News
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.
FEATURED STORIES
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.
FROM OUR EDITORS
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.
THE LATEST
After reaching a settlement with Thermo Fisher earlier this month, the family of Henrietta Lacks is now suing Ultragenyx for its “unjust enrichment” using their matriarch’s immortal cell line.
Insilico’s AI platform has attracted interest from a growing number of biopharma companies. Meanwhile, several other firms highlight their own AI technologies.
The Supreme Court of the United States has granted the Department of Justice’s motion for a stay regarding Purdue’s bankruptcy case and is set to assess the legality of the settlement in December.
At AAIC, researchers presented studies using CRISPR-Cas9 in animal/organoid models of Alzheimer’s disease, but some experts doubt that such approaches will successfully translate to humans.
The Securities and Exchange Commission has joined the mounting antitrust scrutiny of Illumina’s acquisition of the cancer diagnostics company, according to an SEC filing by the DNA sequencing giant.
The FDA has four decisions on deck this week, including ones for two rare disease treatments from Ipsen and Regeneron.
Amid record sales of its obesity treatment Wegovy, as well as diabetes drugs Ozempic and Rybelsus, Novo is buying Canadian biotech Inversago Pharma to further boost its weight-loss pipeline.
Despite being heavily pretreated, patients on J&J’s Talvey saw a 73.6% overall response rate in a Phase II study, winning accelerated approval from the regulator.
The company has lowered its 2023 guidance amid leadership changes and antitrust scrutiny. Illumina now expects a 1% increase in revenue, down from its previous 7% to 10% growth forecast.
After the regulator rejected avasopasem manganese, its candidate for severe oral mucositis, Galera is implementing a restructuring initiative that involves reducing its headcount by 70%.