STAT+: Pharmalittle: We’re Reading About a U.S. Senator Probing FDA, BioNTech Founders Leaving, and Much More
BioNTech co‑founders Ugur Sahin and Özlem Türeci will leave the company by year‑end to launch an unnamed mRNA‑focused venture, with BioNTech retaining a minority stake and licensing key technology. The split allows BioNTech to concentrate on its late‑stage cancer pipeline and continued Covid‑19 vaccine sales. Meanwhile, Republican Senator Ron Johnson announced a Senate investigation into the FDA’s recent denials of rare‑disease treatments, seeking written complete response letters and possible testimony from top officials. The probe adds political pressure on an agency already facing scrutiny from patients and investors.
China Is Going After the World's Most Expensive Drugs: Endpoints Signal
China is actively targeting ultra‑expensive gene therapies, challenging the notion that these treatments are immune to price competition. Recent regulatory approvals and centralized procurement initiatives have forced manufacturers to negotiate steep discounts on therapies that once commanded multi‑million‑dollar price tags....
FDA Widens Use of Leucovorin without New Trial Data
The U.S. Food and Drug Administration has approved the generic form of leucovorin, also known as folinic acid, for treating cerebral folate deficiency (CFD). The decision widens the drug’s label despite the absence of new clinical trial data, relying on...
Cell and Gene Therapy Manufacturing Market to Skyrocket to $146B by 2032
Credence Research forecasts the global cell and gene therapy manufacturing market to surge from $19.3 billion in 2024 to $146.2 billion by 2032. The compound annual growth rate is estimated at 28.8%, propelled by rising demand for advanced therapies, expanding commercialization, and...
AI-Focused Fund Breakout Raises $114M To Support Early-Stage Biotechs
Breakout Ventures announced the close of its third fund, raising $114 million to back AI‑driven biotech startups. The capital will be deployed to early‑stage companies, including a University of Chicago spin‑out focused on computational small‑molecule design and a stealth venture tackling...
Statement on Biomedical Countermeasures Initiative
BIOTECanada welcomes the launch of the National Research Council of Canada’s Biomedical Countermeasures Initiative, a key component of the federal Defence Industrial Strategy. The program is designed to strengthen Canada’s domestic capacity to develop and manufacture diagnostics, vaccines, and therapeutics...
Rising Amid Flurry of CAR T Deals, Stylus Proves Cell Therapy Is Not Dead
Stylus Medicine entered the cell‑therapy arena in May 2025 with an in‑vivo CAR‑T platform that delivers a lipid nanoparticle‑encapsulated recombinase to engineer T cells inside patients. The move comes after major pharma acquisitions—BMS buying Orbital Therapeutics for $1.5 billion and Gilead...
Samsung Biologics, Eli Lilly to Establish Biotech Incubator in Incheon
Samsung Biologics and U.S. pharma leader Eli Lilly have signed an agreement to launch a biotech incubator in Incheon’s Songdo district. The facility will host Lilly’s Gateway Labs platform, offering lab space, equipment, funding and R&D collaboration to emerging biotech firms....
Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data
Dyne Therapeutics reported that its exon‑skipping candidate z‑rostudirsen sustained respiratory and cardiac benefits through 24 months in the Phase 1/2 DELIVER study for Duchenne muscular dystrophy. The therapy maintained forced vital capacity, circumferential strain and left‑ventricular ejection fraction improvements compared with...
Junshi Receives China’s NMPA Acceptance for Toripalimab Injection
Junshi Biosciences has received acceptance from China’s National Medical Products Administration for its subcutaneous toripalimab injection (JS001sc), covering 12 cancer indications. The filing marks the first domestic anti‑PD‑1 monoclonal antibody in a subcutaneous formulation to reach the marketing application stage....
Ascletis Announces Positive Topline Results From U.S. Phase II, 24-Week Study for Its Ultra-Long-Acting Subcutaneous Depot Formulations of Small Molecule...
Ascletis Pharma reported positive topline data from a U.S. Phase II, 24‑week study of its ultra‑long‑acting subcutaneous depot GLP‑1R agonist ASC30. The once‑monthly formulation A1 produced a placebo‑adjusted mean weight loss of 7.5% at week 16 and sustained a 5.8%...
BioNTech Co-Founders to Exit Company and Start a New One
Uğur Şahin and Özlem Türeci announced they will leave BioNTech by the end of 2026 to launch a new company focused on next‑generation mRNA technologies. BioNTech will concurrently narrow its portfolio, concentrating on late‑stage therapeutic candidates and its existing vaccine...
STAT+: Large Drugmakers Are Developing Fewer Antibiotics, Analysis Finds
A new analysis shows the world’s largest pharmaceutical firms cut antimicrobial development by 35% over the past five years, dropping from 92 to 60 candidates. Only five of the 39 pipeline projects aimed at WHO priority pathogens include pediatric formulations...
NewcelX and Eledon Partner for NCEL-101 Programme
NewcelX has entered a collaborative research agreement with Eledon Pharmaceuticals to advance its NCEL‑101 cell therapy for type‑1 diabetes. The partnership integrates NewcelX’s off‑the‑shelf islet replacement product with Eledon’s anti‑CD40L monoclonal antibody, tegoprubart, which has been used in over 100...
For the First Time, Ferring Reports Revenue of Over €2.5 Billion in 2025
Ferring Pharmaceuticals reported revenue above €2.5 billion for 2025, a 7% increase at actual exchange rates and 10% at constant rates. Growth was powered by its flagship reproductive medicine Menopur and the U.S. ramp‑up of gene‑based bladder‑cancer therapy Adstiladrin. Operating profit...
Why the FDA Is Embracing Old Math for New Drugs
The FDA released draft guidance encouraging the use of Bayesian statistics in drug and biologic clinical trials, aiming to shorten development timelines and lower costs. By allowing external data—known as priors—to be incorporated, the approach promises more efficient, adaptive studies,...
Single Pivotal Trials Demand Stronger Data and Risk Strategies
Following the FDA’s recent shift to require only one pivotal trial for new drug applications, sponsors now face heightened pressure to generate robust efficacy and safety data. Regulators expect a single, bullet‑proof study rather than two less conclusive trials, mirroring...
Rapport Therapeutics Partners with Tenacia Biotechnology to Advance RAP-219 in Greater China
Rapport Therapeutics has granted Tenacia Biotechnology exclusive rights to develop and commercialize its TARPγ8‑specific AMPA receptor negative allosteric modulator RAP‑219 in Greater China, covering indications such as focal onset seizures and bipolar mania. The agreement provides Rapport with a $20 million...
How Voluntary Exercise Reshapes Tryptophan Metabolism Through the Gut Microbiota
A study in *Brain Medicine* shows that eight weeks of voluntary wheel running in adult male rats reshapes the gut microbiota, notably decreasing the tryptophan‑metabolizing genera Alistipes and Clostridium. The microbial shift coincides with altered serum metabolites, including a rise...
Mouse Brain Study Reveals Why Blockbuster Weight-Loss Drugs May Work Differently in Females and Males
Researchers at Icahn School of Medicine created the first sex‑specific atlas of GLP‑1 expression in the mouse brain using RNAscope, mapping the peptide across 25 nuclei. The atlas shows pronounced differences between females and males, especially in hindbrain nuclei of...
Cost–Benefit Analysis for Synthetic Nucleic Acid Screening in the European Union
RAND Europe, commissioned by Sentinel Bio, delivered a EU‑wide cost‑benefit analysis of synthetic nucleic acid synthesis screening. The study compares three policy options—voluntary guidance, EU‑funding conditionality, and mandatory regulation—over a ten‑year horizon. Results show all options generate net societal benefits,...
Nonprofit Aims To Modernize Manufacturing by Clearing Capital Hurdle
A nonprofit, the API Innovation Center (APIIC), is tackling the capital barrier that prevents U.S. drug makers from adopting continuous manufacturing. By pooling state, federal, philanthropic and private funds, APIIC installs equipment at manufacturers at no upfront cost, de‑risking the...
Portfolio of SCN2A Gene Variants, and More
A new preprint maps a broad portfolio of SCN2A gene variants onto the Nav1.2 sodium channel, revealing distinct functional impacts that correspond to neurodevelopmental outcomes. Loss‑of‑function mutations linked to non‑syndromic autism reduce channel activity, while co‑expression with wild‑type proteins produces...
Other News to Note for March 10, 2026
Merck released phase III data from its Litespark‑011 trial, showing a potent HIF‑2α inhibitor that could reshape renal‑cell carcinoma therapy. Researchers at INSERM uncovered hypothalamic tanycytes as a primary mechanism for clearing pathological tau, opening new avenues for Alzheimer’s treatment....
Resident Macrophages Play a Role in Maintaining Murine Intraocular Pressure
Duke University researchers discovered that resident tissue macrophages (RTMs) are essential for maintaining intraocular pressure (IOP) in mice. Fluorescent tagging showed that selective removal of RTMs clogged the eye's outflow pathway, causing fluid buildup and elevated IOP, while depletion of...
Biontech SE (BNTX) Q4 2025 Earnings Call Transcript
BioNTech’s Q4 2024 earnings call highlighted a strategic pivot toward oncology, emphasizing the advancement of its bispecific antibody BNT327 and the recent acquisition of Biotheus to secure Chinese manufacturing and development capabilities. The company reported positive Phase 2 data for...
Legend Biotech Corp (LEGN) Q4 2025 Earnings Call Transcript
Biogen reported full‑year 2025 non‑GAAP diluted EPS of $15.28 and $9.9 billion in revenue, a modest 2% YoY increase. Growth‑product sales surged 19% to $3.3 billion, driven by VUMERITY, SKYCLARIS, ZERZUVE and CALSADI, while legacy MS revenues are projected to fall mid‑teens...
Mpox Immune Test Validated During Rwandan Outbreak
Researchers from the University of Birmingham, Rwanda Biomedical Centre and the University of Rwanda have validated an IgG ELISA assay for mpox antibodies during the clade 1b outbreak in Rwanda. The MpoxCARE test, built on four key antibody signatures, demonstrated high...
Hong Kong: Tech Centre Fosters AI-Driven Healthcare Innovation
The Hong Kong Productivity Council has opened the Future Life and Health Tech Centre, a research‑development hub designed to fast‑track AI‑driven innovations across medical technology, biotechnology, functional foods and modernised traditional Chinese medicine. The facility offers an end‑to‑end platform that...
Hidden Blood Mutations Drive Severe Inflammatory Bowel Disease, but a New Treatment Target Is in Sight
Indiana University researchers linked clonal hematopoiesis of indeterminate potential (CHIP) to heightened severity of inflammatory bowel disease. Analysis of UK Biobank and All of Us data showed women with DNMT3A mutations and younger individuals with TET2 mutations face higher Crohn’s...
Gene Edit Makes Probiotic Safer for Immunocompromised Patients
An international team genetically deleted the ENA1 gene from Saccharomyces boulardii, a common probiotic yeast. In immunosuppressed mice, the ENA1‑deficient strain showed no mortality, raising survival from 30‑40% to 100% compared with wild‑type isolates. The edit also reduced osmotic stress...
Stay or Stray? Why some Gut Microbes Persist After Fecal Transplants
Researchers at King's College London identified genetic markers that determine whether donor microbes persist after fecal microbiota transplantation (FMT). By tracking biosynthetic gene clusters in 86 healthy adults over a year, they distinguished stable clusters that remain long‑term from transient...
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...
Vertex Kidney Disease Drug Hits Mark in Late-Stage Study
Vertex Pharmaceuticals announced that its experimental IgA nephropathy drug povetacicept met primary and key secondary endpoints in a Phase 3 trial, cutting urine protein by roughly 50% versus placebo after 36 weeks. The interim results also showed reductions in abnormal antibodies...
CRISPR-Based Technique Unlocks Healing Power of Mitochondria for Heart Failure Therapy
Researchers at Rice University and Baylor College of Medicine used a non‑editing CRISPR system to activate the PPARGC1A gene, boosting mitochondrial production in human cardiomyocytes. The technique safely increased cellular energy output, as shown by higher oxygen consumption in cell...
SAB BIO Reports Full Year 2025 Financial Results and Business Highlights
SAB Biotherapeutics announced full‑year 2025 results, highlighting the launch of its registrational Phase 2b SAFEGUARD trial for SAB‑142 and the completion of a $175 million oversubscribed private placement. Phase 1 data demonstrated a favorable safety profile, no serum sickness and low immunogenicity across...
New Technology Unlocks More Autism Gene Varients
Researchers at UC San Diego used long‑read whole‑genome sequencing (LR‑WGS) on 267 autism families, uncovering 33% more structural variants and 38% more tandem repeats than short‑read methods. By pairing the genomic data with DNA‑methylation profiles, they could directly observe how...
APS BioGroup, Inc - 04/05/2018
The U.S. Food and Drug Administration issued a warning‑letter close‑out to APS BioGroup, Inc. on April 5, 2018, confirming that the company’s corrective actions addressing the July 2017 warning letter were satisfactory. The agency stressed that this closure does not relieve APS BioGroup...
James Findling - 598944 - 12/31/2019
The FDA issued a Warning Letter to Dr. James Findling after a 2019 inspection revealed serious protocol violations in a clinical trial of an investigational drug. The investigator randomized two subjects who had exceeded permitted dose levels and failed to...
Modular Cleanroom Supports Scale-Up of Cosmetic Microneedle Production
CipherX, a biotech start‑up specializing in microneedle platforms, partnered with Connect 2 Cleanrooms to install a 12 m² soft‑wall cleanroom for pilot production. The modular unit, validated to ISO 14644 class 7, was delivered and fully operational within 48 hours. Its PVC curtains, steel framing and...
An Emerging Longevity Supplement May Accelerate Cancer Growth, Scientists Say
Researchers at Tokyo University of Science discovered that polyamines, especially spermidine—a compound touted for longevity—can accelerate the growth of cancer cells. Laboratory experiments on cervical and breast cancer lines showed polyamines promote aerobic glycolysis and increase the oncogenic protein eIF5A2....
Unlocking Hidden Pocket on a Billion‑dollar Drug Target
Researchers led by Harvard chemist Christina Woo have mapped a previously unknown allosteric pocket on cereblon, the E3 ligase that underpins billions of dollars in cancer‑drug activity. The study shows that binding a small molecule to this hidden site can...
AbbVie’s Amylin Candidate ‘Competitive’ in Early-Stage Trial
AbbVie announced top‑line Phase 1 multiple ascending‑dose data for its amylin analog ABBV‑295, showing 7.75‑9.79% weight loss after 12 weeks of treatment. The long‑acting compound was administered every other week then monthly, with a favorable tolerability profile and no serious adverse...
AbbVie, Gubra Post Obesity Data; Regeneron Obesity Drug Succeeds in China
AbbVie and its partner Gubra released Phase 2 data on a long‑acting amylin analogue that produced significant weight loss in obese participants, with reductions approaching double‑digit percentages and a clean safety signal. The study highlighted dose‑responsive efficacy and tolerability, positioning the...
Why Simulating an Entire Cell Cycle Took Years, Multiple GPUs and Six Days per Run
University of Illinois researchers led by Zan Luthey‑Schulten have built a three‑dimensional kinetic model of the minimal bacterium JCVI‑syn3A that simulates an entire 105‑minute cell cycle. By assigning DNA replication to a dedicated GPU and running other cellular dynamics on...
GLP-1 Drugs Modulate Gene Expression via MED14 Phosphorylation
Stable GLP‑1 receptor agonists such as Exendin‑4 and Ozempic improve beta‑cell viability by modulating gene expression. Researchers at the Salk Institute discovered that these drugs induce phosphorylation of Med14, a core subunit of the Mediator transcription complex. Phosphorylated Med14 enables...
Regeneron’s Weight Loss Partner Hansoh Delivers Much-Needed Phase 3 Win in China
Regeneron’s Chinese partner Hansoh announced that its dual GLP‑1/GIPR agonist olatorepatide achieved a 19% mean weight loss in a Phase 3 trial of 604 obese or overweight adults, meeting both co‑primary endpoints. The study reported lower gastrointestinal adverse events compared with...
Bristol Myers Says Second CELMoD Succeeds in Phase 3
Bristol Myers Squibb announced that its oral CELMoD candidate mezigdomide met primary endpoints in the Phase 3 SUCCESSOR‑2 trial for relapsed or refractory multiple myeloma. The open‑label study showed a statistically significant improvement in progression‑free survival compared with the current standard...
Medidata, CRIO Boost Clinical Trials with Integration
Medidata, a Dassault Systèmes brand, has partnered with eSource specialist CRIO to automate clinical data flow from site systems into the Medidata Platform. The plug‑and‑play integration now serves over 2,500 research sites in roughly 30 countries, delivering near‑100% data accuracy and...
Carbios Under Pressure: Financing Struggles, China Expansion, and a Growing Legal Dispute
Carbios, once a flagship of France’s industrial biotech, has seen its enzymatic PET recycling ambitions hampered by financing gaps, a delayed Longlaville plant and a costly reorganisation. The company’s cash fell to €72 million by mid‑2025, prompting a 40 % cut in...
