FDA Grants Seventh Approval Under the National Priority Voucher Pilot Program
The FDA approved Bizengri (zenocutuzumab‑zbco) for adults with advanced, unresectable or metastatic cholangiocarcinoma harboring NRG1 gene fusions, marking the seventh clearance under the Commissioner’s National Priority Voucher (CNPV) pilot. The approval follows a single‑arm trial of 19 patients that showed a 36.8% overall response, with responses lasting up to 12.9 months. Bizengri received Breakthrough Therapy and Orphan Drug designations, and the FDA scheduled a public meeting on June 4 to discuss future CNPV eligibility and processes.
About CDER Small Business and Industry Assistance (SBIA)
The FDA’s Center for Drug Evaluation and Research (CDER) Small Business and Industry Assistance (SBIA) serves as a primary contact point for small pharmaceutical firms seeking regulatory guidance. SBIA provides direct phone and email support, a dedicated web portal, and...
Pharmaceutical Executive Daily: FDA Extends Review of Leqembi's sBLA
The FDA has extended the priority review of Eli Lilly's subcutaneous Leqembi by three months, delaying the agency's action date while requesting additional information. The once‑weekly injection is designed to replace the current infusion regimen for patients with early Alzheimer’s...
Electromagnetic Field Activation of Gene Therapy as an Approach to Reprogramming
Researchers have engineered an electromagnetic‑field (EMF)‑responsive DNA element that remotely activates partial cellular reprogramming genes in mice. By cycling EMF exposure, the system triggers the Oct4‑Sox2‑Klf4 cassette without permanent gene integration, extending median lifespan to 108 weeks—about 70 human years....
Harbour BioMed Gains FDA Clearance for First-in-Human Study of B7H4xCD3 Bispecific Antibody HBM7004
Harbour BioMed announced FDA IND clearance to launch a Phase I first‑in‑human study of its bispecific antibody HBM7004, which targets B7H4 and CD3 in advanced solid tumors. The trial will assess safety, tolerability, pharmacokinetics and early anti‑tumor activity across multiple cancer...
Daiichi Sankyo Posts 'Extraordinary Loss' Of Nearly $1B
Daiichi Sankyo announced it is scrapping its planned antibody‑drug‑conjugate (ADC) manufacturing line, a move that triggered an extraordinary loss of 149.4 billion Japanese yen (approximately $950 million). The loss reflects a write‑down of capital expenditures and R&D investments tied to the abandoned...
How Digital Orchestration Is Redefining Regulatory Infrastructure for Cell and Gene Therapy
The surge in cell and gene therapies is exposing the limits of legacy batch‑centric biopharma systems, which struggle to maintain chain‑of‑identity and chain‑of‑custody for patient‑specific products. SAP’s Cell and Gene Therapy Orchestration (CGTO) platform redesigns digital infrastructure to embed compliance...
New Kind of Liver Cell May Protect Against Common Liver Disease
Researchers at the University of Michigan identified a previously unknown hepatocyte subpopulation that emerges only in metabolic dysfunction‑associated steatohepatitis (MASH) livers. The new cells exhibit high expression of the immune‑related gene THEMIS, which regulates cellular senescence. Mouse experiments showed that...
Biosimilar Ramp-Up to Drive Growth for Biocon in FY27
Biocon is pivoting from a heavy‑investment phase to a profit‑driven model, banking on new biosimilar launches and scale‑up of recent products to fuel FY27 growth. Biosimilars already accounted for 60% of FY26 revenue, delivering 16% year‑on‑year top‑line growth and a...
Primary Cilium Shapes the Developing Brain
A new study published in Cell Reports shows the primary cilium in neural progenitor cells contains over 1,000 proteins, including ribosomal machinery, indicating on‑site protein synthesis. Regional specialization was observed, with more than 40 proteins varying by brain region. Loss...
Some Gene Therapies No Longer Require Clinical Trials, Thanks to New FDA Rule. Is This Safe, and Who Will It...
The FDA has introduced a "plausible mechanism pathway" that lets developers market experimental gene‑editing therapies for rare, monogenic disorders without completing traditional large‑scale clinical trials. The rule relies on prior safety data for the delivery platform and permits customization of...
Can Existing Flu Shots Help Protect Against Bird Flu?
Researchers from National Taiwan University and the University of South Florida analyzed 35 ferret studies spanning two decades and found that seasonal influenza vaccines containing the neuraminidase N1 protein reduced H5N1‑related mortality by roughly 73%. By contrast, vaccines without N1...
Karolinska Study Finds Daily Peanut Exposure Safely Treats 82% of Toddler Allergies
Researchers at Karolinska Institutet reported that 82% of toddlers who received daily oral peanut immunotherapy could safely eat three and a half peanuts after three years, compared with just 12% in a control group. The three‑year study of 75 children...
Safety Debate Heats Up Over Stem Cell Longevity Treatments
Leading researchers and clinic founders are confronting the safety of stem‑cell therapies marketed for longevity, with experts warning that not all products are genuine stem cells and that regulatory oversight remains limited. The debate underscores a booming market driven by...
FDA Sets 2027 PDUFA Date for Taletrectinib in ROS1‑Positive Lung Cancer
The FDA has accepted Nuvation Bio’s supplemental new drug application for taletrectinib in ROS1‑positive non‑small cell lung cancer and scheduled a PDUFA decision for Jan. 4, 2027. The filing adds 10 months of phase‑2 data showing high response rates and durable disease...
Junyue Cao on How the Body Ages, Cell by Cell
Dr. Junyue Cao’s lab at Rockefeller University released the most extensive single‑cell epigenomic atlas of mammalian aging, profiling chromatin accessibility in roughly seven million cells from 21 mouse tissues at three life stages. The study identified about 1,800 distinct cell...
Baylor Study Shows Brain Processes Words Under General Anesthesia
Researchers at Baylor College of Medicine discovered that patients under general anesthesia continue to process spoken words, a finding published in Nature that could reshape anesthesia monitoring and consciousness research.
Remembering J. Craig Venter, PhD
J. Craig Venter, the pioneering genome scientist and biotech entrepreneur, died at 79 after a cancer diagnosis. He co‑led the private effort that rivaled the Human Genome Project, delivering a draft human genome in the late 1990s. Venter’s later work on...
Bayer Announces Positive Data for Investigational PET Radiotracer
On May 7, 2026 Bayer announced that its investigational PET radiotracer I‑124 evuzamitide achieved positive topline results in the Phase III REVEAL study, meeting primary sensitivity and specificity endpoints for diagnosing cardiac amyloidosis. The multicenter trial evaluated 170 adults at 19 U.S....
A Monocyte‐Targeted Nanoplatform for Phagocytosis Activation and Ferroptosis Inhibition in Intracerebral Hemorrhage
Researchers have engineered a monocyte‑targeted nanoplatform (mPDA@DFO‑CpG‑N1) to accelerate hematoma clearance after intracerebral hemorrhage (ICH). The system combines a high‑affinity aptamer for selective monocyte delivery, a TLR9 agonist that overrides CD47‑SIRPα inhibition, and the iron chelator deferoxamine to block ferroptosis....
FDA Delays Leqembi Decision; Artiva Raises $300M; Pharvaris Prices Offering
The FDA has postponed its decision on the subcutaneous formulation of Leqembi, moving the target date to August 24, delaying a potential launch for Eisai and Biogen’s early‑Alzheimer’s therapy. The shift adds uncertainty to the Alzheimer market, where Leqembi could become...
J. Craig Venter: The American Scientist Who Changed Biotech
J. Craig Venter reshaped biotech by launching Celera Genomics, which used shotgun sequencing to finish the human genome in two years, outpacing the $3 billion public Human Genome Project. His 2000 IPO raised $1 billion, cementing a new era of private‑sector competition...
Kanvas Biosciences Receives New Funding to Develop First-In-Category Microbiome Therapeutic for Improving Maternal, Newborn and Child Health
Kanvas Biosciences announced a new investment from the Gates Foundation to develop the world’s first fully synthetic microbiome therapeutic targeting maternal environmental enteric dysfunction (EED). The product will use a complex, multi‑strain consortium designed with AI‑driven discovery and spatial microbiome...
AI-Guided Labs Are Approaching Full Autonomy
Ginkgo Bioworks is building fully autonomous biopharma labs that combine 70 robots, 90 lab devices and AI‑driven software in an 18,000‑square‑foot Boston facility. Scientists can submit dozens of unique protocols each day, which the system executes without human intervention. In...
NEJM Publishes Moderna Flu Data After FDA Refusal
A Friday footnote: The New England Journal of Medicine has published the Moderna influenza vaccine data -- the same that led the FDA's CBER division to refuse to review the vaccine under Vinay Prasad. (That decision, called a refuse-to-file or...
Vistagen Announces Completion of Last Patient Visit in PALISADE-4 Phase 3 Public Speaking Challenge Study of Fasedienol for Acute Treatment...
Vistagen announced that the final participant has finished the randomized, double‑blind portion of its Phase 3 PALISADE‑4 trial evaluating fasedienol nasal spray for acute social anxiety disorder. The company expects to release topline efficacy and safety data in the second quarter...
Etripamil
Etripamil (Cardamyst®) received FDA approval as a rapid‑acting, intranasal L‑type calcium channel blocker for converting acute paroxysmal supraventricular tachycardia (PSVT) episodes to sinus rhythm in adults. The drug leverages an ester‑sensitive phenylalkylamine scaffold to achieve fast onset and a short...
ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing
ParcelBio announced a $13 million seed round led by Breyer Capital, with participation from General Catalyst, Y Combinator and other investors. The funding will accelerate its proprietary Amplified and Prolonged Expression mRNA (APEXm™) platform, which claims to deliver markedly higher and longer‑lasting...
Psilocybin Shows Promise After Decades of Failed Cocaine Treatments
For 50 years the National Institute on Drug Abuse has spent hundreds of millions of dollars to find an effective cocaine addiction medication. After 100+ molecules & many hundreds of studies nothing has been FDA approved. It's the Holy Grail...
Rapamycin's 50-Year Odyssey: From Easter Island to Medicine
The rapamycin sTORy: 50-year journey from Easter Island to the frontiers of biology and medicine https://t.co/Jn67QrGVi6 https://t.co/dwGPgUVB83
Merck Advances Scalable Manufacturing for Oral PCSK9 Therapy
Merck has published a landmark study in Science describing a scalable biocatalytic synthesis platform for its investigational oral PCSK9 inhibitor, enlicitide decanoate. The enzyme‑driven process enables selective peptide fragment formation, coupling, macrocyclization and uses crystallization‑based purification, aiming to reduce reliance...
ZYME ADC
Catching up with $ZYME pan-RAS inhibitor payload ADCs from #AACR26. Seems the setting for each one is tumours that are RAS-mutated as well as expressing the target antigen (PTK7, Ly6E or Claudin18.2). https://t.co/hljX5soZQi
PTCT Jumps 17% on Strong Drug Uptake
$PTCT up 17% on results, really liked that one after writing up this week, nice value with strong uptake in its new drug, and those Jan. calls kept accumulating https://t.co/HEe8G2Sjdx
Bristol Myers Squibb Receives European Commission Approval of Sotyktu (Deucravacitinib) for the Treatment of Active Psoriatic Arthritis in Adults
Bristol Myers Squibb announced that the European Commission has approved Sotyktu (deucravacitinib) for adults with active psoriatic arthritis, either as monotherapy or with methotrexate. The approval follows positive Phase 3 POETYK PsA‑1 and POETYK PsA‑2 trials, which demonstrated statistically significant improvements in ACR20...
Organ‑Specific Aging Clocks Reveal Multi‑Omics Lifespan Patterns
The big advance in the science of human aging is the ability to quantify it and relate the metrics to health and disease. A new paper today @CellCellPress takes this to the next level with organ clocks and multiple biologic...
Industrial Microbiome: Affordable Year-Long Service for $75
Industrial microbiome products are a slept-on sector of the bioeconomy. A year supply for $75. Microbes as a Service. https://t.co/aWyLZ00qMM
Angelini Pharma to Acquire Catalyst Pharmaceuticals for $4.1 Billion
Italian drugmaker Angelini Pharma agreed to purchase Florida‑based Catalyst Pharmaceuticals for roughly $4.1 billion in cash. The transaction values Catalyst at $31.50 a share, a 28% premium, and gives Angelini a foothold in the U.S. rare‑disease market and neurology segment.
Metformin's Primary Action Is Gut, Not Liver
Metformin, one of the most commonly prescribed drugs, was thought to work via the liver. Check that. It's primarily through the gut. @NatMetabolism https://t.co/i2CpZip61B https://t.co/bhsI7p7SYx
Check Gene A–B Co‑Expression in Your Single‑Cell Data
1/ You have a clear question: Is gene A and gene B co-expressed in my cell type of interest? You feel ready. You have single-cell data. https://t.co/9ET61foUET
STAT+: Pharmalittle: We’re Reading About an FDA Gender Rule Change, Its Program for One-Day Inspections, and More
The U.S. Food and Drug Administration announced a proposed rule to strip references to “gender” from its regulations, aligning with a 2025 Trump administration executive order that stresses biological sex. While the agency claims the change won’t affect industry practice,...
Capricor Sues Partner Nippon Shinyaku in DMD Therapy Spat
Capricor Therapeutics has filed a lawsuit against its Japanese partner Nippon Shinyaku and its U.S. subsidiary NS Pharma, alleging a fundamental pricing flaw in their distribution agreement for the Duchenne muscular dystrophy (DMD) cell therapy deramiocel. The biotech claims the...
Biologists Leveraging AI Will Outpace Those Who Don't
1/ AI won't replace you. But a biologist using AI will. Especially in bioinformatics, where the questions never stop coming. https://t.co/VYCu5ukfCT
ASGCT Honors Mohamed Abou‑el‑Enein as Outstanding New Investigator
Mohamed Abou‑el‑Enein, MD, PhD, received the American Society of Gene and Cell Therapy’s 2026 Outstanding New Investigator Award and his lab earned the Best of Molecular Therapy Award. His team’s high‑dimensional spectral flow cytometry platform maps CAR‑T cell states, pinpointing...
Clostridioides Difficile Infection: Developing Drugs for Treatment, Reduction of Recurrence, and Prevention
The U.S. Food and Drug Administration has issued a final Level 1 guidance titled “Clostridioides difficile Infection: Developing Drugs for Treatment, Reduction of Recurrence, or Prevention.” The document replaces the October 2022 draft and provides detailed recommendations for clinical trial design, endpoints,...
GLP‑1 Breakthrough Costs $500/Month, Limiting Access
After catching my breath (unusual for GLP-1 story to discuss their costs approvingly), I suspect both that @continetti (not unusually) is likely directionally right (& I share enthusiasm for free market) - but - at $500/mo for transformative drug -...
Pulmonary Tuberculosis: Developing Drugs for Treatment
The FDA has issued a final Level 1 guidance titled “Pulmonary Tuberculosis: Developing Drugs for Treatment,” replacing the draft released in December 2022. The guidance, docket FDA‑2013‑D‑1319, outlines clinical development expectations for new antibacterial agents targeting pulmonary TB. It provides sponsors with detailed...
Postapproval Pregnancy Safety Studies
The FDA has released its final Level 1 guidance on post‑approval pregnancy safety studies, updating the 2019 draft to provide detailed recommendations for designing investigations of drug and biologic exposure in pregnant women. The guidance outlines methodological standards, data sources, and...
Mobia Medical Prices $150 Million IPO to Fund Stroke‑Device Platform
Mobia Medical, Inc. priced a 10 million‑share public offering at $15 per share, raising roughly $150 million. The Nasdaq‑listed ticker “MOBI” will debut on May 8, giving the stroke‑device firm capital to accelerate product development and market rollout.
Why some Brain Cells Are Particularly Vulnerable to Multiple Sclerosis
Researchers identified that CUX2 cortical neurons, essential for higher cognition, are uniquely vulnerable in progressive multiple sclerosis due to accumulated DNA damage. The protein ATF4 initiates a DNA‑repair kit that safeguards these cells; disabling ATF4 in mice triggers rapid CUX2...
What Is PDUFA—And Why Does It Matter for Biotech Innovators, FDA & Patients?
Congress will reauthorize the Prescription Drug User Fee Act (PDUFA) next year, renewing the FDA’s authority to collect fees from biotech firms for drug reviews. The user‑fee model supplies a stable budget that underpins faster, more predictable approval timelines—about ten...