The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all types of mutations in the CFTR gene. The funding will establish a multi-year program aimed at boosting the development…
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A single dose of MRT5005, Translate Bio‘s first-in-class messenger RNA (mRNA) therapy for the treatment of cystic fibrosis (CF), improves patients’ lung function, interim data from a Phase 1/2 trial shows. While stressing these are early results, the researchers called the findings “promising.” CF is a…
Treatment for three years with Kalydeco (ivacaftor) reduces the risk of lung infection by serious pathogens — including Pseudomonas aeruginosa and Staphylococcus aureus — in people with cystic fibrosis (CF), a U.K. study reports. The study, “Ivacaftor Is Associated with Reduced Lung Infection by Key Cystic Fibrosis…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
People using a nebulizer while lying on their side showed no significant loss in a treatment’s overall deposition in the lungs, a study in healthy adults and in cystic fibrosis (CF) patients reports. Rather, it found that alternating sides instead of sitting upright may better deposit a medicine’s active compounds…
Employees at the Development Bank of Wales, in the U.K., have raised £30,400 (around $37,000) to support the Cystic Fibrosis Trust. Every year, bank employees are asked to nominate a charitable organization to support, with which they have a personal connection. This year, the Cystic Fibrosis Trust was…
The first patient has been dosed in a Phase 2 clinical trial testing Proteostasis Therapeutics‘s combinations of three investigational therapies for cystic fibrosis (CF), the company announced. The three treatments under investigation are all designed to fix the defective CFTR protein due to mutations in the CFTR gene, which is the…
Jeffrey Leiden, who in seven years has taken Vertex Pharmaceuticals to the cusp of developing treatments for about 90% of people with cystic fibrosis (CF), is exchanging his day-to-day leadership role as CEO for an elder statesman position. Leiden — who finished high school at 15, earned…
Five programs helping to empower the cystic fibrosis (CF) community were selected to receive up to $10,000 each under the fourth annual Impact Grants awarded by the Cystic Fibrosis Foundation (CFF). Launched in 2016, the Impact Grants program is designed to support new ideas and projects that aim…
The European Commission (EC) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) granted Mylan the right to market TOBI Podhaler (tobramycin inhalation powder) and TOBI Solution (tobramycin nebulizer solution) in the United Kingdom, the company announced. The decision came after the Committee…
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