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.
A project at Yale successfully adapted gene editing technology to correct the genetic mutation that is most commonly linked to cystic fibrosis.
Vertex will start the new year with a group of new eligible patients for cystic fibrosis drug Kalydeco, thanks to a Monday green light from the FDA.
The Cystic Fibrosis Foundation announced today that it had cashed in its royalty stream from Vertex Pharmaceuticals' breakthrough CF drugs it backed for a $3.3 billion windfall. But where the foundation trumpeted the blockbuster deal with Royalty Pharma as a prelude to more investments into next-gen drugs, the New York Times ' Andrew Pollack had no trouble finding a critic eager to lambaste the foundation for the ultimate sin: putting profits ahead of patients.
Dutch drug developer ProQR Therapeutics is swinging for a $75 million Wall Street debut to fund its work on a treatment for cystic fibrosis, looking to compete with market leader Vertex Pharmaceuticals.