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
J.P. Morgan launched its new life science-focused private equity team, Life Sciences Private Capital, to support early- and growth stage biotech companies.
Verge Genomics dosed its first patient in a Phase I trial studying VRG50635, while Stealth Bio’s SBT-272 was granted Orphan Drug designation by the FDA.
Unity announced advances in diabetic macular edema via Phase II study results, potentially allowing its recipients to return to tasks of daily living, like driving.
Johnson & Johnson announced it was acquiring all outstanding shares of Abiomed for $16.6 billion to expand Abiomed’s cardiovascular technologies and increase patient access.
In a Phase III trial, Pfizer’s RSVpreF, a bivalent RSV vaccine, protected infants against severe medically attended lower respiratory tract illness when administered during the second or third trimester.
On Nov. 16, the FDA’s Cardiovascular and Renal Drug Advisory Committee will meet to discuss the relative merits of Ardelyx’s tenapanor in chronic kidney disease.
Monday, Actinium Pharmaceuticals released promising results Monday from a Phase III trial studying its lead radiotherapy candidate, Iomab-B, for patients with relapsed or refractory AML.
The implications of the Inflation Reduction Act of 2022 (IRA) are beginning to show as Alnylam drops the Stargardt indication for vutrisiran and Sanofi’s CEO reassures investors.
ESSA confirmed that Janssen has stepped away from a prostate cancer collaboration due to “operational recruitment challenges.”
Platform technology company Replay, based in San Diego and London, has launched Eudora, a gene therapy company targeting genetic diseases of the retina.