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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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An independent panel of advisers voted to approve Genentech’s Polivy after internal FDA staff raised concerns regarding the efficacy and risk-benefit profile as a first-line treatment for large B-cell lymphoma.
Three years after the FDA rejected its initial biologic license application, Mesoblast announced its resubmission for Ryoncil. Now, the treatment is getting a second chance.
Women are often at the forefront of innovation, making up nearly half of the life sciences industry despite a shrinking but persistent wage gap. Inceptor Bio is at the forefront of closing inequality.
If biopharma-focused co-working spaces continue to catch on, they could expand job opportunities for those outside of the typical hot spots.
BioMarin announced Tuesday that the FDA has accepted its supplemental New Drug Application to expand Voxzogo for children younger than five years with achondroplasia.
Volastra Therapeutics has in-licensed Amgen’s sovilnesib, a KIF18A inhibitor being studied for high-grade ovarian cancer, triple-negative breast cancer and other solid tumors.
BridgeBio posted data Monday from its small but highly anticipated Phase II study of infigratinib in achondroplasia - a drug that could give BioMarin a run for its money.
Merck presented Phase IIb data at ACC Monday showing that its hypercholesterolemia hopeful met the primary endpoint.
Moderna may seek the FDA’s accelerated approval for its personalized cancer vaccine, president Stephen Hoge said Monday.
The FDA set a decision date of July 6, 2023 for Eisai and Biogen’s recently approved Alzheimer’s drug, Leqembi, Eisai announced Monday.