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Gene therapy approval in ultra-rare immune deficiency
Rocket Pharmaceuticals won FDA approval for Kresladi, a gene therapy for leukocyte-adhesion deficiency type 1 (LAD1), an inherited immunodeficiency that can be fatal in infancy. The company...
Phase 2 lupus readout advances immunology pipeline
Biogen reported positive Phase 2 results for litifilimab, an anti-BDCA2 antibody being developed for lupus. The program showed improvement in skin lesions at 24 weeks, with the company framing the...
AI-enabled targeting of immunotherapy support needs in survivorship care
Researchers at St. Jude Children’s Research Hospital published findings in Communications Medicine showing that AI methods can identify childhood cancer survivors likely to need additional support...
Biomarker test to guide gastric cancer immunotherapy selection
Zhejiang Cancer Hospital and Peking University researchers identified a stomach cancer biomarker intended to predict which patients are likely to benefit from neoadjuvant immunotherapy. The report...
Merck win signals dose strategy shift in Phase 3 heart failure program
At ACC, Merck indicated it is leaning toward using a lower Winrevair dose for a pivotal Phase 3 trial in an uncommon form of heart failure. The decision follows a Phase 2 signal described as...
Cancer drug delivery innovation funded by UKRI
The University of Manchester secured nearly £1 million from UK Research and Innovation (UKRI) to build snail-inspired soft robots for precision drug delivery in bowel cancer. The project aims to...
Targeted protein degradation platform expands proteasome chimeras
Casasampere, Carneros, Roda and colleagues published an approach in Nature Communications that extends the small molecule chimera strategy to directly exploit the 26S proteasome for targeted...
Corporate commercialization deal for AI-discovered oral therapeutics
Insilico Medicine and Eli Lilly inked a commercialization arrangement tied to Insilico’s AI-discovered preclinical candidates for oral therapeutics. The deal includes an upfront payment of $115...
NCI funding supports health-equity cancer research training
The University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center received a $3 million award from the National Cancer Institute for its ARCH K12 Program aimed at training the...
Home-based cervical cancer screening option for disabled women
Researchers at the University of Sheffield reported that home testing kits could help close a cervical screening gap for physically disabled women. The study assesses whether home-based pathways...
Phase 2-to-Phase 3 dose strategy for Merck’s heart failure drug
Merck is adjusting its development path for Winrevair (vericiguat/nearby class reference) in a pivotal Phase 3 trial after Phase 2 results showed its “pretty profound” benefit at the lowest tested...
Biogen advances lupus immunology with Phase 2 win
Biogen reported Phase 2 success for litifilimab in lupus, positioning the anti-BDCA2 antibody as a potential pillar of its immunology pipeline. The company said the therapy improved skin lesions...
Tanabe’s rare-disease strategy hits Phase 3 milestone
Tanabe Pharma disclosed positive Phase 3 data for an oral therapy targeting two rare diseases that cause pain when exposed to light. The company framed the result as its first major clinical...
Otsuka expands neuropsychiatric psychedelics pipeline via Transcend acquisition
Otsuka agreed to buy Transcend Therapeutics in a deal valued at about $1.2 billion, including roughly $700 million upfront, adding to a drug class designed to deliver psychedelic-like therapeutic...
EMA CHMP backs multiple drugs, including label and route changes
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of four new medicines in March and also weighed in on label expansions for existing...
On-the-radar drug pricing policy engagement with pharma
In private meetings, White House officials are working to win support from major pharmaceutical companies for a drug pricing bill, according to people familiar with the discussions. STAT+ reported...
MCED screening funding and CMS coverage ambitions
Maryland’s Department of Health awarded more than $520,000 to 18 groups of fire departments to run multi-cancer early detection screening using 20/20 BioLabs’ OneTest blood-based assay. The...
New implantable “living pharmacy” platform demonstrates multi-drug delivery
Researchers from Northwestern University, along with collaborators at Rice University and Carnegie Mellon University, reported progress toward implantable “living pharmacies” that produce drugs in...
Gut-immune mechanism in multiple sclerosis points to intestinal epithelial targets
Keio University researchers identified a mechanistic gut-immune link in multiple sclerosis-related neuroinflammation, reporting that intestinal epithelial cells promote development of pathogenic T...
Uterus perfusion device keeps donated tissue alive outside the body
A team at the Carlos Simon Foundation demonstrated a perfusion system (PUPER, “preservation of the uterus in perfusion”) that kept a donated human uterus alive outside the body for a day. The...