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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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After winning traditional approval from the U.S. regulator, Eisai’s Alzheimer’s disease therapy Leqembi has seen a sharp increase in patient uptake, with a target of 10,000 patients by March 2024.
Activist hedge fund investor Elliott Investment Management has bought over a $1 billion stake in BioMarin as the biotech grapples with a leadership change and disappointing sales of its hemophilia A gene therapy.
Newer hiring models based on skills applicants have learned and their general potential for growth could avoid the drawbacks of relying on degrees and experience.
A combination of Gilead Sciences’ domvanalimab and Arcus Biosciences’ zimberelimab, along with chemo, has shown an “encouraging” overall response rate in a mid-stage study.
Although it hit primary and secondary endpoints in a Phase II study, Ventyx Biosciences concluded that the results weren’t strong enough to face the stiff competition for the indication.
BioSpace has published its annual 2024 Best Places to Work list.
For $80 million upfront and the promise of more than $1.2 billion in milestones, Novartis has bought the global rights to develop an oral HDAC6 inhibitor from Chong Kun Dang Pharmaceutical.
The cell therapy-focused biotech will use most of the net proceeds from its initial public offering to fund Phase II clinical trials for its lead program, a novel CAR T-cell candidate.
In the third quarter, Vertex Pharmaceuticals reported revenue of $2.48 billion, a 6% increase from the same period in 2022. However, these figures fell short of Wall Street’s consensus estimates.
The Department of Health and Human Services in a court filing last week said that the blockbuster treatment could be “deselected” from the drug price negotiation list, given the recent approval of Amgen’s Stelara biosimilar.