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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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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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There are many reasons why you might want to negotiate your stock options. Read on to discover what stock options are and how to negotiate your stock options with your employer.
Private investors, governmental institutions and longtime life sciences investors provided funding to develop therapies for cancers, dermatological problems and acute ischemic strokes.
Janssen shared positive updates from ongoing trials on candidate therapies for Crohn’s disease and ulcerative colitis at Digestive Disease Week in San Diego, California.
Vanda has again taken legal action against the FDA over its supposed failure to grant tradipant Fast Track designation. In its complaint, Vanda is challenging the FDA’s decision to deny the designation.
This summer, BioSpace will release a series of reports looking at the current state of diversity and inclusion in the life sciences.
Ocugen introduced NeoCart, which is designed to repair full-thickness lesions of the knee cartilage in adults, as a Phase III cell therapy platform technology.
A blood test to diagnose Alzheimer’s disease launched in April by Quest Diagnostics has the potential to screen patients for risk years before symptoms appear.
Genocea’s board of directors voted to wind down ongoing operations and terminate its remaining staff, except for those necessary to see the company’s closing.
Alnylam Pharmaceuticals announced Tuesday new positive results from the Phase III ILLUMINATE-C trial of lumasiran for advanced primary hyperoxaluria type 1 (PH1).
If approved, etranacogene dezaparvovec would be the first gene therapy treatment for hemophilia B.