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The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
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The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.
Maintaining America’s momentum demands that policymakers resist policies that undermine research and development incentives.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
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Rapport Therapeutics tops this year’s list with $250 million in Series A and B financing in just six months.
While GLP-1 drugs have exploded in popularity, they don’t work for everyone, and experts say phenotyping based on a greater understanding of the disease is the future of obesity treatment.
Tome Biosciences has only been on the scene for a short time but on Tuesday notched an acquisition of CRISPR-based biotech Replace Therapeutics for $65 million upfront.
Anavex Life Sciences’ stock dropped by more than a third in value on Tuesday after results for a Phase II/III Rett syndrome study failed to impress investors.
Agios Pharmaceuticals met the primary endpoint in a late-stage trial investigating its drug mitapivat in treating adults with non-transfusion-dependent alpha- or beta-thalassemia.
After reporting positive mid-stage results for its LSD-based therapy for generalized anxiety disorder, MindMed’s chief medical officer sat down with BioSpace to discuss the path forward.
Greg Slabodkin, Heather McKenzie and Tyler Patchen discuss BioSpace’s tenth annual NextGen list of the hottest new life sciences companies.
In a collaboration agreement announced Wednesday, Boehringer Ingelheim will gain access to Ribo’s platform to target hepatocytes with investigational siRNA therapies for nonalcoholic or metabolic dysfunction-associated steatohepatitis.
Over the past year, obesity treatments have become the focus of intense media attention and discussion in healthcare — with increased focus on Wegovy and Zepbound. This attention has highlighted the need to address the global obesity epidemic.
The regulator greenlit 55 new drugs last year, seven of which belonged to Pfizer, including an ulcerative colitis treatment and a migraine nasal spray—both acquired in multibillion-dollar buys.