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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...
Regulatory approvals—gene therapy
The FDA cleared Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), naming it the first gene therapy option for this...
Regulatory updates—Europe mpox indication withdrawal
The European Medicines Agency (EMA) moved to withdraw the mpox indication for Siga Technologies’ tecovirimat while keeping the drug approved for smallpox, cowpox, and adverse reactions to vaccinia...
Immunology pipeline—Phase 2 lupus data
Biogen reported Phase 2 success for litifilimab, its anti-BDCA2 antibody in systemic lupus erythematosus, advancing the program toward what the company is targeting as a potential pillar of its...
Drug development—Phase 3 rare disease pain after light exposure
Tanabe Pharma disclosed positive Phase 3 results for an oral drug targeting two rare diseases characterized by pain upon light exposure, marking the company’s first major clinical milestone since...
Oncology—RET fusion trial readout (Phase I/II)
Final efficacy and safety data from the Phase I/II ARROW study of pralsetinib were disclosed for patients with advanced RET fusion-positive non–small cell lung cancer. The publication is framed as...
Biopharma M&A—Otsuka acquires Transcend Therapeutics
Otsuka announced it will buy Transcend Therapeutics for $700 million upfront (about $1.2 billion including potential milestones), moving into a neuropsychiatric asset intended to promote rapid...
Immunology biology—gut epithelial MHC II links to MS neuroinflammation
Researchers at Keio University mapped a mechanistic gut–immune link to multiple sclerosis–related neuroinflammation, reporting that intestinal epithelial cells can drive encephalitogenic T-cell...
Drug delivery and advanced platforms—implantable living pharmacy
A multi-institution team led by Northwestern University advanced implantable “living pharmacy” technology, engineering cells to continuously produce multiple therapies inside the body. Published...
AI and healthcare operations—clinical trust and governance
A policy-and-operations-focused perspective argues that healthcare leaders won’t get value from autonomous AI if they avoid redesigning how clinical decisions are measured, governed, and audited....
Diagnostics and cancer screening—real-world MCED expansion in Maryland
Maryland’s Department of Health awarded $520,000 to 18 fire departments to run multi-cancer early detection (MCED) screening using 20/20 BioLabs’ OneTest assay, funding more than 1,400...
Regulatory approvals – gene therapy for ultra-rare immune disease
The FDA has granted accelerated approval to Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel), positioning it as the first gene therapy option for severe leukocyte adhesion deficiency-I...
Phase 3 readouts – lupus immunology pipeline signal
Biogen reported Phase 2 efficacy in lupus with litifilimab, its anti-BDCA2 antibody being positioned as a pillar of its immunology pipeline. In the company’s program update for skin involvement,...