Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds 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 funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
Psychedelic Retreat Boom Sparks Safety and Regulatory Alarm
A wave of psychedelic retreats in Jamaica, Peru, Oregon and Colorado is prompting health‑risk warnings and calls for tighter oversight. Researchers cite vague medical roles and mandatory medication washouts, while a new U.S. executive order aims to speed FDA approvals for psychedelic drugs.
FDA Grants Three Priority Vouchers to Psychedelics
The FDA has granted three Commissioner's National Priority Vouchers to psychedelic‑focused firms—Compass Pathways, Transcend Therapeutics and the Usona Institute—following President Trump’s executive order to accelerate mental‑health treatments. All three companies already hold Breakthrough Therapy Designations, and the vouchers promise faster...
ALONE-AF: Stopping OAC After Ablation Doesn’t Reduce Cognitive Function
A substudy of the ALONE‑AF trial found that stopping oral anticoagulation (OAC) after successful catheter ablation did not lead to cognitive decline over two years. Montreal Cognitive Assessment (MoCA) scores rose modestly in both the OAC and no‑OAC groups, with...
Rutgers‑Newark Engineers First Self‑Assembling RNA Nanostructures Inside Living Cells
Rutgers University–Newark researchers have created a self‑assembling RNA nanotechnology that can be programmed inside living human cells, a breakthrough published in Nature Communications and poised to reshape cancer treatment strategies. The interdisciplinary team, led by Professors Fei Zhang and Jean‑Pierre...
Tortugas Neuroscience Nabs $106M
Tortugas Neuroscience announced a $106 million seed and Series A financing round, one of the larger early‑stage biotech raises this year. The capital, led by Cure Ventures and co‑led by The Column Group and AN Venture Partners, will fund the completion of...
FDA Expands Tzield to Children as Young as One Year, Broadening Diabetes Intervention
The U.S. Food and Drug Administration has cleared teplizumab (Tzield) for use in children as young as one year, extending its disease‑modifying label beyond the previous eight‑year minimum. The decision follows the PETITE‑T1D phase‑4 trial and could shift early‑intervention standards...
The BioPharm Brief: Innovation, Gene Therapy Momentum, and Next-Gen ADCs Lead Today’s Headlines
The latest Ron Lanton Report shows biopharma innovation moving beyond Boston and San Francisco into emerging regions powered by academic collaborations, venture capital, and niche talent pools. The European Medicines Agency issued a positive opinion on Novartis’ intrathecal onasemnogene abeparvovec, expanding...
How Advances in Chronic Lymphocytic Leukemia Medications Are Shaping Patient Care Protocols
Over the past decade, chronic lymphocytic leukemia (CLL) treatment has shifted from broad chemotherapy to targeted, oral therapies such as BTK and BCL‑2 inhibitors, monoclonal antibodies, and personalized regimens based on genetic profiling. These drugs deliver higher response rates, longer...
The Slow March of Clinical Biomarkers to Become Surrogate Endpoints
Clinicians are urging the FDA to recognize more clinical biomarkers as surrogate endpoints, hoping to accelerate drug approvals and reduce trial durations. Regulators remain cautious, approving only six biomarkers through the 2007 Biomarker Qualification Programme and often demanding definitive clinical...
Alphamab Oncology to Present Phase 1 Data on Bispecific ADC JSKN016 in HER2-Negative Breast Cancer at ASCO 2026
Alphamab Oncology will unveil Phase 1 results for its bispecific antibody‑drug conjugate JSKN016 at ASCO 2026, focusing on HER2‑negative breast cancer. The first‑in‑human trial in China demonstrated antitumor activity and a manageable safety profile in heavily pretreated patients, including triple‑negative cases. JSKN016...
Sunnybrook Announces $41-Million Gift to Advance Canada’s Global Leadership in Clinical Trials
Sunnybrook Health Sciences Centre announced a $41 million CAD (≈$30 million USD) philanthropic gift to expand its Sunnybrook Clinical Trials program. The funding creates new leadership positions, boosts biobanking, data management and precision diagnostics, and supports first‑time investigators. By strengthening infrastructure and...
Scaling Cheap In‑Vivo Causal Testing for Age‑Related Diseases
AI has made hypothesis generation in bio cheap. Anyone can get an answer to ‘could this play a role in my disease’, but how do we go from ‘could’ to ‘does’? The scarce resource now is causal evidence to test hypotheses...
Semaglutide NAION Risk Higher than Risk with SGLT2 Inhibitors
A VA‑based study published in JAMA Ophthalmology found that patients with type 2 diabetes taking semaglutide experienced more than double the risk of nonarteritic anterior ischemic optic neuropathy (NAION) compared with those on SGLT2 inhibitors. The analysis covered 102,361 veterans, with...
Policy Watch: FDA Moves to Implement Trump Order on Psychedelic Drugs
President Trump’s recent executive order tasked the FDA and ARPA‑H with accelerating psychedelic research. In response, the FDA issued Commissioner’s National Priority Vouchers to three firms—Compass Pathways, Transcend Therapeutics and the Usona Institute—fast‑tracking review of psilocybin and methylone candidates for...
STAT+: FDA to Speed up Review of Three Psychedelics as Mental Health Treatments
The FDA announced it will grant priority‑review vouchers to accelerate the evaluation of three psychedelic therapies—Compass Pathways’ psilocybin for treatment‑resistant depression, Usona Institute’s psilocybin for major depressive disorder, and Transcend Therapeutics’ MDMA‑like compound for PTSD. The move is part of...
Protecting Innovation for Psychedelic Therapies Fast-Tracked Under New Executive Order
President Donald J. Trump signed an executive order that fast‑tracks FDA approval for breakthrough psychedelic therapies targeting serious mental illness. The order creates a Commissioner’s National Priority Voucher program, establishes pre‑approval patient access pathways, and earmarks $50 million in federal funding...
EU Biotech Act: The European Commission’s Landmark Proposal to Strengthen Biotechnology in Europe
On December 2025 the European Commission unveiled the European Biotech Act, a sweeping proposal aimed at cementing the EU’s position as a global biotech leader against the United States and China. The Act introduces fast‑track regulatory pathways, a strategic‑project framework...
Re: England Athletics Promotion of Medical Knee Procedure to Runners on Eve of London Marathon Is Condemned by Experts
England Athletics recently promoted Arthrosamid, a Class IIb implantable knee device, to runners ahead of the London Marathon. The product, marketed as a "drug," bypasses many safety tests because it carries a CE mark that allows manufacturer self‑certification. A UK...
FDA Awards Three Priority Review Vouchers For Psychedelics
On April 24, 2026 the FDA announced it will issue three priority review vouchers to companies developing psychedelic therapies. The vouchers cover two psilocybin programs targeting treatment‑resistant and major depressive disorder, and a methylone program for post‑traumatic stress disorder. The...
FDA Unveils Three Psychedelic-Focused Commissioner Vouchers
The U.S. Food and Drug Administration announced it has issued fast‑track “commissioner vouchers” to three psychedelic‑focused companies. The agency withheld the identities of the sponsors, a move that surprised investors and analysts. The vouchers are designed to expedite regulatory review,...
Pharmaceutical Executive Daily: FDA Issues CRL to AbbVie
The U.S. Food and Drug Administration issued a complete response letter to AbbVie, rejecting its biologics license application for trenibotulinumtoxinE due to manufacturing and CMC deficiencies. The setback delays AbbVie’s entry into the lucrative neurotoxin market, a segment dominated by...
AI-Designed Drugs by a DeepMind Spinoff Are Headed to Human Trials
Isomorphic Labs, the DeepMind spinoff behind AlphaFold, announced that its AI‑designed drug candidates will soon enter human clinical trials. The company’s new IsoDDE engine claims to double the accuracy of AlphaFold 3 in predicting protein‑small‑molecule interactions. Partnerships with Eli Lilly and Novartis...
Designer Baby Companies Are in Turmoil
Two high‑profile germline‑editing startups—Bootstrap Bio and Manhattan Genomics—have ceased operations within a year of launching. Bootstrap Bio folded after running out of capital and was further tarnished by the federal arrest of its chief science officer on child‑sex‑trafficking charges. Manhattan...
Building a Better Delivery System for Gene Editing Machines by Re-Engineering the Cellular Factory
A genome‑wide knockout screen conducted by the Whitehead Institute revealed specific producer‑cell genes that govern the assembly and potency of virus‑like particles (VLPs) used for gene‑editing delivery. Disabling a single brake gene dramatically increased guide‑RNA loading, boosting particle potency across...
Vertical AI Beats Generic Models in Enzyme Engineering
Foundation models are powerful. They're also generic. And in industrial enzyme engineering, generic doesn't cut it. #SynBioBeta2026 is May 4-7th in San Jose, California, you can learn more about the conference and get your tickets here: https://t.co/8abYWJ1GbK The industrial last mile of...
Biopharma Money Raised: Jan. 1-April 23, 2026
Regeneron’s Otarmeni, a gene‑therapy for congenital hearing loss, earned FDA accelerated approval and will be provided free of charge, marking a rare zero‑cost gene‑therapy launch. At AACR 2026, researchers highlighted breakthroughs in minimal residual disease (MRD) detection that sharpen relapse...
Cornell Study Shows Stem‑Cell Vesicles Can Halt Cellular Aging in Lab
Scientists at Cornell University's College of Veterinary Medicine demonstrated that extracellular vesicles derived from embryonic stem cells can completely halt cellular senescence in cultured cells. The breakthrough, published in the Journal of Biological Chemistry, could pave the way for next‑generation...
Postmarketing Requirements and Commitments: Reports
The FDA publishes annual reports in the Federal Register detailing the status of post‑marketing requirements (PMRs) and commitments (PMCs) for drugs and biologics, covering both CDER and CBER. These reports summarize data from internal databases and are updated quarterly on...
Trump's Executive Order Fast‑Tracks Psychedelic Therapy Review, Boosting Compass Pathways
President Donald Trump signed an executive order this weekend to shorten clinical‑trial and regulatory timelines for psychedelic medicines. The move shines a spotlight on Compass Pathways, whose late‑stage psilocybin candidate COMP360 is poised for an FDA filing and has $149.6 million...
TRT Gel/Injection Shows More Adverse Events Than Clomiphene
Interesting retrospective chart-based study in VA population - TRT gel/injection vs. clomiphene for hypogonadism. Matched populations at baseline. Far higher rates of AEs with gel/injection vs. clomiphene. "TRT was associated with an increased incidence of mortality, CVA, CAD, hypertension,...
Human Genetic Optimization Funding Claim Is Pure Fantasy
From Mother Jones story on polygenic selection and embryo editing plans. Absolutely zero chance that human genetic optimization companies raised $36.5 billion in 2024. This number is fantasy. https://t.co/we6euyIMXv
Glutamine Transport Boost Enhances CAR‑Macrophage Cancer Therapy in Mice
A Sun Yat‑sen University team engineered CAR‑macrophages to overexpress the glutamine transporter SLC38A2, dramatically improving phagocytosis and cytokine release against HER2‑positive breast cancer cells. In mouse models, the modified cells suppressed tumor growth far more than standard CAR‑macrophages, highlighting metabolic...
Readout LOUD Interview Explores Kelonia and Eli Lilly
Our Readout LOUD interview with @BRobertsVC about Kelonia and $LLY in print form. https://t.co/DsfGxwcvS3 via @statnews
ER-100 Cleared for Trials; Safety Promising, Translation Uncertain
So far, the data suggest ER-100 should be safe in humans, and the FDA has cleared us to move into clinical trials. Translation from primates to humans is never guaranteed If it doesn’t work as hoped, we’ll learn & improve, just...
WHO Prequalifies First-Ever Malaria Treatment for Newborns and Infants, Adds New Diagnostic Tests
The World Health Organization has prequalified the first antimalarial drug formulated specifically for newborns and infants weighing 2‑5 kg—artemether‑lumefantrine—enabling public‑sector procurement for an estimated 30 million babies born each year in malaria‑endemic Africa. The agency also prequalified three rapid diagnostic tests that...
Creative Biolabs Unveils LNP Conjugation Platform to Accelerate Precision Gene Therapy
Creative Biolabs announced the commercial release of an enhanced lipid‑nanoparticle (LNP) conjugation platform that improves payload stability and tissue targeting for gene‑editing and RNA therapeutics. The platform integrates microfluidic manufacturing and programmable ligand attachment, promising faster preclinical timelines for biotech...
AAN 2026: Tavapadon Post-Hoc Analysis Strengthens Its D1/D5 Agonist Pitch
At the 2026 American Academy of Neurology meeting, AbbVie unveiled post‑hoc results from its Phase III TEMPO‑1 and TEMPO‑2 trials of tavapadon, a once‑daily oral D1/D5 partial agonist for early Parkinson’s disease. The analysis showed statistically significant improvements in seven of...
GPT-5 Autonomously Outperforms SOTA Protein Synthesis by 40%
More on our work with @OpenAI where GPT-5 ran our autonomous lab at @Ginkgo and beat state of the art by 40% in cell free protein synthesis 👇
FDA Clears Single‑Trial Path for Cambium Bio’s Elate Ocular Gene Therapy
The U.S. Food and Drug Administration granted Cambium Bio a single‑trial approval route for its Elate Ocular gene therapy, allowing the CAMOMILE‑3 Phase 3 study to serve as the sole pivotal trial. The decision reduces clinical risk, accelerates market entry and...
AI in Oncology Takes Off, Tackling HIV and Liver Disease, Pharma’s Recent Gains
At AACR 2026 in San Diego, AI-powered platforms took center stage, signaling a rapid shift toward data‑driven oncology. Parallel research revealed CRISPR screens that mapped CD4+ T‑cell genes that either promote or block HIV infection, while synthetic‑biology engineers demonstrated implantable...
Lilly Slides as FDA Plans Psychedelic Voucher Program
Lilly tumbles on Foundayo’s shaky week; FDA to issue vouchers for psychedelics https://t.co/kWvYtFeif0 $LLY - 4% $NVO + 6% $REGN $AMGN $BBOT #biotech
AAN 2026: Head-to-Head Trial Shows Superiority of Qulipta for Migraine Prevention
AbbVie presented Phase IIIb TEMPLE trial data at AAN 2026, showing its oral gepant Qulipta outperforms topiramate in migraine prevention. Over 24 weeks, Qulipta had a 12.1% discontinuation rate versus 29.6% for topiramate and achieved a 73.7% responder rate (>50% reduction in...
Sanofi MS Drug Rejected in US Gets an Endorsement in Europe
Sanofi’s experimental multiple‑sclerosis drug tolebrutinib, marketed as Cenrifki, received a positive recommendation from the European Medicines Agency for secondary progressive MS, after the U.S. FDA rejected it earlier this year. The EMA’s endorsement paves the way for a final approval...
LogiPharma Europe: A New Model for Cold Chain Decision-Making
At LogiPharma Europe 2026, Roche’s global head of Distribution Technology, Raquel Vazquez, outlined a shift from experience‑based to data‑driven cold‑chain decision‑making. She highlighted the need to contextualize fragmented sensor data across lanes, products and environmental conditions to select the right packaging...
New Psychedelic Trials Target Depression and PTSD
Compass Pathways’ psilocybin (COMP360) for treatment-resistant depression (TRD) Usona Institute’s psilocybin for major depressive disorder (MDD) Otsuka’s methylone (TSND-201) for PTSD
Drug Digest: Examining the Architecture of Next-Gen Biotherapeutic Modalities
Minaris Advanced Therapies’ chief commercial and technology officer, Dr. Eytan Abraham, discussed how multifunctional biotherapeutics are merging antibodies, ADCs and engineered cells to create more precise, personalized treatments. He highlighted multi‑targeting cell designs that improve specificity and reduce disease escape,...
FDA Withholds Company Names Unlike Other CNPVs
why is the FDA not releasing the company names? They did for every other CNPV...
Biogen Seeks Darzalex Rivalry in China for Multiple Myeloma with Felzartamab Deal
Biogen has secured exclusive rights to TJ Biopharma’s anti‑CD38 antibody felzartamab in Greater China for up to $850 million, including control of a pending biologics licence application for relapsed and refractory multiple myeloma. The drug will be manufactured domestically at TJ...
Lantern Pharma to Debut Public Demonstration of withZeta.ai – A Platform to Conquer Rare Cancers on April 30 Following AACR...
Lantern Pharma announced a public, unscripted demonstration of its withZeta.ai AI co‑scientist platform on April 30, 2026. CEO Panna Sharma will lead two live sessions—morning and afternoon ET—to showcase real‑time drug‑discovery workflows for rare cancers. The event follows the platform’s...
Novartis Pulls Pluvicto Prostate Cancer Label Expansion Filing in the EU
Novartis has pulled its European Medicines Agency (EMA) application to extend the label of Pluvicto, its lutetium‑177 PSMA‑targeted radioligand therapy, into earlier‑stage prostate cancer. The drug already received approval for this indication in the United States and the United Kingdom,...