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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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Mike Davis previously worked as clinical team leader at the FDA’s Division of Psychiatry before serving as chief medical officer for Usona Institute, a research organization advancing psychedelic science.
Despite Nektar’s share price shooting up as much as 175% on Tuesday, William Blair maintained that “there is an absence of clear differentiation” for rezpegaldesleukin in the atopic dermatitis space.
Robert F. Kennedy Jr. testified in front of largely combative congresspeople on vaccine policy, his MAHA report and more; the mass leadership exodus at the FDA continues as CDER and CBER shed key staff; Kennedy’s revamped CDC vaccine advisors convene for their first meeting; Novo and Lilly present new data at the American Diabetes Association’s annual meeting; and BioSpace recaps BIO2025.
In the latest installment of his column, Kaye/Bassman’s Michael Pietrack shares five ways leaders can help their teams after a layoff, from acknowledging emotions to reestablishing culture.
Leading companies spent $1.4 billion upfront on licensing deals and embarked on vast R&D programs. Clinical setbacks mean many companies are unlikely to ever recoup their investments.
With much to cover, Democrats tackled Kennedy’s MAHA report; the firing of all members of the CDC’s ACIP committee; and much more. Little was accomplished, as Kennedy demurred and members of Congress accused him of risking American lives.
In addition to cutting most of its staff, including two C-suite executives, Leap Therapeutics is winding down research and development activities and considering a sale or partnership opportunities.
Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C. Wainwright. Sangamo plans to use pivotal Phase I/II data to build an accelerated approval case for the asset.
In May, Revolution Medicines projected its cash and equivalents of $2.1 billion would last into the second half of 2027. With new funding from Royalty Pharma, the biotech has withdrawn that runway end date.
The star of Monday’s deal is gusacitinib, a small-molecule drug that Formation is developing for chronic hand eczema. Sanofi will explore additional indications for gusacitinib in a Phase I study.