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 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.
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.
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.
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 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.
With its cystic fibrosis combo drug awaiting a likely approval at the FDA, Vertex is beefing up its pipeline in the field, paying $80 million upfront and promising up to $1.1 billion more in milestones to partner with Parion Sciences on a new class of CF-related drugs.
A panel of FDA advisers voted in favor of Vertex Pharmaceuticals' closely watched combination treatment for cystic fibrosis, looking past some efficacy doubts and setting the stage for a likely approval.
As an FDA review nears for Vertex's Kalydeco-lumacaftor tandem--approval of which could help Kalydeco reach 10 times the number of cystic fibrosis patients it currently does--the agency's staffers have some concerns.
Vertex Pharmaceuticals has made a major difference--and a lot of money--in cystic fibrosis with its targeted treatments, drugs tailored for patients with particular genetic defects. But upstart Spyryx Biosciences believes it may have discovered a way to treat all forms of CF with a single therapy, and the company raised $18 million in Series A cash to advance the idea.