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Every morning
Weekly
Tuesday recap
![[Review] Autosomal Dominant Polycystic Kidney Disease](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/e5c772f073be33a83118f46a1ac91f13.webp)
Autosomal dominant polycystic kidney disease (ADPKD) remains the most common hereditary cause of chronic kidney disease, imposing substantial morbidity, mortality, and healthcare costs worldwide. Recent advances in molecular genetics and high‑resolution imaging have sharpened diagnostic criteria and enabled more accurate risk stratification. Robust trial data now support early use of the vasopressin‑V2 antagonist tolvaptan in patients at high risk of rapid renal decline. Comprehensive care pathways that integrate screening for cyst infections, intracranial aneurysms, and polycystic liver disease are essential for optimal patient outcomes.
![[Comment] Safeguarding Genomic Integrity in Pluripotent Stem-Cell Therapies](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/fba52b2aab9c591bf0e059f57acce9ea.webp)
Human pluripotent stem cells (hPSCs) are emerging as a transformative platform for Parkinson’s disease, with recent phase I/II trials showing successful engraftment of hESC‑ and iPSC‑derived dopaminergic neurons. Yet extensive data reveal that cultured hPSCs frequently acquire recurrent genetic lesions—most...
Abbott received FDA approval for its next‑generation CardioMEMS HERO reader, a pulmonary artery pressure device for heart‑failure patients. The HERO unit is 60% lighter than earlier readers and incorporates built‑in Wi‑Fi and cellular connectivity, allowing measurements anywhere. The upgrade builds on...
Ultrasonic spray coating is emerging as a core engineering discipline for drug‑eluting implants, with parameters such as drug‑to‑polymer ratio, nitrogen carrier‑gas flow, nozzle height, and spray power dictating coating thickness, profile, and elution behavior. The article explains how variations in...
Researchers at Cornell’s Weill Institute introduced QUASARR‑seq, a high‑throughput assay that measures promoter and enhancer activity simultaneously. The study found that most human regulatory elements can function as both promoters and enhancers, following a unified regulatory logic. A bidirectional feedback...
Researchers at Italy’s National Research Council have created an all‑optical bio‑photovoltaic interface using iron‑doped lithium niobate crystals. By projecting patterned laser light, they inscribe reversible electric fields that trap, align, and deform fibroblast cells without any electrodes or wiring. Cells...
Researchers have engineered size‑shifting lipid nanoparticles that grow from ~100 nm to >300 nm after intraperitoneal injection, exploiting a capsule‑filter mechanism that blocks entry into the liver and spleen while allowing passage to the pancreas. The enlarged particles deliver mRNA payloads—including CRISPR‑Cas9...
This morning @generate_biomed celebrated its IPO on the @NasdaqExchange. We founded Generate at @FlagshipPioneer in 2018, asking: What if we could generate novel protein therapeutics using generative AI tools – without relying on traditional trial-and-error discovery? Today’s IPO is a major...
Eli Lilly unveiled LillyPod, a DGX SuperPOD built with 1,016 NVIDIA Blackwell Ultra GPUs delivering more than 9,000 petaflops of AI performance. The system powers genomics, protein‑diffusion, small‑molecule graph neural networks and foundation models, allowing billions of in‑silico experiments. Constructed...
The FDA approved Hernexeos through the national priority voucher program, a mechanism that fast‑tracks drugs addressing unmet medical needs. Industry leaders are evaluating direct‑to‑consumer (DTC) models to broaden access to HIV prevention tools, leveraging digital and telehealth channels. Additionally, the...
$NKTR is not getting a *fraction* of the credit it deserves. One positive development after another, and the stock simply cannot break out. Look at all that has happened since the stock was at these same levels 2...
Calorie restriction (CR) and broader dietary restriction (DR) remain the most robust, evolutionarily conserved interventions for extending lifespan and healthspan across species. In rodents, CR can increase lifespan by up to 40%, while human data suggest only modest gains of...
An intriguing tale of three cities. How do the PD1xCTLA4 bispecific agents lorigerlimab, volrustomig, and cadonilimab stack up when it comes to design and performance? Some useful and pointed lessons here for biotech execs: https://t.co/xaSW15Fvdl https://t.co/dhXbiI3N6v
Three bispecific antibodies targeting PD‑1 and CTLA‑4—lorigerlimab, volrustomig, and cadonilimab—illustrate divergent engineering strategies. Lorig erlimab relies on a knob‑into‑hole heterodimer, volrustomig adopts a common light chain format, and cadonilimab incorporates Fc‑silencing mutations. Their distinct designs have produced markedly different safety...
Bayer's HYRNUO (sevabertinib) received accelerated FDA approval on November 19, 2025 for adult patients with HER2‑mutated, non‑squamous non‑small cell lung cancer who have progressed after prior therapy. The approval is based on the single‑arm SOHO‑01 trial, which enrolled 122 patients across 78...
Atrium spins out of Avidity, aiming to target rare heart diseases with RNA https://t.co/xLp2oQDHGy $RNA $NVS #startups
A round‑up of leading experts highlights how cellular reprogramming has moved from a laboratory curiosity to a near‑clinical anti‑aging platform. Researchers describe partial, epigenetic‑focused approaches that can rejuvenate cells without erasing identity, and four biotech firms outline their distinct delivery...
“The era of de novo proteins is coming a lot faster than probably people recognize,” Generate's CEO Mike Nally tells me. “I think within the next year or two you’ll start seeing de novo designs enter the clinic.” https://t.co/u3WQcDD3jL
The Block layoff news was a big deal yesterday. I’m sorry to say that ML driven automated labs are going to do the same to lab jobs in life sciences that AI is starting to do to other industries.
Precise Bio successfully implanted PB-001, the world’s first 3D‑bioprinted, cell‑based corneal graft, in a legally blind patient during a phase‑1 trial. The implant combines human endothelial cells with a transparent collagen scaffold, replicating the properties of donor tissue. By using...
AI is helping to make the diagnosis of rare diseases. A new multicenter study in Japan showed smartphone AI diagnosis of acromegaly via hands was better than experienced endocrinologists https://t.co/qBiazetToV https://t.co/dSZpJ2us84
Here's the link to watch yesterday's webinar on venture capital, a companion to this year's top 100 biotech VCs list with @ChrisDoko Lots of positive feedback here. Post your thoughts. https://t.co/c16y04mFsD
Cure Parkinson’s has expanded its Research Committee by adding eight new members from leading UK and European institutions, spanning neurology, bioinformatics, pharmacology and more. The charity also welcomed three early‑career researchers as interns, extending the programme beyond the UK to...
Elizabeth Cairns' piece on $Novo is reminiscent of Bristol Myers' strategic fail against Merck and Keytruda. They were focused on the wrong things, made bad trial design decisions and got stuck in the mud of their own making. Everyone should...
The AGBT Day 2 video recap features Julianna LeMieux and Kevin Davies highlighting the meeting’s headline announcements. Vizgen unveiled a spatial‑omics platform aimed at organoid research, while Illumina introduced the TruPath Genome product, formerly known as Constellation mapped reads. Bruker launched...
The episode covers three main business stories: Tamarin Bio’s $13.6M Series A to build an AI model‑coordination platform that makes tools like AlphaFold accessible to wet‑lab biologists; the FDA’s reversal on Moderna’s mRNA‑1010 seasonal flu vaccine, spurring a modest stock...
AMSBIO (Oxford, UK) now provides ready‑to‑use lentiviral particles with titers exceeding 1×10⁷ IFU / mL, eliminating the need for in‑house virus production. The third‑generation self‑inactivating vectors are supplied in research‑grade and GMP‑grade formats, requiring only BSL‑2 containment. High‑titer, purified particles enable efficient...
1/ The hardest skill in bioinformatics? It's not coding. It's not stats. A hiring manager told me their biggest challenge: finding bioinformaticians who can talk to biologists. Here's why that matters: https://t.co/76HjMDdGIK
Genetic Alliance UK’s equity report warns the NHS is falling short for the 3.5 million Britons living with rare diseases. A quarter of patients wait three years for a diagnosis, only 5 % of conditions have approved treatments, and just 10 % of...
How does obesity catalyze neurodegeneration? Our paper just out @NatMetabolism https://t.co/kx25urgpJx free access https://t.co/aL1FBN9MyI Phenomenal work by @chen_bandy pulling this together
“You can’t stand there after the fact and blame the open-label design,” he said. “You designed it. You designed a noninferiority margin. You picked the patients. And then the trial doesn’t work.” https://t.co/acS5i5s5NU
A year-long trastuzumab emtansine (T‑DM1) regimen showed 98% three‑year invasive‑disease‑free survival in early‑stage HER2‑positive breast cancer, comparable to standard paclitaxel‑plus‑trastuzumab. The ATEMPT trial of nearly 500 patients found similar overall survival but markedly lower neuropathy and hair loss with T‑DM1,...
Great to see our former National School of Tropical Medicine @BCM_TropMed trainee Dr. Leroy Versteeg at my Brown Molecular Medicine talk at @UTHealthHouston in our @TXMedCenter https://t.co/F9IGkVNkfM
The FDA granted accelerated approval to Boehringer Ingelheim’s oral HER2 kinase inhibitor Hernexeos six weeks after the company filed its application, marking the second approval under the new Commissioner’s National Priority Voucher (CNPV) program. The decision expands Hernexeos use to...
Today’s brief highlights four pre‑clinical advances: Paratus Sciences unveiled PS‑1001, a pan‑inflammasome blocker targeting IL‑1β and IL‑18 for hidradenitis suppurativa; researchers identified the SCAN circuit as a core pathway disrupted in Parkinson’s disease; RX‑10616 demonstrated enhanced radiotherapy response in head‑and‑neck...
Prospect Therapeutics announced the identification of a new series of small‑molecule inhibitors targeting Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2). The lead compounds demonstrate nanomolar potency and selectivity in biochemical assays and effectively suppress cytokine signaling in cellular...
1/@TesseraTx has announced that the U.S. FDA has granted Fast Track and Orphan Drug designations to TSRA-196 - its leading In Vivo Gene Editing program which is being jointly developed with $REGN, for the treatment of patients with alpha-1 antitrypsin deficiency...
The FDA’s Drug Trials Snapshots program publishes concise, consumer‑focused summaries of the demographic makeup and outcomes of pivotal clinical trials for newly approved drugs. Launched in 2015, the tool covers only New Molecular Entities and original biologics approved after that...
Researchers analyzed plasma‑derived extracellular vesicles (EVs) from a healthy aging cohort using size‑exclusion chromatography, surface profiling, nanoparticle tracking and small‑RNA sequencing. They observed age‑dependent shifts in EV surface markers—decreases in CD3, CD56, HLA‑A, CD45 and increases in CD14, CD69—signaling immunosenescence...
Ionis Pharmaceuticals received FDA acceptance of its supplemental NDA for olezarsen and a priority‑review designation, with a PDUFA action date of June 30, 2026. The decision is backed by two Phase III CORE studies—CORE (n=617) and CORE2 (n=446)—that evaluated once‑monthly subcutaneous...
GSK’s depemokimab, marketed as EXDENSUR, received FDA approval on December 16, 2025 as a six‑month subcutaneous add‑on for severe eosinophilic asthma in patients aged 12 and older. Approval was based on two 52‑week, double‑blind, placebo‑controlled trials (SWIFT‑1 and SWIFT‑2) that...
Researchers highlight that senescent cells arising from DNA replication errors that generate whole‑chromosome duplications—polyploidy‑induced senescence (PIS)—are biochemically distinct from senescence caused by other stresses. Existing literature often fails to separate polyploid senescent cells from diploid counterparts, obscuring their unique roles...
UW Medicine and Seattle Children’s Hospital received $328,133 from the Sudden Unexplained Death in Childhood (SUDC) Foundation to launch a four‑year study using PacBio HiFi long‑read sequencing as a first‑line test. The project will sequence 200 child‑parent trios to uncover...
The FDA issued a suite of new guidance documents in early 2026 aimed at easing development of therapies for rare diseases, including the Rare Disease Evidence Pathway, Plausible Mechanism Pathway, and innovative trial designs for cellular and gene therapies. The...
Eli Lilly’s oral GLP‑1 small‑molecule orforglipron demonstrated greater A1C reduction and weight loss than oral semaglutide, while offering flexible dosing without fasting. GSK announced a $950 million acquisition of 35Pharma, securing HS235, an activin‑signaling inhibitor aimed at pulmonary hypertension. The deal taps...
Researchers engineered a series of antifreeze peptides (AFPTs) with systematically varied charges on their non‑ice‑binding sites (NIBSs) to uncover how charge influences ice‑growth inhibition. They found that moderate net‑negative charges improve peptide adsorption onto ice via hydration‑mediated interfaces, while excessive...
Quotient Sciences has extended its commercial partnership with Ipsen to manufacture Sohonos (Palovarotene), the approved therapy for fibrodysplasia ossificans progressiva (FOP), an ultra‑rare disease affecting fewer than 1,000 people worldwide. The deal includes Ipsen's investment in a pneumatic closed‑transfer system...
ENDRA Life Sciences announced that its Taeus Liver device achieved high measurement consistency in a MASLD study, recording an intraclass correlation coefficient of 0.89 and a standard error of measurement of 3.3%. The trial involved 14 participants and 56 data...
Cowellnex Co., Ltd. and Metagen, Inc. will begin joint research in February 2026 to create new gut‑microbiota test items and a personalized food‑recommendation algorithm using Cowellnex’s three‑year shotgun metagenomic dataset. The partnership leverages Cowellnex’s high‑precision Japanese microbiome data and Metagen’s...
Researchers introduced MICRO, a multimaterial, single‑step 3D‑printed electrochemical device that integrates electrodes, microfluidic channels, and housing in one build. By leveraging dual‑extrusion printing of conductive and insulating polymers, the platform eliminates wiring, sealing, and machining steps traditionally required for screen‑printed...
