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Ultragenyx prepares major cuts after two failed phase‑3 brittle bone trials
Ultragenyx Pharmaceutical announced phase‑3 failures in two brittle‑bone disease trials and signaled a significant cost‑cutting program in response. The company will restructure spending and...
FDA holiday rejections hit developers: Outlook’s third CRL and surprises for Sanofi, Corcept
Regulatory setbacks piled up over the holiday window as Outlook Therapeutics received a third complete response letter (CRL) from FDA for its bevacizumab biosimilar ONS‑5010, triggering a steep...
Adenine base editors refined—directed evolution cuts bystander noise
Researchers reported two complementary advances on Jan. 2, 2026 that aim to improve the safety profile of CRISPR base editors. One team used directed evolution and 3′-extended guide RNAs to reduce...
RNA editors go modular: single-strand deaminases rewrite transcripts
Two teams published new RNA-editing platforms that expand the scope and precision of transcript rewriting. One Nature Biotechnology paper describes a single-strand deaminase-assisted system that...
Host-defense peptide micelles... co-deliver drugs for potent antifungal combo
Researchers presented converging preclinical evidence that self-assembling peptide micelles can improve antifungal therapy. A Nature Biotechnology report describes a host-defense peptide mimic...
SMRTnet predicts small-molecule–RNA interactions without 3D structures
A new deep-learning framework called SMRTnet predicts small-molecule–RNA interactions using RNA secondary structure and multimodal AI models. The method fuses large-language models with...
Viral vectors retooled to edit macrophage RNA in vivo for sepsis
Scientists engineered a bioengineered viral delivery system that performs RNA editing directly in macrophages to modulate sepsis responses, according to a Nature Communications report. The...
Holiday deals replenish pipelines: AbbVie, Gilead make strategic buys; Blossomhill extends Series B
Big biopharma and venture investors moved over the holidays to shore up oncology and kinase pipelines. AbbVie agreed to pay $100 million upfront for an option to license Zejing’s DLL3-targeting...
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...