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Cell therapy safety: CAR-T trial pauses after fatalities
Novartis and Bristol Myers Squibb moved to pause and investigate CAR-T programs after serious immune complications. Novartis said it is halting and reviewing several clinical trial programs...
Rare disease: FDA approval of first therapy for Alexander disease
Ionis Pharmaceuticals secured FDA approval for Zanvastro, described as the first disease-modifying therapy for Alexander disease, a rare and deadly neurological condition. The therapy is approved...
Rare neurodevelopment: Ultragenyx’s Phase 3 Angelman failure
Ultragenyx reported a Phase 3 failure for apazunersen (GTX-102) in Angelman syndrome, missing both the primary and key secondary endpoints in its Aspire trial. The antisense oligonucleotide did...
Immuno-oncology: AbbVie’s etentamig Phase 3 win in myeloma
AbbVie announced positive Phase 3 results for etentamig, a T-cell engager targeting multiple myeloma. The company said etentamig cut the risk of disease progression or death by 60% compared with...
CNS immunology: epicardial NRG1–ERBB4 pathway stimulates heart regeneration
Researchers reported that unloading the heart can boost cardiomyocyte regeneration via an epicardial NRG1–ERBB4 signaling pathway. The study points to a specific inter-tissue molecular route that...
Virology–HIV cure research: venetoclax reduces intact SIV reservoir in macaques
Emory University researchers reported that a short course of venetoclax, a BCL-2 inhibitor approved for certain blood cancers, reduced the intact SIV reservoir in rhesus macaques when given at...
Celiac disease: Teva’s gluten challenge positive in antibody trial
Teva reported success for its experimental anti-celiac antibody in a gluten challenge study, saying the therapy significantly reduced gluten-induced intestinal damage compared with placebo over...
Medtech/devices: Selio hydrogel device to prevent pneumothorax in CT-guided lung biopsy
Selio Medical obtained FDA clearance for a hydrogel device designed to seal CT-guided lung biopsy punctures before pneumothorax can occur. The product targets the most common and costly...
Metformin and Down syndrome: restores mitochondrial quality control
A new study reported that metformin can restore mitochondrial quality control in Down syndrome fibroblasts. Published in Cellular and Molecular Life Sciences, the work links the drug to...
FDA approvals / accelerated approval
The FDA cleared Ionis Pharmaceuticals’ first disease-modifying therapy for Alexander disease, approving Zanvastro for children and adults following results from a pivotal trial. The primary...
Late-stage failures and pipeline reset
Ultragenyx Pharmaceuticals’ Phase 3 program in Angelman syndrome collapsed, with apazunersen (GTX-102) failing both key endpoints in the Aspire study. The result cuts against early signals that...
Gene therapy regulatory push
UniQure moved to secure regulatory momentum for Huntington’s disease, submitting applications to the FDA and the UK’s MHRA for AMT-130 and requesting priority review. The company is seeking...
Next-gen drug delivery licensing
Novartis agreed to pay up to $3.2 billion to Alteogen for its ALT-B4 subcutaneous drug delivery platform, adding another major Big Pharma validation of Hybrozyme technology. The deal grants...
Cancer clinical development: bispecific / PD-1xVEGF
Akeso and Summit Therapeutics reported updated Phase 3 data for ivonescimab, arguing the updated dataset clears the trial’s overall survival threshold bar in certain lung cancer patients. The...
Immuno-oncology dealmaking and preclinical pipeline
Roche licensed SIM-0600, a preclinical trispecific T-cell engager from Simcere Zaiming Pharmaceutical, in a potential $1.53 billion transaction. The agreement centers on a CD79a/CD19/CD3-targeting...
Virology / HIV cure strategies using approved oncology drugs
Emory University researchers reported that short-course venetoclax, a BCL-2 inhibitor approved for blood cancers, reduced the intact SIV reservoir in rhesus macaques when administered at the start...
Psychedelic neuroprotection in oncology
Researchers at UT MD Anderson reported in Science that psilocybin can prevent chemotherapy-induced peripheral neuropathy in mice. In models using platinum-based chemotherapy, two psilocybin doses...
Cardiovascular risk imaging with AI
Japanese researchers reported that variational autoencoder models can detect coronary artery disease in myocardial perfusion SPECT images more accurately than standard quantitative methods. The...
Biopharma trial/regulatory operations tooling
A research team proposed a standardized benchmarking tool for clinical trial sites, designed to identify operational inefficiencies across recruitment, protocol adherence, and regulatory...
FDA approval — rare disease first-in-class antisense therapy
Ionis Pharmaceuticals won FDA approval for Zanvastro, the first disease-modifying therapy for Alexander disease, in a pivotal program designed around walking speed as a core motor endpoint. The...