Know What's Happening in BioTech
Know What's Happening in BioTech
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EMAIL DIGESTS
Daily
Every morning
Weekly
Tuesday recap
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 practices already accepted in rare‑disease and cell‑and‑gene‑therapy approvals. Experts stress early engagement with agencies, comprehensive modeling, and adherence to the new ICH E6(R3) risk‑based quality guidelines. Artificial intelligence and real‑world evidence are being positioned as critical tools to meet these stricter evidentiary standards.
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...
I used the extracted gDNA from tonight's prep to try and PCR a larger chunk of petunia genome hunting for one of its ubiquitin genes. I'm using a published genome as reference but its not my cultivar so need to...
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...
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...
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,...
In this episode, Professor David Fernández‑Rivas discusses the role of engineers as problem‑solvers and the necessity of interdisciplinary collaboration, especially between physics, chemistry, and bioengineering. He explains microfluidics—manipulating fluids at the micrometer scale—and its parallels with microelectronics, then delves into...
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...
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...
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....
Researchers at the Chinese Academy of Sciences unveiled a ferritin‑based biomimetic platform called FACE that links CD71 on CAR T cells to CD71 on leukemia cells, strengthening cell‑cell engagement without re‑engineering the CAR. In patient‑derived xenograft models, FACE‑augmented CAR T...
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...
Intergenerational Transmission of Metabolic Changes in Oocytes From Aged Mice 👉 “ Our findings indicate a remodelling of lipid homeostasis in oocytes of female mice derived from AMA great-grandmothers and highlight the need to take a closer look at the inheritance...
Back at peaceful campus of VISTEC 🇹🇭 all this week for their SynBio/Biocatalysis symposium with speakers from around the world. Nice to escape the heat and hustle of Bangkok. https://t.co/s9ZRM3uyDu
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...
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
Genetically modified crops are the dog that never barks in China. People are wary and there's very little of it outside animal feed. The next FYP is ambiguous. https://t.co/ixkR3lGCCI
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...
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...
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...
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...
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...
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 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...
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 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...
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 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...
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...
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...
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...
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...
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...
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...
A much needed effort that @Danbelsky and I have now been funded for to support the FAST initiative ($8 million over 18 months): https://t.co/aZsaZuEpoo The goal of this is to provide a set of biomarkers that will tell a person if...
I've covered most of the big controversies the FDA has faced over the past 25 years: Vioxx, drug-coated stents, and Aduhelm all spring to mind. In all of these cases, the FDA's advisory panel system, in which the agency calls together...
Taisho Pharmaceutical’s vornorexant (TS‑142), marketed as Vorzzz®, received Japanese regulatory approval in August 2025 as a dual orexin‑1/2 receptor antagonist for insomnia. The drug distinguishes itself from existing DORAs through rapid absorption and a short elimination half‑life, aiming to minimize...
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....
Would you like to hear an opinion on how the future of drug discovery will look like as foundation models become more capable in drug discovery tasks? Check out our dialogue and the first pilot with the foundation model genius @ramin_m_h...
OpenAI ran 86,000 experiments in 2 days. AI is talking to robotic labs in a tight loop. Result: a protein synthesized at 40% lower cost. This is the new scientific method.
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...
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%...
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
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...
Bristol Myers claims success in study of another next-gen blood cancer drug https://t.co/fqPbZ2Szv8 by @gwendolynawu $BMY
Exciting & important new study from my lab. Have you ever wondered how strong that alleged connection is between psychedelic use and #Schizophrenia? This project aims to point the telescope there & give it a focused look. Please share widely. @AlexaM_Molinaro...
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
Why is it vital for humanity to fight aging? Why are common longevity worries not big problems? My new review: https://t.co/tIcXYBFE7U a preprint of a book chapter for the upcoming radical longevity book that many in the field have contributed to. https://t.co/NFTYSMALzD
UniQure leads genetic medicine biotech rally after news of Prasad’s exit https://t.co/zu1CtJfj29 by @realJacobBell $QURE $RGNX $ATRA $LXEO #GeneTherapy
Just published @NatureMedicine A daily multivitamin (MVM) slowed epigenetic aging in a randomized trial after 2 years; effect was small (~2 months) and not seen with cocoa extract supplement (vitamin was Centrum Silver) https://t.co/snOMNsTzW7 https://t.co/a6MhMuRhJb
