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BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
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Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
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Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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The Taiwan-based company is establishing a manufacturing center for the U.S. market.
The pact between Madrigal Pharmaceuticals and Ruzhou Ribo Life Sciences could complement the former’s Rezdiffra, the first FDA-approved therapy for MASH. That drug made $287.3 million in the third quarter of 2025.
With 10 launches planned in the coming quarters, Gilead isn’t feeling the same acquisition urgency as its pharma peers—though the prospect of a takeover isn’t off the table.
The percentage of patients achieving total clearance of eczema lesions in a Phase 2b trial increased with prolonged rezpeg treatment.
After receiving a letter directly from CBER Director Vinay Prasad, Moderna said the FDA had previously signed off on the use of a licensed flu vaccine as a comparator for a Phase 3 study of mRNA-1010.
A lawsuit and FDA warning ensued after Hims & Hers launched a compounded version of Novo Nordisk’s new obesity pill, more Big Pharma report earnings—including from weight loss rivals Novo and Eli Lilly—and the gene therapy space sees another rejection.
Looking for a biopharma job in Boston? Check out the BioSpace list of nine companies hiring life sciences professionals like you.
A rapturous response to data published last year for Pelage’s hair loss candidate overwhelmed the biotech. Now, the company is ready to show the world the science behind the breakthrough.
Pfizer, Eli Lilly, Novartis, Bristol Myers Squibb and AstraZeneca are all ramping up the use of AI, but drug discovery is not the primary success story—yet.
The newly public Evommune shared data showing that EVO301, an IL-18 targeting protein, cleared symptoms comparably to Regeneron and Sanofi’s mega-blockbuster in a mid-stage atopic dermatitis clinical trial.