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
The tech giant is investing $50 million in the Utah-based biotech to accelerate development of its AI foundation models for drug discovery.
The FDA recently approved the first cellular therapy for Type 1 diabetes and others may not be far behind. But experts say challenges still exist to the widespread application of these treatments.
For closing its $8 billion acquisition of Grail without regulatory approval, the European Union has slapped Illumina with a record $476 million fine—the maximum sanction allowed under the EU’s rules.
Following the regulator’s request for more data beyond the scope of its Phase III TIDES trial, Takeda decided to voluntarily withdraw the Biologics License Application for its TAK-003 dengue vaccine.
The company’s ruxolitinib cream Opzelura led to higher rates of treatment success in children with atopic dermatitis than with a control cream.
Severe respiratory problems killed seven patients and affected five others, though the company said that most of these were likely unrelated to the drug.
After paying $300 million upfront to BeiGene for option rights to ociperlimab in December 2021, Novartis has dropped the agreement and given the rights back to the Chinese biotech.
A handful of biotechs and pharma companies have posted sizable IPOs in 2023, despite a tight economy. Still, the numbers are way down from last year.
Burjeel Holdings and BridgeBio Pharma ink partnership deal in rare genetic diseases with a plan to set up headquarters in Abu Dhabi.
Bristol Myers Squibb is paying a total of $95 million to the two companies for exclusive global rights to programs developed within long-term partnerships designed to further BMS’ neuro pipeline.