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Sec61 inhibition sprint: Pfizer and Enodia expand target validation
Pfizer has signed a research collaboration and licensing agreement with Enodia Therapeutics to discover and develop small-molecule inhibitors of Sec61, the translocon channel that helps synthesize...
Obesity licensing and cashflow: Roche backs ALK7 RNAi via Rona
Roche is extending its deal momentum in obesity and metabolic disease with a pact involving Rona Therapeutics’ RNA interference asset targeting ALK7, a genetically validated regulator implicated...
Gene therapy cash engineering: Ultragenyx sells rare pediatric PRV
Ultragenyx agreed to sell a rare pediatric priority review voucher (PRV) for $210 million, monetizing regulatory incentive value derived from its gene therapy GENGLYCOS (pariglasgene...
AI in Alzheimer’s-adjacent tau biology: Arrowhead’s brain-targeting RNAi tau knockdown
Arrowhead Pharmaceuticals reported early clinical data showing its experimental RNA interference drug ARO-MAPT can lower tau protein levels after subcutaneous dosing in healthy volunteers. The...
CAR-T control for solid tumors: genetic switch activates only on tumor contact
Italian Institute of Technology researchers described CAR-T cells engineered with a synthetic genetic switch that activates therapeutic genes only upon contact with tumor cells. The approach...
RNA therapeutics funding: Soufflé Therapeutics raises $135 million
Soufflé Therapeutics raised $135 million in venture capital to advance at least three oligonucleotide medicines in development, according to the report. The financing extends the runway for a...
Rare childhood cancer roadmap: experts prioritize targets for desmoplastic small round cell tumor
An international expert workshop identified priority therapeutic targets and trial designs to break a long-running treatment stalemate in desmoplastic small round cell tumor, an ultra-rare...
Vertex-era economics: NIH grant transparency plan triggers scientific ire
A proposed NIH change to how grant scores are revealed has drawn pushback from scientists concerned about reduced transparency and accountability in peer review. Under the plan, applicants would...
Antibiotic resistance containment: five-year surveillance shows divergent superbug trends
A five-year hospital surveillance program in China found that nearly half of clinical isolates were multidrug-resistant, with sharp divergence across pathogen species. Carbapenem-resistant...
UST: diagnostic AI in neonatal care trains across hospitals without data sharing
A multicenter effort evaluated an adult-trained AI model for newborn chest X-rays by enabling continual learning across 30 neonatal intensive care units without sharing patient data. The work is...
Deal activity reshapes oncology platform bets
Vir Biotechnology and Gilead struck a new solid-tumor T cell engager partnership, extending Vir’s oncology platform footprint into another large pharma collaboration. Financial terms include a $65...
Strategic capital and manufacturing scale-up in the US
UCB dedicated its future U.S. biologics manufacturing campus in Rowen, Georgia, marking a major step in expanding capacity for current and next-generation biologics. The planned $2 billion site is...
Obesity pharma investment accelerates via AI-designed therapeutics
Zealand Pharma said it is moving forward with AI-enabled protein-based obesity and metabolic disease programs through a partnership with Generate Biomedicines. Zealand plans to develop new protein...
Targeted immunology and cellular engineering in early human data
Ohio State reported early phase I results for an enhanced CAR T-cell approach targeting three B-cell proteins and incorporating OX40 costimulation. In the trial, CAR T cells were manufactured in...
First-in-human gene medicines expand with Prime Editing in patient cells
Aarhus University researchers demonstrated a prime-editing approach that corrected part of the disease-causing GATA2 deficiency defect directly in a patient’s own blood stem cells. The team...
Regulatory/market access shifts for global rare disease financing
Ultragenyx agreed to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million, following FDA approval of its one-time gene therapy GENGLYCOS (pariglasgene brecaparvovec-opnr)...
Clinical trial failure in obesity: risk signal for near-term GLP-1 pill strategy
Verdiva Bio terminated a Phase 2 trial of its lead GLP-1 pill, citing “market evolution,” according to company commentary reported in the update. The decision follows ongoing investor scrutiny...
Regulatory scrutiny and real-world safety debate in rare disease
STAT reported escalating safety concerns tied to Neurocrine Biosciences’ Vykat XR, a Prader-Willi syndrome therapy. Patient advocates and some experts are raising alarms after seven reported...
Synthetic biology platform expands with Pfizer Sec61 inhibitor collaboration
Enodia Therapeutics signed a research collaboration and licensing agreement with Pfizer targeting small-molecule inhibitors of the Sec61 translocon. Enodia will lead discovery activities to screen...
Cybersecurity and clinical AI safety: auditing medical models in production
Separate updates highlight the growing need to keep clinical and operational AI systems auditable and safe in real-world environments. One report discusses uncertainty-aware and calibration...