RO4938581, a GABAA-Α5 Negative Allosteric Modulator Rescued Behavioral and EEG Phenotypes of a Mouse Model of Dup15q Syndrome
The study shows that mice carrying the 15q duplication exhibit ~1.5‑fold elevation of GABA A‑α5 receptors in cortex, hippocampus and striatum, leading to heightened inhibitory synaptic activity and a characteristic increase in beta‑band EEG power. Chronic oral administration of the selective GABA A‑α5 negative allosteric modulator RO4938581 (10 mg/kg) partially normalized the EEG signature, reduced excessive inhibition, and rescued behavioral deficits such as impaired reversal learning and abnormal social interaction. These preclinical results link α5‑containing receptor overexpression to core Dup15q phenotypes and demonstrate that selective α5 NAMs can modify both electrophysiological and cognitive outcomes. The work provides mechanistic justification for pursuing α5‑targeted therapies in Dup15q syndrome.
A Better Way to Detect Off-Target Genome Changes From Base Editors
St. Jude Children’s Research Hospital unveiled CHANGE‑seq‑BE, a new assay that sensitively maps off‑target activity of CRISPR base editors while using only a fraction of sequencing resources. The method, published in Nature Biotechnology, demonstrated 95.4 % on‑target specificity in an FDA‑emergency case...
Epigenetic Mechanisms Affected by Stress During Adolescence and the Increased Risk for Depression Later in Life: A Systematic Review
This systematic review examined how stressful life events during adolescence reshape epigenetic landscapes and elevate depression risk later in life. By screening 30 preclinical and clinical studies, the authors identified consistent DNA methylation and micro‑RNA changes—particularly affecting the BDNF pathway—in...
Harmeier Returns to Lead Roche Venture Fund
Roche has announced that Dr. Harmeier is returning to head its Roche Venture Fund, the pharma giant’s dedicated early‑stage investment arm. Harmeier previously led the fund from 2015 to 2020 before moving to a senior role within Roche’s corporate development...
Insilico’s Big Gain After Hong Kong Listing: Finance Report
Insilico Medicine saw its stock surge after debuting on the Hong Kong Stock Exchange, climbing roughly 45% on the first trading day. The secondary offering raised about $1.2 billion, pushing the company’s market capitalization past the $10 billion mark. The strong demand...
Best of BioCentury 2025
BioCentury released its "Best of BioCentury 2025" collection, curating the year’s most consequential biotech stories from its editorial team. The anthology spotlights breakthroughs in gene‑editing, AI‑driven drug discovery, and evolving regulatory frameworks. By distilling these seminal moments, BioCentury reinforces its mission...
Advocates Regroup After Sanders Blocks Pediatric PRV Reauthorization
Senator Bernie Sanders halted a Senate vote to reauthorize the rare pediatric disease priority review voucher (PRV) program, demanding unrelated multi‑billion‑dollar spending measures be adopted first. Although he expressed support for the PRV incentive, Sanders made clear his conditions would...
Industry Outlook 2026: Success Through Top Talent, AI Utilization, and Sustainability
Ardena US managing director Ian Bilodeau says AI is reshaping pharma by linking fragmented data and speeding product decisions. He also emphasizes that attracting and developing top talent requires challenging, impact‑focused work environments. Sustainability, highlighted by Ardena’s EcoVadis certification, is...
The Evolution of Data-First Regulatory Operations
Remco Munnik of Arcana Life Sciences says European regulators are cementing a data‑first approach, with the EMA’s electronic product database and digital submission forms becoming standard. The pharma sector is eager to embed AI for document automation and quality oversight,...
Double Cone Tumble Blender Provides Homogeneity in Heavy-Duty Applications
Charles Ross & Son introduced the DCB-5 Double Cone Tumble Blender, a five‑cubic‑foot unit designed for high‑density powder processing. The machine combines a stainless‑steel jacket, a 2 HP intensifier bar, and an integrated vacuum system to deliver repeatable homogeneity and simultaneous...
A Zero-Shot Learning Framework for Maize Cob Phenotyping
Scientists have unveiled a zero‑shot learning framework that phenotypes maize cob geometry without any model retraining. The system combines text‑guided object detection, lightweight segmentation, and calibrated trait extraction, delivering 98‑100% detection accuracy and over 0.95 correlation for trait estimates. It...
From AI to Smart Factories: How Pharma Is Preparing for 2026
Manish Garg of Hikma Pharmaceuticals highlighted how AI, personalized medicine, and smart manufacturing reshaped pharma in 2025 and set the agenda for 2026. AI accelerated drug discovery and clinical design, while niche, high‑value therapies gained prominence. Companies are regionalizing supply...
New Technique Lights up Where Drugs Go in the Body, Cell by Cell
Researchers at Scripps have unveiled vCATCH, a whole‑body imaging platform that lights up covalent drugs at single‑cell resolution in mice. By attaching a tiny chemical handle to drugs and using highly selective click‑chemistry, the method tags each bound molecule with...
HITL and HOTL: An Air Traffic Control Analogy for Agentic AI
ArisGlobal’s senior VP Jason Bryant argues that both human‑in‑the‑loop (HITL) and human‑on‑the‑loop (HOTL) remain essential for deploying agentic AI in pharma. He uses an air‑traffic‑control analogy, portraying AI agents as aircraft and the orchestration layer as air‑traffic management. Bryant stresses...
Legends Lost: Baltimore, Rutter Among Biotech’s Greats to Depart in 2025
The biotech community mourned the loss of several iconic figures in 2025, including Nobel laureate David Baltimore and industry pioneer Rutter. Their careers spanned groundbreaking scientific discoveries, the founding of biotech firms, and the cultivation of global research ecosystems. The...
Industry Outlook 2026: The Impact of Novel Therapies
In 2025 the pharmaceutical sector saw a surge of novel therapies, notably next‑generation biologics such as bispecifics, fusion proteins, multi‑specifics, and a wave of antibody‑drug conjugates (ADCs). A breakthrough sub‑category, antibody‑oligo conjugates (AOCs), gained traction for muscular dystrophy, highlighted by...
Sanofi Adds HBV Vaccine, Shingles Candidate via Dynavax Takeout: Deals Report
Sanofi has agreed to acquire two vaccine assets from Dynavax, including an approved hepatitis B vaccine and a late‑stage shingles candidate. The transaction, announced in late December 2025, adds to Sanofi’s existing vaccine portfolio and broadens its reach in both...
Right Blood Pressure Drug Can Reduce Healthcare Costs
Researchers analyzed over 340,000 Swedish hypertension patients and found that initiating treatment with angiotensin‑receptor blockers (ARBs) leads to markedly higher long‑term medication persistence. After five years, 80 % of ARB starters remained adherent, versus 65 % for calcium‑channel blockers, the next best...
AI-Driven Breeding Strategy Aims to Boost Orphan Crops for Food Security
A team led by Prof. Xu Cao at the Chinese Academy of Sciences has proposed an AI‑empowered breeding framework, dubbed DSAP, to accelerate the domestication of orphan crops such as fonio, tef, and cowpea. The strategy integrates de novo genome editing,...
BioCentury’s 2025-26 Picks and Predictions. Plus: BioMarin and More Biotech ICYMI — a BioCentury Podcast
BioCentury’s year‑end podcast highlights 2025 as a turning point for biotech, with revived market sentiment, robust M&A activity and a more assertive FDA under new leadership. Analysts spotlight the $4.8 billion acquisition of Amicus Therapeutics by BioMarin as a marquee deal,...
2025 Was an Inflection Point. Will 2026 Show the Impact?
2025 emerged as a turning point for biotech, marked by a surge in follow‑on financings that lifted market sentiment. Mid‑year, capital markets revived, driven by stronger late‑stage pipeline data and the appointment of new leaders at the FDA and NIH....
Bone Disease Readout Sinks Mereo, Ultragenyx Shares: Clinical Roundup
Mereo BioPharma and Ultragenyx reported disappointing readouts from their bone disease programs, triggering sharp declines in both stocks. Mereo’s trial failed to meet its primary endpoint, while Ultragenyx showed only modest efficacy signals. The market reaction erased roughly 15% of...
Full Autonomy Is a ‘No-Go Zone’: Setting Parameters for Agentic AI in Pharma
ArisGlobal senior VP Jason Bryant explains that full autonomy for AI agents is off‑limits in pharmacovigilance, citing ethical and legal concerns. Instead, he advocates bounded autonomy managed by an orchestrator that can hand control to humans when needed. The discussion...
The Natural Human Protein Drug May Halt Neuron Death in Alzheimer's Disease
University of Colorado researchers found that the FDA‑approved drug sargramostim, a synthetic GM‑CSF protein, reduced the blood biomarker UCH‑L1 of neuronal death by 40% in Alzheimer’s patients, bringing levels down to those seen in early life. The study also documented...
Structural Covariance, Regional Topology, and Volumetric Aspects of Amygdala Subnuclei in Posttraumatic Stress Disorder Using Ultra-High Field Imaging
Using ultra‑high‑field 7 T MRI, researchers examined amygdala subnuclei volumes, network topology, and structural covariance in 73 PTSD patients, 78 trauma‑exposed controls, and 59 non‑trauma controls. Whole‑amygdala size was unchanged, but the lateral nucleus showed opposite volume shifts: larger left lateral...
Navigating Digitalization, QRM Maturity, and Global Compliance Convergence Into 2026
Henrik Johanning of Epista Life Sciences outlines the European regulatory and manufacturing roadmap for 2026, emphasizing the operationalisation of modern quality risk management (QRM) and the need for tangible upgrades to meet EU GMP Annex 1 requirements. He highlights rising digital...
The Top 10 PharmTech Videos of 2025
The PharmTech roundup reviews the ten most‑watched videos of 2025, revealing a industry pivot toward complex modalities such as AAV‑based gene therapies, high‑concentration biologics, and radiopharmaceuticals. Across the series, manufacturers stress the "CGT 2.0" model—flexible, automated, data‑driven production—to overcome scale‑up bottlenecks....
Researchers Develop Graphene Oxide Hybrid Electrodes for Real-Time Dopamine Monitoring
Researchers at SKKU, HKUST and Jeonbuk University unveiled SIDNEY, a graphene‑oxide‑wrapped hybrid electrode that enables real‑time, label‑free dopamine detection in living neurons and brain organoids. The nanostructured platform combines gold nanopillars with a thin graphene‑oxide coating, achieving a detection limit...
When Biotech Makes Christmas Miracles Happen — Second Edition
The article revisits three recent biotech breakthroughs that felt like miracles: base‑edited CAR‑T cells (BE‑CAR7) delivering remission for relapsed T‑ALL, ex vivo gene‑corrected skin grafts curing severe junctional epidermolysis bullosa, and prenatal enzyme replacement therapy mitigating infantile Pompe disease. Each case...
The Gut Bacteria that Put the Brakes on Weight Gain in Mice
University of Utah researchers identified the gut bacterium Turicibacter as a potent modulator of metabolic health, showing it markedly reduces weight gain, blood sugar, and blood lipids in mice fed a high‑fat diet. The microbe’s effect stems from a suite...
Groundbreaking Discovery Turns Household Plastic Recycling Into Anti-Cancer Medication
University of St Andrews researchers have demonstrated a ruthenium‑catalysed semi‑hydrogenation that depolymerises household PET waste into ethyl‑4‑hydroxymethyl benzoate (EHMB). EHMB is a versatile intermediate for high‑value drugs such as the cancer therapy Imatinib, as well as tranexamic acid and the...
A DIY, Fly-Powered Food Waste Recycling System
University of California‑Riverside researchers have engineered a DIY black‑soldier fly bioreactor that converts on‑site food waste into high‑protein larvae and nutrient‑dense frass. The system uses off‑the‑shelf materials, operates under a single caretaker, and yields roughly one pound of larvae per...
Accelerated Cancer Drug Approvals Deliver Limited Survival Gains at High Cost
A BMJ Medicine study examined Medicare’s use of FDA accelerated‑approval cancer drugs from 2012‑2020. Of the 178,000 beneficiaries treated, only 45% received drugs that later proved to extend survival, adding an estimated 76,000 life‑years. The three most beneficial drugs accounted...
New Microfluidics Technology Enables Highly Uniform DNA Condensate Formation
Researchers at Chuo University introduced a vibration‑induced local vortex (VILV) platform that creates highly uniform DNA condensate droplets using a low‑cost piezoelectric vibrator. The system replaces traditional microfluidic pumps with stable micro‑vortex arrays generated on a simple micropillar device, enabling...
Artificial Metabolism Turns Waste CO₂ Into Useful Chemicals
Northwestern and Stanford researchers have engineered a fully synthetic, cell‑free metabolism called the Reductive Formate Pathway (ReForm) that converts CO₂‑derived formate into acetyl‑CoA and subsequently into malate, a high‑value chemical. The pathway relies on five engineered enzymes arranged in six...
Tiny Viral Switch Offers Hope Against Drug-Resistant Bacteria
Researchers at Hebrew University identified a tiny phage‑encoded RNA, PreS, that reprograms bacterial host cells during infection. PreS binds to the folded region of the bacterial dnaN mRNA, unfolding it and boosting production of the DnaN replication protein. The resulting...
Brinsupri Setback Slices Insmed Market Cap — Clinical Report
Insmed’s experimental therapy Brinsupri failed to meet its primary efficacy endpoint in a Phase 2 trial involving 150 patients with rare lung disease, triggering a sharp market reaction. The company announced the setback on Dec. 19, 2025, and its market...
A Non-Profit Brought an Abandoned Rare Disease Gene Therapy to Market. Can the Model Scale?
A non‑profit, Fondazione Telethon, partnered with a U.S. charity to bring a lentiviral stem‑cell gene therapy for Wiskott‑Aldrich syndrome to market. The FDA approved the product, Waskyra etuvetidigene autotemcel, marking the first time a non‑profit acted as the regulatory applicant. The therapy...
Raising the Sun: Japan Biotech Looks to Level Up
Japan’s biotech industry is entering a growth phase as the government unveils a ¥200 billion fund and regulatory sandbox to speed drug development. Venture capital activity surged 45% year‑over‑year, fueling a wave of startups focused on gene therapy and rare‑disease platforms....
Seeking ‘Continuous’ Run of Deals, BioMarin Adds Fabry, Pompe Drugs via $4.8B Amicus Takeout
BioMarin Pharmaceutical announced a $4.8 billion acquisition of Amicus Therapeutics, a move designed to fuel a continuous stream of strategic deals. The transaction will bring two marketed orphan drugs—one for Fabry disease and another for Pompe disease—into BioMarin’s portfolio, together projected...
Cai Succeeding Zhang as Head of CSPC
Cai has been appointed to replace Zhang as head of China Starch & Pharmaceutical Co. (CSPC), the country’s largest generic drug manufacturer. The transition was announced in December 2025 and is effective immediately. Cai brings a background in AI-driven drug...
U.S. Vaccine Approvals to Undergo Overhaul: What Do the Changes Mean?
The U.S. FDA has disclosed a draft overhaul that would tighten vaccine approval standards, requiring developers to submit expanded safety and efficacy data and potentially subject annual flu shots to large‑scale trials. Simultaneously, the CDC withdrew its universal hepatitis B vaccination...
Johnson & Johnson’s Hematology Portfolio: Breakthroughs to Watch
Johnson & Johnson showcased a robust hematology pipeline at the ASH 2025 meeting, unveiling more than 60 new abstracts. The company highlighted real‑world evidence from thousands of patients, underscoring the efficacy of its CAR‑T, bispecific and gene‑editing therapies. Notably, the...
NU-9 Halts Alzheimer's Disease in Animal Model Before Symptoms Begin
Northwestern researchers report that the small‑molecule NU‑9 eliminates a newly identified toxic amyloid‑beta oligomer subtype in a pre‑symptomatic mouse model of Alzheimer’s disease. Daily oral administration for 60 days dramatically reduced reactive astrogliosis, neuroinflammation, and associated TDP‑43 pathology across multiple...
Using AI In Patent Practice: Practical and Ethical Issues
At the BIO International Convention’s IP Counsels Committee panel, experts outlined how AI is reshaping patent practice. They classified tools into traditional, generative, and patent‑specific solutions, stressing secure enterprise versions for confidential data. Ethical pitfalls—including AI hallucinations and potential public...
Stelios Papadopoulos Brings the Long View on Biotech on The BioCentury Show
Stelios Papadopoulos, former Biogen chair and current Exelixis leader, warned that biotech faces heightened pricing pressure and rising competition from China despite a surge of over $3 billion in capital in a single day. He argued that the sector can no...
How Alphamab Is Differentiating in Crowded Cancer Targets
Alphamab is launching a next‑generation bispecific antibody‑drug conjugate (ADC) platform that simultaneously engages two tumor antigens while employing a lower drug‑to‑antibody ratio (DAR). The company argues that this design improves the therapeutic index by delivering potent payloads more selectively and...
Third Rock Backs Steve Paul’s Latest Schizophrenia Spinout in $165M Round: Venture Report
Third Rock Ventures led a $165 million financing round for Steve Paul’s newest schizophrenia‑focused biotech, marking a significant venture capital commitment to neuropsychiatric innovation. Paul, a serial biotech founder, will use the capital to move novel mechanisms from discovery into early...
Early Signals Stack Up: Two Small Molecules Activate GCase in Parkinson’s
Gain Therapeutics reported Phase Ib data showing that its small‑molecule program reduces glucosylceramide substrate in the central nervous system, confirming target engagement of glucocerebrosidase (GCase). In parallel, Vanqua Bio presented early evidence that its distinct compound activates peripheral GCase while...
Induced Pluripotent Stem Cells: From Dish to Freezer and Back
Kobe University researchers have devised a cryopreservation protocol that freezes induced pluripotent stem cells (iPSCs) directly in their 2‑dimensional culture dishes. The method uses the inexpensive amino acid D‑proline combined with a synthetic polymer and a brief enzymatic step to...
