cystic fibrosis

Latest Headlines

Latest Headlines

Proteostasis and AveXis test the troubled IPO waters as biotech valuations wane

A pair of early-stage biotech companies has joined the thinning queue of drug developers trying to go public amid a painful market correction, angling to raise more than $135 million combined as the industry sputters.

Galapagos moves AbbVie-partnered drug into PhI as multipronged attack on CF takes shape

Galapagos has kicked off a Phase I trial of its AbbVie-partnered cystic fibrosis (CF) drug, GLPG2222. The start of the trial marks another milestone in the rolling advance of Galapagos' multipronged attack on CF, which is set to be bolstered later this year by the emergence of more next-generation correctors.

NIH commits $313M over four years to sequencing genomics of human disease

The National Institutes of Health has launched a program to explore the role of genomics in common diseases such as heart disease, diabetes, stroke and autism. This program has gained a commitment for $313 million in NIH funding from various agencies over the next four years.

Scripps team partially corrects impact of a cystic fibrosis mutation

Investigators focused on cystic fibrosis know that a mutation--ΔF508, or delta-508--in the gene that encodes a protein called the cystic fibrosis transmembrane conductance regulator interferes with the proper folding of CFTR needed for it to be processed in cells, making it a leading cause of the disease.

ProQR tanks after slow enrollment pushes back readout from cystic fibrosis trial

ProQR Therapeutics has pushed back the date on which it expects to deliver data from its cystic fibrosis clinical trials. The delay means it may be late 2016 before ProQR presents data on its challenger to Vertex Pharmaceuticals' grip on the cystic fibrosis market.

Australia's Paranta Biosciences raises A$7M for PhI of cystic fibrosis candidate

Australia's Paranta Biosciences has raised A$7 million to fund a Phase I clinical trial of cystic fibrosis treatment candidate PB01, adding to the A$16 million already raised since the company started operations in 2011, as hopes for biotech growth in the country get an official push.

Columbia U. team delivers small liquid drug doses to the lungs for targeted treatment

A team of researchers at Columbia University has developed a method of drug delivery to the lungs that could lead to more efficient dosing for many kinds of lung diseases using microvolume liquid concentrations. By delivering drugs directly to the disease site, treatment can be relegated to the necessary areas without causing dangerous side effects in other nearby organs.

Israeli nitric oxide drug-device player files for up to $36M Nasdaq IPO

Rehovot, Israel-based Advanced Inhalation Therapies (AIT) has thrown its hat in the ring to raise up to $36.2 million in an IPO on the Nasdaq Exchange. That's despite the recent rocky market and a dearth of med tech IPOs of late.

Gene therapy for cystic fibrosis improves lung function

A research consortium involving investigators at the University of Oxford has developed a gene therapy for cystic fibrosis that has demonstrated some proof-of-concept results that are encouraging new work on a hopefully more effective approach.

Imperial Innovations shopping cystic fibrosis gene therapy asset after posting clinical data

Imperial Innovations is negotiating with pharma companies over its gene therapy that has shown the potential to treat cystic fibrosis. A trial of the gene therapy posted patchy but promising data this week, putting the tech-transfer business in a position to strike a deal for further development.