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Big pharmas write off candidates—J&J, Genmab and Ultragenyx prune pipelines
Major companies moved to terminate or reprioritize assets after disappointing data. Johnson & Johnson halted its IL‑4/IL‑31 candidate JNJ‑95475939 following an interim futility analysis in atopic...
Neurology pipeline setbacks force closures and reprioritizations
The neurology sector saw two mid‑late stage setbacks over the holidays. Nido Biosciences closed after its lead neurologic candidate failed to show meaningful benefit in a phase 2 study, prompting...
FDA hands post‑holiday setbacks to Sanofi and Corcept
Regulatory decisions over the holidays resulted in disappointing news for two drugmakers. Sanofi and Corcept reported surprise rejections from the U.S. FDA for applications filed late in 2025,...
Regulatory wins for small markets: Vanda cleared, Axsome gets priority review
The FDA delivered two regulatory updates affecting modestly sized but commercially meaningful indications. Vanda Pharmaceuticals won approval for Nereus (tradipitant), the first new drug...
RNA editing leaps: Single‑strand deaminase rewrites transcripts
A team published a single‑strand deaminase‑assisted platform that enables multi‑base editing within individual RNA transcripts. The study, reported in Nature Biotechnology, demonstrates a...
Base‑editor safety…CHANGE‑seq‑BE maps off‑targets
Researchers introduced CHANGE‑seq‑BE, a genome‑wide method tailored to profile off‑target activity induced by base editors with high sensitivity. Published in Nature Biotechnology, the approach...
Adenine editors tightened: Directed evolution improves precision
Scientists used directed evolution and 3′‑extended guide RNAs to sharpen adenine base editor specificity, reducing bystander edits that hinder clinical applicability. The Nature Biotechnology...
Antifungal micelles: Host‑defense mimic codelivers drugs
Nature Biotechnology published a study showing that a self‑assembling host‑defense peptide mimic forms micelles that codeliver antifungal drugs, producing potent combinatorial activity and lower...
SMRTnet predicts drug‑RNA binds without 3D structures
Researchers unveiled SMRTnet, a deep‑learning framework that predicts small molecule–RNA interactions using RNA secondary structure and multimodal modeling, bypassing the need for tertiary...
Engineered viruses edit macrophage RNA to fight sepsis
A Nature Communications report described bioengineered viral vectors that edit RNA inside macrophages in vivo to modulate immune responses in sepsis models. The chemogenetic approach enabled...
Ultragenyx slashes spend after brittle‑bone phase‑3 flops
Ultragenyx reported failure in two phase‑3 trials for brittle bone disease and announced plans for major cost reductions. The late‑stage setbacks forced management to reassess R&D priorities and...
Big pharm pulls projects: J&J, Genmab drop candidates over hurdles
Johnson & Johnson halted development of JNJ‑95475939 after an interim analysis failed to meet the company’s efficacy bar, while Genmab shelved acasunlimab, a PD‑L1x4‑1BB bispecific. J&J acquired...
Vanda gets FDA nod – First new motion‑sickness drug in 40 years
The U.S. FDA approved Vanda’s oral tradipitant (Nereus) for preventing vomiting from motion sickness, based on three pivotal studies including two real‑world phase‑3 trials. The approval ends a...
FDA issues complete response to Corcept’s relacorilant
The FDA issued a complete response letter for Corcept’s relacorilant in Cushing’s syndrome, requesting additional efficacy evidence; the company said it will meet with regulators to chart a path...
FDA greenlights motion‑sickness pill: Vanda’s market win
The U.S. Food and Drug Administration approved Vanda Pharmaceuticals’ oral drug Nereus (tradipitant), marking the first new prescription treatment for motion sickness in more than four decades....
FDA grants priority review: Axsome’s AXS‑05 for Alzheimer’s agitation
The FDA accepted and granted priority review to Axsome Therapeutics’ supplemental new drug application for AXS‑05 (Auvelity) to treat agitation associated with Alzheimer’s disease, scheduling a...
FDA issues CRL to Corcept – relacorilant setback shifts focus to oncology
The FDA issued a complete response letter (CRL) to Corcept Therapeutics for relacorilant in hypertension secondary to hypercortisolism (Cushing syndrome), requesting additional evidence of...
Viral RNA editing in situ: new approach to treat sepsis
Researchers reported a bioengineered viral platform that performs RNA editing in macrophages in vivo to treat sepsis, as published in Nature Communications. The system combines targeted viral...
Gene therapy restores SynGAP1: seizure and behavior rescue reported
A preclinical gene‑replacement study reported restoration of SYNGAP1 function that rescued epilepsy and behavioral deficits in models of SYNGAP1‑related disorders, according to a recent...
AAV9 silence‑and‑replace prevents hereditary spastic paraplegia symptoms
Researchers published a silence‑and‑replace gene therapy using an AAV9 vector that prevented symptoms of hereditary spastic paraplegia (HSP) in preclinical models. The two‑pronged approach...