britain’s Cystic Fibrosis Trust to develop innovative treatments for cystic fibrosis (CF). The money, equivalent to $118,000, will support the development of CF therapies based on Enterprise Therapeutics’ bronchosphere technology platform. Cystic fibrosis blocks airway passages. The condition stems from faulty airway-surface…

AbbVie is accepting applications for its 2017 AbbVie CF Scholarship program that helps to support outstanding young adults with cystic fibrosis (CF) as they pursue higher education. A total of 40 scholarships, each worth $3,000, are available for the current 2017–18 academic year. Awards based on established criteria that…

Spyryx Biosciences presented new data and updates on its cystic fibrosis (CF) treatment SPX-101 at two recent events: the European Cystic Fibrosis Society Basic Science Conference, which took place March 29-April 1 in Albufeira, Portugal, and the invitation-only Needham & Company’s 16th Annual Healthcare Conference, held April 4-5 in New…

The first patient has been dosed in a Phase 2 clinical trial evaluating RPL554 as a treatment for cystic fibrosis (CF), according to its maker, Verona Pharma. RPL554 is a first-in-class drug candidate designed to inhibit the enzymes phosphodiesterase 3 and 4 (PDE3/PDE4). It has been shown to have…

Dutch pharmaceutical company ProQR Therapeutics now has two key patents protecting its first-in-class ribonucleic acid (RNA)-based oligonucleotide QR-010 for the treatment of cystic fibrosis (CF) in the United States and Europe. The patents are valid until at least July 2033. The new U.S. patent, announced April 3, covers methods of targeting…

Corbus Pharmaceuticals recently reported that its Phase 2 clinical trial examining multiple doses of anabasum (formerly known as JBT-101 or Resunab) compared to placebo in patients with cystic fibrosis (CF) achieved the trial’s primary endpoint demonstrating an acceptable safety and tolerability profile at all doses, with no serious or severe adverse…

Vertex Pharmaceuticals recently announced positive results of two Phase 3 clinical trials, the EVOLVE and EXPAND studies, evaluating the effects of a combination of tezacaftor (VX-661) and ivacaftor (sold under the brand name Kalydeco) in cystic fibrosis (CF). The results showed that the new combined therapy significantly improved…

David M. Rodman is the new chief development strategy officer at ProQR Therapeutics, a leader in RNA therapies for cystic fibrosis (CF) and other diseases. Rodman, a medical doctor, will be tasked with advancing ProQR’s treatment candidates. Dr. David M. Rodman. Photo credit: ProQR A key therapy…