Excelsior Sciences: Automating Small Molecule Chemistry
Excelsior Sciences, backed by Deerfield, unveiled an automated platform for small‑molecule discovery that leverages modular "smart blocs" and generative AI. The system integrates iterative carbon‑carbon bond formation, robotic synthesis, and in‑vitro assays into a continuous make‑test‑learn loop. By translating chemical features into a standardized, machine‑readable format, the platform aims to replace artisanal chemistry with scalable, data‑driven processes. Co‑founder Martin Burke’s university‑originated methodology underpins the technology’s core chemistry.
HHS Unveils MANA Plan
On April 1, 2026, the U.S. Department of Health and Human Services (HHS) unveiled a new initiative dubbed the MANA plan, short for “Make America Normal Again.” The announcement was delivered by HHS Secretary Robert F. Kennedy Jr. during a briefing at the...
Zag Bio: Rewiring the Thymus for Antigen-Specific Tolerance
Zag Bio, a Cambridge‑based biotech, announced an $80 million Series A round to advance its lead program that rewires the thymus for antigen‑specific tolerance. The company’s approach employs bispecific antibodies to ferry self‑antigens into thymic cells, aiming to re‑educate T‑cells and induce...
RFK Jr.’s Peptide Deregulation Threatens the Foundations of Drug Safety
HHS Secretary Robert F. Kennedy Jr announced on a podcast that the federal government will lift restrictions on 14 injectable peptides, allowing compounding pharmacies to produce and sell them without the usual FDA review. The move bypasses the scientific advisory...
Going ‘Golden’: K-Biotech May Be Prepping for Its Moment on the Global Stage
Korean biotech is gaining global visibility after the East‑West Biopharma Summit highlighted its first‑in‑class pipelines. The Korean Ministry of Health and Welfare announced that Eli Lilly will invest $500 million over the next five years to collaborate with domestic firms. The deal...
MHRA’s Tallon on the Tall Order of Boosting the U.K. as a Destination for Clinical Trials
Lawrence Tallon, the MHRA chief, is set to oversee the launch of the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025 on April 28, 2026. The amendment represents the most extensive overhaul of U.K. clinical‑trial rules in two decades, targeting faster approval,...
AL-S Pharma Tests How Far SOD1 Biology Extends Into Sporadic ALS
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Recursion’s Khan: AI Will Be Judged by the Medicines It Makes
Recursion Pharmaceuticals CEO Najat Khan says AI’s worth will be judged by the medicines it helps create, not by the sophistication of the tools. She emphasizes deploying AI selectively in chemistry, trial execution, and out‑of‑domain predictions to generate clinical proof...
R1: China-to-U.S. NewCo Planning Global Testing of New CKD Mechanism
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Rare Disease Spotlight: Friedreich Ataxia Moves Beyond Mitochondrial Bandages
Friedreich ataxia (FA) received its first FDA‑approved therapy in 2023 when omaveloxolone, marketed as Skyclarys, earned accelerated approval. Biogen’s $7.3 billion acquisition of Reata Pharmaceuticals secured the drug and highlighted the market’s appetite for rare‑disease assets. Skyclarys works by activating the...
Kenai: Off-the-Shelf Parkinson’s Progenitors with Precision Transplant Tech
Kenai, a new allogeneic cell‑therapy company, is developing off‑the‑shelf iPSC‑derived progenitor cells for Parkinson’s disease, paired with a precision brain‑delivery platform that uses real‑time functional imaging. The approach builds on Japan’s recent approvals of iPSC‑based Parkinson’s treatments and aims to...
Solid Eyes Accelerated Approval as DMD Gene Therapy Faces FDA Uncertainty
Solid Biosciences reported Phase I/II data for its DMD gene therapy SGT‑003, showing higher microdystrophin expression and a greater proportion of positive fibers than Sarepta’s Elevidys. The results strengthen SGT‑003’s case for accelerated FDA approval, but the pathway is clouded by...
Can FDA Tolerate Cancer Risk for Rare Pediatric Disease Gene Therapies?
The FDA placed a clinical hold on Regenxbio’s RGX‑111 and RGX‑121 gene‑therapy trials after a pediatric MPS I patient developed a tumor four years post‑treatment. The case marks the first documented long‑latency cancer linked to an adeno‑associated virus (AAV) vector in...
Science Spotlight: New Ways to Attack Β-Amyloid Plaques in Alzheimer’s
Two pre‑clinical studies propose active clearance of β‑amyloid as a new Alzheimer’s strategy. Researchers at Washington University engineered astrocytes with chimeric antigen receptors (CARs) that engulf plaques, while another team designed bispecific peptides that ferry amyloid into cells for lysosomal...
Roche’s Giredestrant Miss Refines Treatment Settings for Oral SERDs
Roche’s oral selective estrogen receptor degrader (SERD) giredestrant failed to meet its primary endpoint in a late‑stage Phase III trial, but the data revealed meaningful activity in specific patient subgroups, particularly those with ESR1 mutations. The miss prompted Roche to...
RTW-Backed Prolium Emerges with $50M a Year After China Licensing Deal: Finance Report
Prolium Bioscience, a NewCo‑style biotech backed by RTW Investments, announced its public debut after securing a $50 million financing package. The capital influx follows a licensing agreement with China’s Innocare Pharma and Keymed Biosciences, granting the partners rights to develop Prolium’s...
EpiciphAI: Reading Disease States From Blood’s Epigenetic Fingerprints
EpiciphAI, a Chinese pre‑seed biotech, is building a liquid biopsy that reads histone modifications on cell‑free chromatin to pinpoint a fragment’s tissue of origin and its disease state. Unlike most epigenetic tests that focus on DNA methylation, this platform targets...
Promising Henlus Data Tripped up by Psychedelic Trial Design Dilemma
Helus Pharma reported Phase II data for its psychedelic candidate HLP004, indicating symptom improvement in generalized anxiety disorder patients for up to six months. However, the trial’s low‑dose active control arm showed similar efficacy, making it difficult to isolate the...
Promising Helus Data Tripped up by Psychedelic Trial Design Dilemma
Helus Pharma reported Phase II data for its psychedelic candidate HLP004 in generalized anxiety disorder, showing symptom improvement lasting six months. However, the trial’s active low‑dose control produced outcomes similar to the therapeutic dose, making it difficult to isolate the drug’s...
Asia Deals Aim for First-in-Class Biology, with at Least Four Deals for New Targets
Asian biotech firms outside China announced 72 deals in 2024‑25, emphasizing first‑in‑class assets over me‑better or me‑too programs. At least four transactions involve novel targets such as CNTN4, GPR52, MTARC1 and OK‑1, spanning new modalities and indications. Global pharmaceutical companies...
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...
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
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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...
