Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech financings
Spanish biotech Ona announced a $86.6 million Series B round, ranking among the largest venture financings in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funds will expand Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical pipeline.
Deaminet 2026: Breakthroughs in Base Editing, Deaminase Biology, and Therapeutic Translation
Deaminet 2026 showcased accelerating advances in base and prime editing, highlighted by rapid Addgene distribution growth and new mechanistic insights such as PE6d’s heightened processivity and ABE8e’s dimer‑driven efficiency. Novel off‑target detection platforms like beCasKAS and Inrich‑seq revealed far more unintended edits, underscoring safety challenges as therapeutic programs move forward. Translational breakthroughs included the disease‑agnostic PERT suppressor‑tRNA strategy, immune‑evasive oDNA donors, and Prime Medicine’s PM577a for Wilson’s disease, while RNA‑editing platforms (AIMers, adaptamers, editopes) entered clinical trials. The meeting emphasized the dual role of deaminases in cancer biology and the expanding toolkit driving faster bench‑to‑bedside pipelines.
BioNTech Founders Launch New Biotech, Eye FDA Acceptance
Sticking with the science. I find that really impressive. BioNTech founders Uğur Şahin and Özlem Türeci are leaving the biotech they founded to start a new one. Maybe by the time they have something for the clinic, the FDA will...
GSK Secures Approval Yet Refuses to Produce Drug
$GSK is ostensibly the "sponsor" that won the approval but is once again saying it won't produce the drug.
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...
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...
The Reputational Risk Hidden Inside Drug Pricing
Drug pricing is increasingly viewed as a reputation issue, not just a financial decision. Carreen Winters of MikeWorldWide argues that pricing choices shape public perception of a company's values, as illustrated by Novo Nordisk’s 50% cut to GLP‑1 drug prices....
Pharma Pulse: Ipsen’s Global Tazemetostat Withdrawal and FDA Approval of Deucravacitinib for Psoriatic Arthritis
French biotech Ipsen announced an immediate, global voluntary withdrawal of its oncology drug tazemetostat (Tazverik) after safety signals in the SYMPHONY‑1 confirmatory trial indicated a rise in secondary hematologic malignancies. The company is coordinating with the FDA to manage the...
India's Exports of Active Pharma Ingredients at Rs 41,500 Cr Surpassed Imports in FY25
India’s active pharmaceutical ingredient (API) market posted a landmark trade balance in FY25, with exports climbing to roughly Rs 41.5 billion while imports lagged at Rs 39.2 billion. The surplus marks the first time the sector has out‑exported its imports, signaling a maturing domestic...
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...
How FDA's Removal of the Two-Trial Requirement Affects Development Programs
The FDA has eliminated the longstanding requirement for two pivotal clinical trials, adopting a single‑trial default to speed drug approvals. The agency pairs this change with a mandate for more rigorous post‑market surveillance to catch safety signals after launch. Pharmaceutical...
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...
Researchers Take a Step Closer to Finding a Treatment for a Rare Genetic Neurodevelopmental Condition
Researchers at Texas Children’s Duncan Neurological Research Institute and Baylor College of Medicine demonstrated that skipping exon e2 of the MECP2 gene boosts MeCP2 protein production by 50‑60%, rescuing neuronal function in Rett syndrome mouse models and patient‑derived cells. The study...
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...
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...
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...
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....
PCSK9 Drives Vascular Aging, Offers New Therapeutic Targets
PCSK9 in Vascular Aging and Age-Related Diseases Comprehensive summary of PCSK9's regulatory functions in vascular aging, highlighting potential therapeutic targets for combating age-related cardiovascular diseases. https://t.co/uo3PaSJLCb https://t.co/w8UZgIduPV
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...
ADC Therapeutics SA (ADCT) Q4 2025 Earnings Call Transcript
ADC Therapeutics reported Q4 2025 product revenue of $15.8 million, down from $18 million a year earlier, while GAAP net loss narrowed to $41 million ($0.30 per share). Non‑GAAP operating expenses fell 12.1% to $45 million, driven by lower R&D spend. A $60 million private...
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...
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...
Senate Dems Ask How Trump’s MFN Deals Mesh With GENEROUS Model
Senate Democrats have pressed major pharmaceutical companies to clarify the secretive most‑favored‑nation (MFN) pricing agreements they struck with the Trump administration in December. Lawmakers are questioning how those deals align with the Centers for Medicare & Medicaid Services’ new GENEROUS...
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...
AI Predicts Asymmetric Cross‑coupling, Slashing Lab Time and Cost
A new AI-driven workflow predicts the outcomes of asymmetric cross-coupling reactions, enabling chemists to efficiently identify optimal molecular structures while significantly reducing laboratory time and costs. drugdiscovery
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...
Accrufer Becomes First FDA-Approved Prescription Iron Therapy for Children 10+
Shield Therapeutics announced that the FDA has approved Accrufer, its ferric maltol oral iron formulation, for children ages 10 and older, making it the first prescription oral iron therapy for this age group. The approval expands the drug’s label beyond...
10 Drugs Expected to Lead US Sales in 2026
Statista projects that the U.S. pharmaceutical market will be led by ten blockbuster drugs in 2026, with Merck’s Keytruda topping the list at $12.7 billion in sales. Four oncology therapies—Keytruda, Opdivo, Imbruvica and Ibrance—are among the top ten, underscoring cancer’s continued...
De‑risking Synthetic Biology: From Lab to Market
What does it actually take to bring a biological product to market? For more than 20 years, Ingenza Ltd has helped teams avoid the most common failure points in synthetic biology. Freedom to operate issues. Low productivity. Fragile processes. Late CMC...
IPV Doesn’t Block Infection, but Stops Paralytic Polio
Actually he has it a bit backwards, those vaccinated with IPV can still acquire poliovirus and shed virus, but it prevents the virus from entering the bloodstream + protects almost 100% paralytic polio, just like the Covid vaccine protects 80-90%...
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...
Vertex Drug Cuts Kidney Disease Marker in Late-Stage Trial
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
Bristol Myers Reports Positive Results for Next‑Gen Blood Cancer Drug
Bristol Myers claims success in study of another next-gen blood cancer drug https://t.co/fqPbZ2Szv8 by @gwendolynawu $BMY
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...
FDA Drugs Show Promise as Future Geromedicines
Check out this article I was apart of @MensHealthMag where I discuss currently approved FDA drugs that are potential geromedicines and our work on centenarians. https://t.co/e95XvnY9fb
Health Pharma USA LLC - 588155 - 12/18/2019
The FDA issued Warning Letter #588155 to Health Pharma USA LLC after a May‑June 2019 inspection uncovered multiple CGMP violations at its Rahway, New Jersey facility. Major deficiencies included a dysfunctional quality‑control unit that released products before review, incomplete batch...
FDA Draft Guidance Streamlines Low‑Cost Biosimilar Approval
New FDA draft guidance outlines important streamlining of path to getting low-cost biosimilars to the market - could reduce cost of pharmacokinetic studies and allow ex-U.S. comparitor products to be used for proving biosimilarity to U.S.-licensed drugs https://t.co/ErjNOga0qo
Pharmaceutical Executive Daily: Vinay Prasad Set to Leave CBER for Second Time
Vinay Prasad has left the FDA’s Center for Biologics Evaluation and Research (CBER) for the second time in under a year, signaling a notable leadership turnover at a key regulatory hub for biologics, vaccines and gene therapies. In parallel, GlaxoSmithKline...