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Ultragenyx to slash costs after late‑stage brittle‑bone failures
Ultragenyx disclosed failure of two phase‑3 trials in brittle‑bone disease and announced plans for major cost reductions. The company said interim analyses did not meet efficacy thresholds and...
NIH leadership churn deepens — neuro director exits amid instability
The NIH continues to lose senior leadership as National Institute of Neurological Disorders and Stroke (NINDS) director Walter Koroshetz, M.D., departs, leaving many institutes under interim...
Antifungal micelles enable combinatorial therapy: potent, lower toxicity
Researchers published a Nature Biotechnology paper reporting a self-assembling peptide micelle that codelivers an antifungal drug and a host‑defense peptide mimic to achieve combinatorial...
Single‑strand deaminase assists multiplex RNA edits: new editing platform
A Nature Biotechnology study details a single‑strand deaminase‑assisted RNA editing platform that rewrites multiple bases within single RNA transcripts. The authors present a modular system that...
CHANGE‑seq‑BE maps base‑editor off‑targets: unbiased genome‑wide profiling
Researchers published CHANGE‑seq‑BE in Nature Biotechnology, a tailored genome‑wide assay that sensitively and unbiasedly profiles off‑target activity induced by base editors. The method...
Directed evolution tightens adenine base editors – fewer bystanders
A Nature Biotechnology report shows a directed‑evolution campaign combined with extended guide RNAs produced adenine base editors with markedly improved precision and reduced bystander edits. The...
Palbociclib restores venetoclax sensitivity in AML: preclinical combo breakthrough
An Oregon Health & Science University team reported in Cell Reports Medicine that pairing venetoclax with the CDK4/6 inhibitor palbociclib produced stronger and more durable anti‑leukemia activity...
Rare regulatory variant slows clonal hematopoiesis: lowers leukemia risk
A Science paper from Vijay G. Sankaran’s lab reports a noncoding regulatory variant, rs17834140‑T, that weakens MSI2 activity and substantially lowers the risk of clonal hematopoiesis of...
Transcenta licenses HiCB continuous‑biomanufacturing tech to EirGenix
Transcenta Therapeutics announced a non‑exclusive license of its highly intensified continuous bioprocessing (HiCB) platform to Taiwan CDMO EirGenix, including continuous perfusion and hybrid...
Holiday deal rush: AbbVie buys ex‑China DLL3 T‑cell engager; Gilead picks up Polθ asset
Two licensing deals announced over the holidays reshuffled oncology pipelines. AbbVie paid $100 million upfront for ex‑China rights to Zelgen’s trispecific DLL3 T‑cell engager alveltamig, a deal...
Outlook plunges after third FDA CRL for bevacizumab biosimilar filing
Outlook Therapeutics received a third complete response letter for its bevacizumab formulation ONS‑5010 to treat wet age‑related macular degeneration, triggering an almost 62% intraday stock...
Ultragenyx to cut costs after phase‑3 failures in brittle‑bone trials
Ultragenyx reported failure of two phase‑3 trials in brittle bone disease and announced plans for a major cost‑reduction program. The company said the late‑stage readouts did not meet endpoints...
Directed evolution sharpens adenine base editors – extended guides reduce bystanders
Researchers reported two technical advances aimed at improving adenine base editor (ABE) precision. A study published in Nature Biotechnology used directed evolution to alter enzyme properties and...
Single‑strand deaminases rewrite RNA – multi‑base edits in one transcript
Two complementary papers in Nature Biotechnology unveiled single‑strand deaminase platforms that enable programmable, multi‑base editing of RNA transcripts. One report describes a...
Self‑assembling micelles co‑deliver antifungals – combination works in vivo
Nature Biotechnology published paired advances showing self‑assembling peptide and polymer micelles that co‑deliver antifungal drugs and host‑defense mimics to improve efficacy and safety. One...
SMRTnet AI predicts small‑molecule–RNA interactions without 3D structures
A deep‑learning model named SMRTnet was introduced to predict small‑molecule–RNA interactions using RNA secondary structure and multimodal training data, sidestepping the need for tertiary...
GWAS finds rs17834140‑T variant that restrains clonal hematopoiesis and leukemia
A large GWAS meta‑analysis spanning more than 640,000 individuals identified a rare noncoding variant, rs17834140‑T, that lowers risk of clonal hematopoiesis of indeterminate potential (CHIP) and...
Venetoclax plus palbociclib overcomes resistance in AML – OHSU data
Oregon Health & Science University researchers reported that combining the BCL2 inhibitor venetoclax with the CDK4/6 inhibitor palbociclib produced stronger, more durable anti‑leukemia responses...
Holiday licensing rush: big pharma buys clinical‑stage oncology assets
During the year‑end period major pharma deployed upfront payments to secure clinical oncology assets. Deals include AbbVie’s high‑value optioning of a DLL3‑targeting trispecific T‑cell engager and...
Transcenta licenses HiCB platform to EirGenix for continuous biologics manufacturing
Transcenta Therapeutics granted EirGenix a non‑exclusive license to its highly intensified continuous bioprocessing (HiCB) platform, including continuous perfusion and integrated hybrid continuous...