rare disease
CF patients, once left behind, may have a shot at treatment
Vertex Pharmaceuticals has nearly cornered the cystic fibrosis market: Its medications can work wonders in as much as 90% of patients with the inherited respiratory disease. But what of the rest? About 10% of cystic fibrosis patients have seen the lives of their peers change dramatically, while they have had to continue using old medications that do not work well.
Vertex hasn't forgotten about these patients. The company is beginning to test an mRNA-based drug that might target mutations that have yet to respond to treatments. And it's not just Vertex: A $500 million effort from the Cystic Fibrosis Foundation is emboldening other companies to use new approaches — including mRNA, gene therapy, even gene editing — to find one that might work.
"For so long, it felt like those of us in the 10% were watching innovation happening around us," one CF patient and advocate told STAT. "And for the first time, it feels like we're the focus."
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future pandemics
Karuna repurposing Goldfinch assets from kidney to psychiatric indications
Goldfinch Bio sputtered to a close this past week after running low on capital, and has begun distributing its various assets. In a $520 million deal, Karuna Therapeutics just acquired exclusive rights to Goldfinch's experimental drug GFB-887, along with other assets in a class of drugs called TRPC4/5 channel candidates. GFB-887 has been tested in Phase 2 studies for a kidney disease, but Karuna has other plans.
Karuna plans to use GFB-887 to treat psychiatric conditions, noting that the class of drugs its in demonstrated "anxiolytic and antidepressant properties" in preclinical and clinical data. The drug's mechanism "could represent a completely novel approach to treating mood and anxiety disorders," CEO Bill Meury said in a statement.
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