News
FDA’s rare disease decisions are strongest when the patient community has a voice in advisory committee decisions.
FEATURED STORIES
Psychedelics are gaining momentum in depression, with one treating physician predicting that the drug class could “wipe out the SSRIs” if safety and durability hold up.
Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.
Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Congressional letters sent to the CEOs of Eli Lilly, Pfizer, Merck, BMS and AbbVie this week voicing concerns about the pharmas’ clinical trials in China highlight an ongoing discrepancy in how government and industry think about the rise of the Asian country’s biotech industry.
THE LATEST
COVID-19 is turning out to be a particularly tenacious disease. And researchers are starting to understand why. Also, a new study found that COVID-19 deaths were higher in Republican-voting counties.
Bluebird bio faces a two-day crucible as the U.S. Food and Drug Administration’s Cell, Tissue and Gene Therapies Advisory Committee will give two lentiviral vector gene therapies a thumbs up or down.
The Forbion Growth Opportunities Fund II will invest mainly in later-stage biopharmaceutical firms in Europe, focusing on marketing treatments for diseases with high unmet needs.
Takeda announced results from its Phase III clinical trial on TAK-003, its dengue fever vaccine candidate, at the Northern European Conference of Travel Medicine.
Degron Therapeutics, based in Shanghai, announced Thursday that it has secured $22 million in Series A financing for its unique molecular glue-based targeted protein degradation platform GlueXplorer.
Precision BioSciences’ clinical program exploring allogeneic CAR T therapy may well produce a market pioneer after initial data showed its ability to deliver a 100% overall response rate.
Mineralys Therapeutics, a clinical-stage biopharmaceutical company focused on developing a best-in-class, novel therapy for high blood pressure, closed a $118 million Series B investment round.
The new space will enable Astellas to establish a global supply chain of gene therapies as well as widen the scope of the therapeutic products it creates.
South San Francisco-based Rigel Pharmaceuticals reported its Phase III Forward trial of fostamatinib did not show statistical significance in warm autoimmune hemolytic anemia
At ENDO 2022, Ascendis will share Phase II and III data showing clinical efficacy for its investigational product candidate for hypoparathyroidism. For this and more, see inside.