Agentic AI, Virtual Cell, LNP Vaccine Boosters, Engineered Organs, and Mergers
In this episode of Touching Base, the Gen editorial team discusses the latest advances in AI for life sciences, including NVIDIA’s GTC announcements on agentic AI, the deployment of 3,500 GPUs by Roche, and the emergence of open‑source autonomous agents like OpenClaw and NemoClaw. They dive deep into Zara Therapeutics’ new "Xcel" virtual‑cell model—a 4‑billion‑parameter AI trained on a 25.6‑million‑cell CRISPR perturbation dataset—highlighting its potential for predicting cellular responses and accelerating drug discovery. The conversation then shifts to lipid‑nanoparticle (LNP) innovations, featuring University of Pennsylvania researchers who engineered aromatic ionizable lipids (ARO LNPs) that preferentially target lymph nodes instead of the liver, promising more efficient mRNA therapies. Finally, the hosts cover a breakthrough in regenerative medicine: a lab‑grown, decellularized pig esophagus engineered with autologous cells that can replace a segment of a human esophagus without immunosuppression, illustrating the growing feasibility of engineered organs.
AI Tool Can Screen Unknown Bacteria for Disease-Linked Genes, Moving Closer to Preventing Pandemics
Researchers at Denmark's DTU unveiled PathogenFinder2, an AI system that screens unknown bacteria for disease‑linked genes using protein language models. Trained on over 21,000 genomes, the tool predicts pathogenic potential even for species with no known relatives and highlights the...
Nanoplastics Released by ‘Eco-Friendly’ Bioplastics May Slow Fetal Development in Mice
A study by Anhui Medical University and Fudan University shows that polylactic acid (PLA), a widely used biodegradable bioplastic, breaks down into oligomeric lactic acid (OLA) nanoplastics that cross the placental barrier in mice. Exposure to environmentally relevant OLA doses...
Association Between Apolipoprotein E Gene Polymorphisms and the Effects of High-Intensity Interval Training on Body Composition in University Students
A 12‑week high‑intensity interval training (HIIT) program was administered to 236 non‑athletic Han Chinese university students and genotyped for APOE variants. The promoter SNP rs405509 emerged as the sole polymorphism linked to body‑composition outcomes, with the GG genotype showing higher...
Pharmaceutical Executive Daily: Trump Administration Misses Deadline to Nominate Permanent CDC Director
The Trump administration failed to meet the 210‑day deadline to nominate a permanent CDC director, leaving the agency without confirmed leadership for most of the second term. Meanwhile, the FDA granted accelerated approval to Denali Therapeutics’ Avlayah, the first enzyme...
Antibodies Connect Cancer with Autoimmune Brain Disease
Researchers at Cold Spring Harbor Laboratory published a Nature paper showing that antibodies generated against NMDA‑receptor‑expressing tumors can both boost anti‑cancer immunity and trigger autoimmune encephalitis. In mouse models, strong anti‑NMDA antibody responses correlated with robust tumor control, yet the...
Pinnacle Medicines Raises $89M Series B
Pinnacle Medicines announced the closing of an oversubscribed $89 million Series B financing, co‑led by LAV and Foresite Capital. The biotech firm leverages a proprietary platform that blends physics‑based molecular simulations, AI‑driven design, and advanced peptide chemistry to create oral peptide therapeutics....
New AI Platform Uncovers Hidden Cancer Targets, Raises $10M
Most cancer drugs go after the same targets: EGFR. PD-L1. HER2. Not because they’re the best targets. Because they’re the only ones we’ve been able to see. RyboDyn Inc. is going after what’s been invisible. The San Diego team, led by Imad Ajjawi, PhD,...
The Natural “Biological Clock” Of Stroke Recovery
The ESPRESSO trial tested whether adding 90 minutes of high‑intensity hand and arm therapy each day for the first two weeks after stroke improves recovery. Sixty‑four participants received either immersive video‑game‑based or conventional therapy alongside standard care, but three‑month outcomes...
Biopharma Industry Pushes Back on FDA's 'America First' User Fee Proposals
The FDA’s upcoming user‑fee framework, dubbed "America First," seeks to tighten eligibility for the small‑business waiver, limiting it to U.S.‑based applicants. Industry groups argue the change politicizes fee policy and could disadvantage foreign‑owned biotech firms that rely on the waiver...
Pan-Cancer Variance Decomposition Nominates Translationally Actionable Therapeutic Antigen Candidates Across 33 Cancer Types
Researchers applied a genome‑wide variance decomposition across TCGA’s 60,656 genes and 33 cancer types to prioritize therapeutic antigens, moving beyond traditional mean‑expression screens. The analysis yielded 17 candidates that met functional dependency, safety, and immune‑cold criteria, with three highlighted: CRIPTO/TDGF1,...
Identification of Crucial Genes and Biological Pathways in Lung Adenocarcinoma by Network Pharmacology, Molecular Docking, and Simulation Studies
Researchers applied network pharmacology, molecular docking, and molecular dynamics to assess bioactive compounds from Pleurotus membranaceus for lung adenocarcinoma therapy. Four compounds passed drug‑likeness filters, linking to 226 overlapping disease targets and highlighting five hub genes. Docking showed isosorbide binding...
Phenylalanine-Associated Ocular Risk Stratification in Early-Treated Children with Phenylketonuria: A Cross-Sectional Study
A cross‑sectional study of 33 early‑treated PKU children found ocular abnormalities common, linked to higher serum phenylalanine. Comprehensive eye exams revealed anterior and posterior segment issues. ROC analysis defined phenylalanine thresholds that predict elevated ocular risk. Findings suggest metabolic control...
Valneva to Participate in Multiple Events at the 26th World Vaccine Congress in Washington DC
Valneva SE announced its participation in the 26th World Vaccine Congress in Washington, D.C., from March 31 to April 2, 2026. The company’s CEO, Thomas Lingelbach, and senior executives will present data on the chikungunya vaccine IXCHIQ® and join a...
Transgene to Deliver an Oral Presentation on Its Individualized Neoantigen Therapeutic Vaccine TG4050 at the World Vaccine Congress
Transgene (Euronext: TNG) will deliver a 30‑minute oral presentation on its individualized neoantigen therapeutic vaccine TG4050 at the World Vaccine Congress in Washington, D.C. on April 1, 2026. TG4050, built on the AI‑driven myvac® platform, targets patient‑specific tumor mutations. Phase 1 data in...
Editorial. Weighty Matter
The GLP‑1 drug semaglutide has entered the Indian market as generic versions after its patent expired last week, driving monthly prices down from roughly $144 to $36. The steep discount makes the medication affordable for a broader segment of diabetics...
UR Medicine Calls for Nutrition Counseling with GLP‑1 Weight‑Loss Drugs
The University of Rochester Medical Center’s Center for Community Health & Prevention is urging patients on GLP‑1 weight‑loss medications to enroll in nutrition counseling. The guidance highlights 80‑100 g of daily protein and adequate hydration to protect muscle mass and curb...
Study Finds Frequent Ejaculation Boosts Sperm Quality, Raising IVF Success by 10%
Researchers led by Dr Krish Sanghvi at Oxford analyzed 115 studies of 55,000 men and found that longer abstinence increases sperm DNA damage. Men who ejaculated less than 48 hours before IVF achieved a 46% pregnancy rate versus 36% for the standard...
Cellular Senescence and Senotherapeutics: The Expert Roundup
Cellular senescence has become a focal point for longevity medicine, prompting a surge of senolytic and senomorphic drug development. Pioneering studies showed that clearing senescent cells can extend healthspan, leading biotech firms like Rubedo, SENISCA, Deciduous Therapeutics, and Arda Therapeutics...
Magnetic Silk‑Iron Nanoparticles Offer Precise Steering of Drugs to Hard‑to‑Reach Disease Sites
A research team has created magnetic silk‑iron nanoparticles that can be steered with external magnetic fields to deliver therapeutics to otherwise inaccessible disease locations. The nanoplatform merges biocompatible silk fibroin with iron oxide, enabling magnetic control while maintaining safety, a...
WHO Issues New Guidance for Simpler Tuberculosis Tests
The World Health Organization announced new policy guidance aimed at simplifying tuberculosis detection tests, signaling a major push for more accessible diagnostics. While the guidance outlines a framework for low‑cost, point‑of‑care testing, specific technical details were not disclosed in the...
Verdesian Life Sciences’ Clare Doyle on Bio-Tech Solutions in 2026
Clare Doyle, chief scientist at Verdesian Life Sciences, outlined the company’s 2026 bio‑tech roadmap, emphasizing engineered nutrient‑delivery platforms and AI‑driven field management. She highlighted partnerships with agri‑tech startups to accelerate gene‑edited microbial solutions and noted upcoming regulatory filings for next‑generation...
High‑Dose Psilocybin Outperforms Nicotine Patches Sixfold
One large psilocybin dose beat nicotine patches by 6x odds for smoking cessation. 82 otherwise-healthy cigarette smokers, 42 received a single high-dose 30mg/70kg psilocybin session, and 40 initiated an 8- to 10-week course of nicotine patch treatment. At 6 months; participants were 6x...
Jona and Everlywell Partner to Launch AI-Powered Gut Microbiome Test
Jona, an AI‑driven gut microbiome startup, has teamed up with Everlywell to bring a high‑resolution microbiome test to the U.S. consumer market. The test employs deep shotgun metagenomic sequencing, delivering strain‑level data on bacteria, fungi, viruses and parasites. Jona’s proprietary...
Kodiak’s Phase 3 Eye Drug Success; Innate Discontinues Anti-CD20 Program
Kodiak Sciences announced that its experimental eye drug Zenkuda (tarcocimab tedromer) achieved positive topline results in the GLow2 Phase 3 trial for diabetic retinopathy, outperforming sham treatment. The study met its primary endpoint, delivering a statistically significant gain in visual acuity...
Researchers Gene-Edit the Bitterness Out of Grapefruit
Researchers at Israel's Volcani Center used CRISPR/Cas9 to inactivate the 1,2RhaT gene in Citrus paradisi, effectively halting production of bitter compounds such as naringin, neohesperidin, and poncirin in leaf tissue. The gene edit eliminates the bitter taste pathway, and the...
Frozen Does Not Mean Stable: Rethinking Cryopreservation in Cell and Gene Therapy Manufacturing
Cryopreservation remains a linchpin for cell and gene therapy (CGT) manufacturing, yet frozen material is not inherently stable. Real‑world operations introduce transient warming events (TWEs) when products are moved, accessed, or shipped, silently degrading viability and potency. Traditional reliance on...
PharmaShots CXO Talks | Women’s History Month Special: A Conversation with Dr. Emanuela Offidani of Tris Pharma
Dr. Emanuela Offidani, Tris Pharma’s Medical Director of Digital Health Strategy, highlighted that ADHD is increasingly recognized as a lifelong condition that often goes undiagnosed in women because symptoms are less overt. She explained that Tris Pharma’s proprietary LiquiXR delivery...
Eggs as Cheap, Scalable Factories for New Medicines
I have a story today about the quest to turn eggs into low-cost factories for medicines. Gift link: https://nyti.ms/4bzSabB
Pistoia Alliance Publishes First Best-Practice Framework for Ethical Social Media Use in Drug Development
The Pistoia Alliance has published a peer‑reviewed best‑practice framework guiding ethical use of social‑media listening in drug development. The framework, co‑authored by experts from Bayer, Roche, Boehringer Ingelheim, Chiesi and Semalytix, outlines standards for data anonymization, bias mitigation, and AI‑driven...
Corcept Therapeutics Shares Surge 20% on FDA Approval of Lifyorli
Corcept Therapeutics saw its shares climb almost 20% after the U.S. FDA approved its lead drug Lifyorli in combination with nab‑paclitaxel for platinum‑resistant ovarian, fallopian tube and primary peritoneal cancer. The approval, based on a 381‑patient trial, validates the company's...
Precigen Posts 149% Revenue Surge, Forecasts $18M Q1 as Papzimius Gains Momentum
Precigen (PGEN) reported fiscal Q4 2025 revenue of $9.7 million, up 149% year‑over‑year, and projected first‑quarter 2026 sales to exceed $18 million. The surge stems from the rapid commercial rollout of its FDA‑approved gene‑therapy Papzimius and broader payer coverage across the United...
How to Turn a Chicken Egg Into a Drug Factory
Biotech start‑up Neion Bio is pioneering a method to turn chicken embryos into miniature drug factories. Scientists micro‑inject genetic constructs into three‑day‑old embryos, reprogramming the developing bird to synthesize pharmaceutical compounds within the egg. The approach promises faster, cheaper production...
Novocure Reports Topline P-II (PANOVA-4) Trial Data on TTFields Therapy for Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC)
Novocure disclosed topline Phase‑2 PANOVA‑4 data showing that Tumor‑Treating Fields (TTFields) combined with Tecentriq and gemcitabine/nab‑paclitaxel achieved a 74.4% disease‑control rate in metastatic pancreatic ductal adenocarcinoma (mPDAC). The trial, enrolling 78 patients, outperformed the historical MPACT control arm’s 48% DCR. Secondary...
Lifespan Extension Doesn't Guarantee Morbidity Compression, Study Shows
“These findings challenge the assumption that lifespan extension necessarily compresses morbidity, highlighting the need to consider lifespan, healthspan, and CoM as endpoints when evaluating anti-aging interventions. We do not claim that life-extending interventions categorically fail to achieve CoM; rather, we demonstrate...
Vitamins B3 Plus B6 May Boost Muscle Repair After High Intensity Exercise: Nestlé Study
A Nestlé‑backed randomized trial gave healthy men 714 mg nicotinamide and 19 mg pyridoxine daily for nine days after intense eccentric exercise. The B‑vitamin combo boosted muscle stem cell numbers by 29% and accelerated differentiation markers—MyoD+ cells rose 67%, myogenin+ cells 34%,...
Tech Accelerates Clinical Trial Recruitment of Ideal Participants
I spoke to a startup building software to help clinical trials recruit more of the right kinds of people more quickly - a serious challenge at present.
Cells Store Inflammation Memory via Epigenetic Chromatin Domains
🆕 @ScienceMagazine How do our cells have long term memory of inflammation that can later lead to persistent, chronic inflammation and disease? Through specific epigenetic chromatin changes, aka "memory domains" @ScienceVisuals https://t.co/mt70OwH144 https://t.co/DMk7SaIuBc https://t.co/FaPpN0331q
Oral Peptides Biotech Pinnacle Medicines Gets $89M From US, China Investors
Pinnacle Medicines, a two‑year‑old biotech focused on oral peptide therapeutics, secured $89 million from a mix of U.S. and Chinese investors. The financing will fund its platform that converts injectable peptides into pill form, building on the recent success of Novo...
FDA Guidance Sparks Innovation Path for Inherited Disorders
My article in today's issue of the Journal of the American Medical Association Health Forum, on FDA's new rare disease guidance, and how the agency can build on these policy steps to promote innovation for inherited disorders, authored with Maarika...
FDA Approval Propels Denali, Boosts Rare Disease Biotech
FDA clears Denali drug in ‘clear step’ for rare disease biotechs https://t.co/x9yL4nyPaj by Kristin Jensen $DNLI #biotech
IPHYF Falls Short of Promise, Future Uncertain
Of all the IO companies I've met and covered in the past ~15 years I'm struggling to think of one that's been more disappointing vs its initial promise than $IPHYF. Will it even last beyond Q3? Via @ByMadeleineA -> https://t.co/DepdZfDBdY
Allogene's Interim Data Hints at Off‑Shelf CAR‑T Breakthrough
This week's Biotech Scorecard: Allogene $ALLO: A preview of its April interim analysis. Very interesting (to me, at least) There just might be a path forward for off-the-shelf CAR-T therapy in B-cell lymphoma. Frontline consolidation. Read all about it: https://t.co/tcPFelUH3C
Gene Therapy Turns Aging Into Editable Code
Gene therapy isn't just targeting diseases anymore—it's targeting aging itself. The hallmarks of aging are becoming editable code. Longevity Escape Velocity isn't a fantasy. It's an engineering problem. And we're solving it.
Harvard's Light Chip Enables Sorting Mirror-Image Molecules
Harvard’s twistable light chip could give drug developers a new tool for sorting mirror-image molecules https://t.co/WkjjD38exo https://t.co/5kr4d9f6sa
Code Ran Fine, but Biology Flagged Label Error
I almost sent a figure with wrong cluster labels to my collaborators. The R code ran perfectly. No errors, no warnings. I caught it because cluster 2 showed upregulated genes in RNAseq but reduced chromatin accessibility in ATACseq. Biologically, that's backwards....
BiotechTV Live Interviews at MassBio State of Possible
Pretty sweet setup for BiotechTv interviews at @MassBio State of Possible Conference today. If you are here, please stop by and say hello.👋 https://t.co/CQtXLi36Qk