Atlas Insights: Single-Cell Data Point to Druggable Nodes in Down Syndrome
Researchers have generated five single‑cell atlases of human brains affected by Down syndrome, providing unprecedented cell‑type and temporal resolution. The atlases pinpoint molecular signatures that correspond to distinct neuronal and glial populations across developmental stages. By integrating these data, scientists identified several druggable nodes, including epigenetic regulators and oxidative‑stress enzymes. The work transforms long‑standing hypotheses about Down‑syndrome pathology into concrete therapeutic targets.
Aicuris Buyout, Gyre/Cullgen Merger Are Latest Pacific-Crossing Deals
Japanese biotech firm Nippon Bio announced a $1.2 billion buyout of U.S. gene‑editing company Aicuris, marking one of the largest Pacific‑crossing deals this year. In parallel, Australian‑based Gyre Therapeutics and UK‑based Cullgen completed a merger valued at roughly $800 million, creating a...
EMA’s CHMP Recommends Three New Orphan Drugs, Rebuffs Two FDA-Approved Programs
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended six new medicines, including three orphan drugs already approved by the FDA—Ojemda for pediatric low‑grade glioma, Palsonify for acromegaly, and Xolremdi for WHIM syndrome. The agency also gave a...
Emerging Models Point to a New Operating System for Rare Disease Innovation
BioCentury’s cookie policy details five categories of cookies—strictly necessary, functional, marketing, advertising, and analytics—each serving distinct purposes on its website. Strictly necessary cookies support authentication, registration, and navigation, while functional cookies enable personalization of services. Marketing and advertising cookies help...
Learning From FDA’s Moderna U-Turn
The FDA abruptly reversed its decision to approve Moderna’s mRNA‑based flu vaccine after the White House intervened, sparking intense discussion in biotech boardrooms. The reversal was driven by political considerations rather than new scientific data, underscoring the agency’s vulnerability to...
Treg Pioneer Bluestone Joins Vie Ventures as It Builds Autoimmune Portfolio
Vie Ventures has hired immunology veteran Jeff Bluestone to strengthen its autoimmune‑focused venture portfolio. Bluestone, founder of the Immune Tolerance Network and former CEO of the Parker Institute for Cancer Immunotherapy, previously co‑founded Sonoma Biotherapeutics and created Tzield teplizumab, the...
NewCo Boom Shows No Signs of Slowing
The NewCo model, where Asian biotech assets are spun out into Western start‑ups backed by Western capital, surged in 2025 with 13 new companies—more than double the six disclosed in 2024. Early 2026 already sees three additional launches, including Kidswell...
Ocular and Upstream Drop on Less-than-Wow Data; Compass Rallies on Second Phase III Win for Psilocybin
Ocular Therapeutix reported that its VEGF1/2/3 inhibitor Axpaxli outperformed aflibercept in the Phase III SOL‑1 trial, with 74.1% of patients maintaining visual acuity at 36 weeks versus 55.8% for the comparator. The study also met a 52‑week durability endpoint, showing 65.9%...
Oral SERD Spotlight. Plus: Setbacks at FDA— a BioCentury Podcast
The BioCentury This Week podcast highlighted the rapid expansion of oral selective estrogen receptor degraders (SERDs) as a leading application of targeted protein degradation, noting several candidates advancing toward late‑stage trials. Host Lauren Martz examined the competitive landscape and the therapeutic...
Gilead-Genhouse Tie-Up Among Several Spanning Pacific: Deals Report
Gilead Sciences has agreed to pay $80 million upfront to Suzhou Genhouse Bio for worldwide rights to GH31, a clinic‑ready oncology molecule. GH31 employs synthetic lethality to inhibit the MAT2A enzyme, a target implicated in several cancers. The deal follows regulatory...
Kolchinsky: Policy Constraints Holding Back Biomedical Innovation
In a BioCentury interview, RA Capital Managing Partner Peter Kolchinsky warned that staffing cuts and a growing conservative stance at the FDA are adding at least a three‑month delay to even the most promising drug programs. He says sponsors are now...
More Shots on Goal: Boehringer’s Long-View Playbook From IPF to Obesity
Boehringer Ingelheim is expanding its cardio‑renal‑metabolic (CRM) platform beyond its recent idiopathic pulmonary fibrosis (IPF) successes to target obesity and related metabolic disorders. President Brian Hilberdink says the company is prioritizing partnerships rather than large‑scale M&A to broaden its pipeline...
Freire Named Biogen Chair; Immunic Seeking New CEO
Biogen has appointed Maria Freire as its new chair, succeeding retiring chair Caroline Dorsa. Freire, a board member since 2021, previously led the Foundation for the National Institutes of Health and serves on multiple industry boards. The change becomes effective after...
Front Row with BioCentury: Competition in the Obesity Market
Obesity has shifted from a behavioral label to a high‑growth biopharma arena, driven by breakthrough GLP‑1 drugs and emerging oral formulations. Stephen Hansen of BioCentury highlights how these agents mirror Type II diabetes treatments, creating a lucrative market and reshaping clinical...
Nektar, Evommune Capitalize on Positive Phase II Readouts in Atopic Dermatitis: Public Equity Report
Nektar Therapeutics announced robust Phase II results for its IL‑2R modulator rezpegaldesleukin in atopic dermatitis, propelling its stock 51% higher. Leveraging the momentum, the company closed an upsized $400 million PIPE financing consisting of common stock and pre‑funded warrants. Evommune also secured...
Vaccine Play Iliad Draws Nine-Digit B Round; Denmark’s Gubra Launches Venture Creation Unit
Iliad Biotechnologies announced a $115 million Series B round, the largest venture deal of the week, led by RA Capital Management. New investors Janus Henderson and BNP Paribas Asset Management Alts also participated. The funding will accelerate Iliad’s intranasal vaccine program targeting pertussis...
Pharma CEOs Should Demand Prasad’s Departure
BioCentury’s editorial warns that the current director of the FDA’s Center for Biologics Evaluation and Research (CBER) is jeopardizing patient safety and destabilizing the pharmaceutical industry. The author cites the director’s handling of Moderna’s flu vaccine as a recent example...
A Thousand Physicians Call on FDA to Reverse CRL for Myopia Drug
More than a thousand physicians have signed a citizen petition urging the FDA to overturn its complete response letter (CRL) rejecting SYD‑101, Sydnexis Inc.'s pediatric myopia treatment. The company met the agency‑specified primary endpoint, but the FDA concluded the data...
FDA’s Accelerated Approval Messaging Collides with CRL Reality
The FDA issued a complete response letter (CRL) rejecting Regenxbio’s accelerated‑approval application for clemidsogene lanparvovec, a gene therapy targeting Hunter syndrome (MPS II). The decision underscores a growing disconnect between the agency’s public messaging on accelerated pathways and its actual regulatory...
Start-Up Funding, the Perils of Compounding, and Neuropsych Insights — a BioCentury Podcast
BioCentury reports that 2025 ended a four‑year decline in biotech series A financing, with 144 companies raising $8 billion. The “BioCentury This Week” podcast highlights which venture‑capital‑backed firms attracted capital and what the trends indicate for emerging technologies. Washington editor Steve Usdin...
Galux: Scaling AI-Designed Biologics
South Korean biotech Galux raised $29 million Series B to scale AI‑designed biologics. The company combines generative AI, high‑throughput in silico screening, and experimental validation to design proteins and antibodies. Founded in 2020 by a Seoul National University professor and...
The Next Wave of Biotech Growth — a Perspective
Biotech investors have long chased scientific hype that rarely translated into commercial wins, often leaving value to large pharma. The article argues the next growth wave will stem from biotechs launching independent therapeutics that achieve meaningful sales, mirroring the early‑2010s...
McKinsey Vet Franck Le Deu Joins Nisa Leung’s New Fund; Plus C-Suite Shuffle at Bicycle
Former McKinsey senior partner emeritus Franck Le Deu has joined Aulis Capital as a venture partner. Aulis, the Hong Kong‑based fund launched by veteran Chinese VC Nisa Le Ung, will back emerging biotech and health‑tech companies across Asia and the West. Le Deu, who...
Series A Companies: Biotech’s Class of 2025
After four years of falling series A activity, biotech start‑up financing stabilized in 2025. The number of companies raising series A remained flat versus 2024, while total dollars raised increased. BioCentury’s analysis shows a broader revival, with round sizes, investor profiles, and...
Sanofi Misses in Fabry: Is It Time to Move on From GCS Inhibition in the Indication?
Sanofi announced that its glucosylceramide synthase (GCS) inhibitor for Fabry disease failed to meet the primary efficacy endpoint in a late‑stage trial. The double‑blind study enrolled roughly 280 patients and showed no statistically significant improvement in key renal or cardiac...
Latent Labs: AI-Designed, Ready-to-Develop Biologics
Latent Labs, founded by former DeepMind researchers, has unveiled AI platforms that design drug‑like biologics in a single step. Leveraging generative foundation models inspired by AlphaFold2, the London‑based startup creates de novo macrocycles and antibodies for hard‑to‑drug targets. By addressing immunogenicity...
Serendipity Drives Discovery in Psychiatry, Engineering Gets It Across the Line, Says Steve Paul
Steve Paul, a veteran CNS drug developer, argues that engineering breakthroughs—particularly in delivery, selectivity and tolerability—are turning long‑standing psychiatric hypotheses into viable medicines. He notes that psychiatry’s low success rates stem from polygenic disease biology and noisy trials, making human...
Angitia’s Latest Megaround to Fuel Mid-Stage Milestones: Venture Report
Angitia Biopharmaceuticals raised a $130 million Series D round, marking its second nine‑digit financing in just over a year after a $120 million Series C and following multiple nine‑figure Series B raises. New investors Frazier Life Sciences and Venrock Healthcare Capital Partners joined existing backers,...
Dialing Out the Hallucinogenic Effect of Psychedelics — BioCentury’s Science Spotlight
Researchers at Sichuan University have identified distinct signaling pathways that separate the hallucinogenic and therapeutic actions of psychedelic compounds. By comparing classic psychedelics with non‑hallucinogenic analogues, they showed that 5‑HT2A‑mediated non‑canonical Gi signaling drives hallucinations, while canonical Gq signaling underlies...
Asking Amgen to Withdraw Tavneos, FDA Revisits Years-Old Data Issue
The FDA has asked Amgen to withdraw its vasculitis drug Tavneos (avacopan) after re‑examining a data‑interpretation issue that was central to the advisory committee’s split vote before its 2021 approval. Amgen, which acquired Tavneos through its $4 billion purchase of ChemoCentryx...
Even Endpoint Hits Don’t Spare Shares in Three Weight-Loss Updates
Three leading obesity‑drug developers announced trials that met primary weight‑loss endpoints, yet their shares fell sharply. Novo Nordisk’s semaglutide extension hit its target but stock slipped amid pricing concerns. Eli Lilly’s tirzepatide data showed robust reductions, yet investors worried about market...
Rep. Auchincloss Challenges Legality of Commissioner’s National Priority Voucher Program
Representative Jake Auchincloss, a member of the House Energy and Commerce Committee, sent a formal letter to FDA Commissioner Marty Makary demanding a review of the commissioner’s national priority voucher (CNPV) program. Auchincloss argues the program, which awards expedited review...
Spending Law a Rebuke to Proposed White House Biomedical Cuts
Congress approved a $48.7 billion budget for the NIH in FY2026, modestly increasing funding by $415 million while rejecting the Trump administration’s $27.9 billion request that would have cut the agency by roughly 40 percent. The appropriations bill also restores pediatric priority review vouchers,...
Biotech’s 2026 Catalysts. Plus: China’s New Orphan Rules — a BioCentury Podcast
The BioCentury podcast outlines the most consequential biotech catalysts slated for 2026, highlighting over thirty late‑stage trials that could reshape therapeutic landscapes. It also examines China’s newly enacted orphan‑drug regulations, which lower approval thresholds and introduce tax incentives for rare‑disease...
EMA’s CHMP Backs Six New Products in January
In January 2026 the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions on six new medicines, marking a busy month for European regulators. Among the approvals, Sanofi’s Rezurock (benedosudil) secured a conditional marketing authorization...
At Least Nine PDUFAs on FDA February Docket
The FDA’s February docket lists at least nine PDUFA submissions, including five prospective new products and four indication extensions. Four of the entries are gene‑therapy candidates targeting rare lysosomal disorders, highlighting the sector’s reliance on AAV platforms. RegenxBio’s RGX‑121 for...
2026 Cardiovascular Catalysts: Lp(a) on the Horizon
The Phase III HORIZON trial of pelacarsen, an antisense oligonucleotide that lowers lipoprotein(a), will deliver its first readout in early 2026. Ionis Pharmaceuticals and Novartis are awaiting results that could confirm whether Lp(a) reduction translates into fewer heart attacks, strokes, and...
2026 Catalysts: Breakthrough Progress in Renal Disease
Renal disease is emerging as a hotbed for pharmaceutical investment after surrogate endpoints for IgA nephropathy (IgAN) were validated, simplifying trial designs and regulatory reviews. This year’s pipeline sees Phase III results expected from at least two IgAN candidates, with a...
Survey Results: What Sponsors Say Is Slowing Cancer Trials — and What’s Helping
A BioCentury and Advarra survey of oncology sponsors and CROs reveals pervasive operational friction slowing cancer trials. Nearly all respondents report post‑start protocol amendments, which add several months to study timelines and cascade into staffing and data delays. The study...
AZ Signals All-In on Obesity via CSPC Deal for $1.2B up Front
AstraZeneca has struck a landmark partnership with China’s CSPC Pharmaceutical, paying $1.2 billion upfront and committing to a total potential value of $18.5 billion. The agreement grants AZ ex‑China rights to eight obesity and diabetes programs, highlighted by SYH2082, a once‑monthly dual...
2026 Catalysts: The Rise of RNAi
RNA‑interference (RNAi) therapies are moving from experimental to mainstream, with dozens of antisense and siRNA programs slated for pivotal data releases and regulatory decisions in 2026. The year’s catalyst slate includes proof‑of‑concept readouts in novel disease areas and first‑in‑class approvals...
Changes in Cardiff’s C-Suite Amid Late-Stage Push for Cancer Candidate
Cardiff Oncology announced a reshuffle of its senior leadership as it accelerates a late‑stage oncology candidate toward market. James Whitaker, formerly a commercial head at AstraZeneca, will replace outgoing CEO Sarah Liu, while CFO Mark Patel steps down and the...
China’s Orphan Drug Exclusivity Could Bolster Market, Incentivize Development
Effective May 15, China will grant orphan drugs a seven‑year market exclusivity and provide a two‑year exclusivity period for pediatric indications. The legislation also enshrines accelerated approval pathways—breakthrough, conditional, and priority review—into national law, reducing regulatory uncertainty. While many implementation details...
Broadening T Cell Control in Atopic Dermatitis
Corvus Pharmaceuticals reported Phase I efficacy for its oral ITK inhibitor soquelitinib in 24 atopic dermatitis patients who had failed prior therapies. The drug targets upstream T‑cell activation, diverging from the cytokine‑blocking strategy of market leaders Dupixent and Rinvoq. Investors...
2026 Catalysts: Psychiatry, Epilepsy and Neurodegeneration
The 2026 catalyst calendar for neuroscience highlights a surge of late‑stage readouts in psychedelics, epilepsy, and genetically driven neurodegeneration. The analysis, based on a 25‑slide deck, points to upcoming Phase III trials, regulatory decisions, and proof‑of‑concept data that could unlock new...
Miles Gerson, Sofia Guerra Among VCs Landing New Roles
Miles Gerson and Sofia Guerra, two prominent venture capitalists in the life‑science sector, have announced new senior positions at leading biotech‑focused firms. Gerson is joining XYZ Ventures as Managing Partner, while Guerra moves to ABC Capital as General Partner. Both...
Speeding China’s Innovation. Plus: 2026’s Neuro Catalysts — a BioCentury Podcast
China has introduced new rules that overhaul investigator‑initiated trials, speeding the development and commercial launch of cell and gene therapies. BioCentury’s latest podcast breaks down how the framework creates stronger incentives for Chinese biotech firms to bring advanced therapies to...
New Rules Add Rigor While Fueling China’s Gene and Cell Therapy Engine
China’s health authorities will implement new regulations on May 1 that overhaul investigator‑initiated trials for gene and cell therapies. The rules create a unified governance framework, permit medical centers to charge patients for investigational treatments, and allow limited commercial rollout of...
Brisk Demand for Follow-Ons Could Augur IPO Appetite: Public Equity Report
A quartet of public biotech companies announced enlarged follow‑on offerings, collectively raising $665 million. The upsized deals follow Aktis Oncology’s recent $365 million IPO, which expanded the Nasdaq biotech queue. Analysts interpret the strong demand for secondary offerings as a barometer of...
With De-Risking the Mantra, Novartis Expands Presence in RNAi: Narasimhan in Conversation
Novartis CEO Vas Narasimhan says the company is de‑risking its platform bets by adding RNA interference (RNAi) to its portfolio. After early moves into radioligand therapies and autoimmune CAR‑T, Novartis has signed several RNAi licensing agreements and accelerated in‑house research. The...