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.
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Advaxis and Ayala Pharmaceuticals entered a reverse merger deal to focus on two clinical-stage cancer therapies: AL102 for desmoid tumors and ADXS-504 for prostate cancer.
Backed by Pfizer, Merck KGaA and J&J, Nucleome Therapeutics secured £37.5 million (about $42.16 million) in an oversubscribed Series A financing round.
Ambrx Biopharma Inc. announced it will lay off 15% of its workforce and suspend development of its lead candidate in a strategic realignment initiative.
Genetic engineering company Empyrean Neurosciences stepped into the CNS arena with $22 million and a small molecule platform derived from plants and fungi.
Salarius Pharmaceuticals voluntarily paused enrollment of its Phase I/II trial of seclidemstat for Ewing sarcoma and FET-rearranged sarcomas after a patient’s death.
GlaxoSmithKline (GSK) announced Tuesday morning it has struck a $70 million deal with Tempus in exchange for expanded access to Tempus’ AI-based patient data platform.
Ikena Oncology and AstraZeneca have entered into an agreement to evaluate Tagrisso (osimertinib) with Ikena’s IK-930 in EGFR-mutated non-small cell lung cancer.
Eli Lilly is acquiring precision medicine biotech Akouos for $487 million in cash in a deal focused on developing gene therapies for hearing loss.
As part of its third-quarter report, Roche announced it has cut two Phase II programs, one for hepatitis B and the other for geographic atrophy, a form of eye disease.
Mereo BioPharma will cut 40% of its headcount and significantly reduce expenses to support the advancement of its lead programs through the clinic.