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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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Project 2025, a blueprint for a potential second Trump term that highlights the IRA as a potential target, took a starring role in this week’s Democratic National Convention.
The regulator on Thursday said the mRNA vaccines, Pfizer-BioNTech’s Comirnaty and Moderna’s Spikevax, will better protect against currently circulating variants as COVID continues to surge in many parts of the U.S.
Despite the promise of its technology and funding from big backers, Tome Biosciences is “operating at reduced capacity” and weighing its “strategic options” as the Massachusetts-based company faces an uncertain future.
The Danish biotech on Thursday reported stronger-than-expected earnings and forecasts hitting the upper range of its full-year revenue target amid the ongoing mpox outbreak.
The Oncologic Drugs Advisory Committee in a Sept. 26 meeting will discuss whether the regulator should restrict approval of checkpoint inhibitors based on PD-L1 expression levels.
Looking to build on the success of Chinook Therapeutics, founded by Versant in 2019 and acquired by Novartis last year, the companies on Thursday launched Borealis Biosciences with $150 million in funding to develop RNA therapeutics for kidney diseases.
With the potential passage of the BIOSECURE Act looming, WuXi Biologics reported a 24% drop in net profit in the first half of 2024.
As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.
With the help of third-party investors, the new venture will focus on three genetic and rare diseases: tuberous sclerosis complex, erythropoietic protoporphyria and alpha-A1 antitrypsin deficiency.
While approved by the Medicines and Healthcare products Regulatory Agency, the Alzheimer’s drug failed to win the backing of the U.K.’s National Institute for Health and Care Excellence, which said that its benefits were “too small to justify the cost.”