Know What's Happening in BioTech
Know What's Happening in BioTech
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Tuesday recap
EMAIL DIGESTS
Daily
Every morning
Weekly
Tuesday recap
Kyowa Kirin announced it will cease all clinical trials of rocatinlimab, an anti‑OX40 antibody once touted as a potential eczema blockbuster, after a safety review identified emerging malignancy signals. The review, conducted with former partner Amgen, uncovered a confirmed and a suspected case of Kaposi’s sarcoma, raising concerns about the drug’s mechanism. Although the incidence is below background rates, the company concluded the risks could outweigh benefits and will halt studies after participants finish safety follow‑up. The decision follows Amgen’s earlier relinquishment of rights and mixed results for similar OX40 drugs.
Scientists at Ludwig Maximilian University discovered that loss of the DJ-1 protein triggers ATP deficiency in human dopaminergic neurons, leading to reduced VMAT2 levels and impaired dopamine vesicle loading. The resulting dopamine oxidation fuels accumulation of pathological α‑synuclein species, a...
Real‑world evidence from Duke’s PRECISION platform shows Pluvicto (Lu‑177 vipivotide tetraxetan) delivers a median progression‑free survival of 13.5 months in PSMA‑positive metastatic castration‑resistant prostate cancer (mCRPC) after androgen‑receptor pathway inhibitor (ARPI) therapy, matching the pivotal PSMAfore trial. Patients who received Pluvicto...
The FDA granted priority review to Takeda’s rusfertide, a first‑in‑class hepcidin mimetic for polycythemia vera, after Phase III trials more than doubled response rates. A regulatory decision is expected in Q3 2026. The agency also expanded Novo Nordisk’s once‑weekly Sogroya to treat children...
A March 2026 study in Pediatric Research applied whole exome sequencing to seven patients with methylmalonic acidemia (MMA) and cystathionine beta‑synthase (CBS) deficiency, uncovering both known and novel pathogenic variants in MMUT, MMAA and CBS genes. The researchers linked specific mutation...
Researchers have engineered a nanocomposite incorporating silver (Ag) nanoparticles that markedly improves anticancer efficacy. The hybrid material boosts reactive oxygen species generation and enhances tumor cell uptake while sparing normal tissue. In vitro studies show synergistic activity with standard chemotherapeutics,...
The FDA has approved the Teal Wand, the first at‑home cervical cancer screening device, now available nationwide for women ages 25‑65 at average risk. The wand collects a dry cervical sample at home, which is mailed to a lab for...
The FDA has published a batch of newly added guidance documents, spanning drug exclusivity, post‑approval safety reporting, real‑world data studies, Bayesian trial methods, and patient‑preference research. Ten documents are highlighted, with six in draft form and four finalized, dated between...
A new Nature Metabolism study identifies extracellular matrix protein ECM1 as a pivotal early regulator of kidney fibrosis. Global ECM1 knockout mice develop spontaneous fibrosis and premature death, while AAV9‑mediated ECM1 knockdown in fibroblasts markedly lessens fibrotic burden. Mechanistically, ECM1...
The Anti‑Ageing Skin Care Conference 2026 will be held June 23‑24 at the Royal College of Physicians, featuring two days of presentations on healthy ageing, biomarker science, and emerging skin technologies. A roster of leading academics and industry R&D heads—including...
Chip Baird, after selling 2seventy bio to Bristol Myers Squibb, launched Poplar Therapeutics as CEO in early 2025. The company secured a $50 million Series A and an additional $45 million extension to fund Phase 1 trials of its anti‑IgE antibody PHB‑050. PHB‑050 aims to...
A MEDSIR‑hosted webinar titled “The Future is Collaborative: Transforming Clinical Trials” examined how collaborative‑initiated trials and investigator‑initiated trials (IITs) are reshaping oncology research. Speakers Dr. Javier Cortés and Dr. Antonio Llombart‑Cussac, both leading breast‑cancer oncologists, discussed their roles in recent...
A team of researchers proposes "pharmaceutical superintelligence," an AI‑driven pipeline that can turn a plain‑language request into a preclinical drug candidate and even outline clinical trial plans. The framework stitches together generative chemistry models, multi‑agent reasoning, and robotic labs into...
Researchers applied Bayesian machine‑learning to uncover risk factors for hepatosplenic multimorbidity in schistosomiasis. By fusing clinical records, environmental exposure data, and host genetic markers, the model pinpointed predictors such as specific immune‑gene variants and poor sanitation. Its capacity to continuously...
Researchers at Karolinska Institutet reported that phosphorothioate antisense oligonucleotides (ASOs) directly bind key DNA‑repair enzymes, forming nuclear condensates that trigger a false DNA‑damage response. The effect was observed at concentrations commonly used in laboratory assays, though clinical doses reach lower...
An international panel of 147 oncology leaders, including Prof. John Bridgewater, issued a consensus calling for sweeping reforms in the diagnosis, treatment, and research of cholangiocarcinoma (bile duct cancer). The disease remains deadly, with three‑quarters of patients dying within a...
Philadelphia’s biotech ecosystem, long known for CAR‑T breakthroughs, is diversifying into obesity, metabolic disease, gene editing, and solid‑tumor immunotherapies. Nine local companies illustrate this shift, from Alveus Therapeutics’ $197 million Series A obesity program to Cabaletta Bio’s autologous CD19 CAR‑T for autoimmune...
Multi‑cancer early detection (MCED) tests analyze blood, urine or saliva for tumor‑derived DNA, RNA or proteins, promising a single‑sample screen for dozens of cancers. While early‑stage detection could complement established organ‑specific programs, sensitivity varies widely, with some cancers identified in...
February 2026 saw a surge of biosimilar activity, with key regulatory approvals for products such as Sandoz Enzeevu (Eylea), Accord Filkri (Neupogen), and STADA Gotenfia (Simponi) across the US, Canada and the EU. Companies forged new licensing and commercialization deals that extend biosimilar...
More cases of Kaposi sarcoma for OX40. OX. 40. Is. Done. $SNY still projecting $3-$5B peak sales for Amli is a pipe dream. This value is going to $NKTR. $NKTR is, IMO, the single most undervalued name I...
Quell Therapeutics has launched the phase 1/2 CHILL trial of its autologous CAR‑Treg therapy QEL‑005 in rheumatoid arthritis and systemic sclerosis, after pausing the liver‑transplant program QEL‑001. The multi‑center study will enroll patients in the UK, Germany and Spain, with read‑out...
Boehringer Ingelheim has terminated its metabolic dysfunction‑associated steatohepatitis (MASH) collaboration with OSE Immunotherapeutics after an exploratory Phase 2 trial failed to meet its primary endpoint. The original 2018 deal included a €15 million upfront payment and up to €1.1 billion in milestone potential,...
RecovryAI, emerging from stealth, received FDA Breakthrough Device Designation for its physician‑prescribed Virtual Care Assistants (VCAs), AI tools that guide patients through post‑operative recovery. The designation, reserved for devices that can substantially improve care standards, accelerates the company’s engagement with...
In this episode, Bruno Quinney highlights four major developments: a long‑acting injectable HIV regimen (cabotegravir + rilpivirine) that cut virological failure risk by nearly half versus daily oral therapy; SolasCure’s ORES wound gel, which accelerated debridement 22‑fold and healing seven‑fold in chronic...
Lynk Pharmaceuticals announced topline Phase III data for its oral JAK inhibitor zemprocitinib in 356 patients with moderate‑to‑severe atopic dermatitis. Both the 12 mg and 24 mg doses met the co‑primary endpoints at week 16, delivering 38‑46 percentage‑point improvements in EASI‑75 and roughly 30 percentage‑point gains...
The GAO report reveals that, despite a conditional approval pathway introduced in 2004 and expanded in 2018, FDA‑approved animal drugs for minor species and niche uses remain scarce. From FY2018 through FY2025, only 13 drugs received conditional approval, all targeting...
New reporting from me taking you inside the $QURE - FDA Huntington's gene therapy imbroglio. I spoke to a senior FDA official late yesterday, who told me agency reviewers "are not convinced there’s any therapeutic benefit of the product. If...
The FDA concluded that UniQure’s experimental gene therapy for Huntington’s disease has not demonstrated therapeutic benefit based on existing clinical data. Reviewers said they are not persuaded by the evidence, prompting the agency to block the company from submitting a...
Alzheimer’s research is increasingly looking beyond plaques and tangles to the brain’s immune response. A study published in Elsevier's Pharmacological Research journal used AI to screen 1.6M compounds for a Galectin-3 (Gal-3) blocker that could cross the brain barrier. In Alzheimer’s-like...
Pierre Fabre seeks to revive US approval chances for spurned cell therapy https://t.co/gJeklT8asd @ByJonGardner $ATRA $QURE $RGNX
Jacksonville‑based startup MMI is preparing the first U.S. microrobotic surgeries to treat Alzheimer’s by clearing lymphatic drainage pathways in the neck. The FDA granted clearance for an initial safety cohort of 15 patients, with five slated for the first procedure...
Did the FDA’s individualized therapy guidance put a spike in the heart of a startup biotech? Fake news, says the HHS. Yeah, it did, says an insider. Exclusive from @RLCscienceboss and @Jared_Whitlock https://t.co/IN9o4VIp03
A new systematic review of LLMs in medicine @NatureMedicine @ekoermann https://t.co/npvDeqUvel “Despite thousands [4,609] of publications since late 2022, only a small fraction use real clinical data and just 19 randomized trials exist.”
Innovent’s pirtobrutinib, marketed as Jaypirca, received Chinese NMPA approval for adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma who have undergone at least one prior therapy, including a BTK inhibitor. The decision follows the phase‑III BRUIN CLL‑321 trial,...
The hardest problem in pharma data science isn't the algorithm. It's getting three different omics datasets to agree with each other. That's exactly what we'll dig into at #ddpeast26 on June 10 in Waltham. I'm joining as a panelist and roundtable moderator....
T-cell engagers for autoimmunity. Listen to Ken Song describe the opportunity as CEO of San Diego-based Candid Therapeutics. Sponsored by @AlphaSenseInc & Dash Bio. https://t.co/Ct2viH00c4
The cGAS‑STING pathway, a DNA‑sensing immune circuit, becomes aberrantly activated in the aging brain as mitochondrial and nuclear DNA escape into the cytoplasm. This chronic activation drives low‑grade neuroinflammation, contributing to neurodegenerative diseases such as Alzheimer’s, Parkinson’s and ALS. Preclinical...
$APGE should can the OX40 bispec IMO. You’re basically just tacking on a black box with that MOA at this point…
A recent analysis of 6,069 cognitively normal women examined whether epigenetic aging clocks predict incident mild cognitive impairment or dementia. Second‑ and third‑generation clocks (AgeAccelPheno, AgeAccelGrim2, DunedinPACE) were compared with first‑generation Horvath and Hannum measures. Only the AgeAccelGrim2 clock showed...
Patent term extension (PTE) offers up to five extra years of exclusivity for FDA‑approved drugs, but biologics present a unique hurdle because the law hinges on defining the “active ingredient.” Unlike small molecules, biologics are large, variable structures, making it...
The U.S. Centers for Medicare & Medicaid Services (CMS) has pushed back the deadline for manufacturers to join its GENEROUS pilot, extending the initial cut‑off to April 30 while keeping the final deadline at June 30. The voluntary program applies a Most‑Favoured...
Eight leading European microphysiological systems companies have launched the Industry Alliance for Microphysiological Systems (IAMPS), the world’s first trade association dedicated to MPS technologies. IAMPS will represent organ‑on‑chip, organoid and related NAM developers, aiming to harmonize standards, promote data sharing,...
Merck and Eisai disclosed Phase III LITESPARK-011 results showing that the oral combination of Welireg (belzutifan) and Lenvima (lenvatinib) outperformed cabozantinib in 747 patients with advanced renal cell carcinoma who progressed after anti‑PD‑1/PD‑L1 therapy. At a median follow‑up of 29...
Researchers introduced ApoFilter, an aptamer‑functionalized mesh filtration system that selectively removes plasma lipoproteins while allowing extracellular vesicles (EVs) to pass. The platform targets ApoA1 and ApoB100, depleting over 99% of HDL and (V)LDL within a minute using gravity‑driven flow. Integrated...
Dermatology research is booming, with more than 16,000 trials completed, ongoing, or planned by the end of 2025. The Asia‑Pacific region now hosts roughly 8,000 studies, a five‑fold increase over the past decade, matching North America and Europe combined. Low...
Researchers at Vilnius University used cryogenic electron microscopy to map eleven CRISPR‑Cas protein complexes, including three variants of a newly described Cas9‑Cas1‑Cas2‑Csn2 supercomplex. The study shows that Cas9, traditionally viewed only as a DNA‑cutting enzyme, also directs the selection and...
Researchers demonstrated that a clinically‑available 1.5 T static magnetic field can trigger the release of encapsulated molecules from magnetoliposomes (MLs). The study used citric‑acid‑stabilized Fe₃O₄ nanoparticles, with and without a chitosan coating, and monitored structural changes via SAXS and DLS. Fluorescence...
Lee Hood’s three‑decade instrument‑building effort birthed the automated DNA sequencer that made the Human Genome Project feasible and founded the Institute for Systems Biology, the cradle of systems‑level medicine. He coined the four P’s—predictive, preventive, personalized, participatory—arguing the first three are...
Earlier this year the UK government pledged £82.6 million to AI‑focused drug‑discovery firms, underscoring its ambition to lead the global digital health transformation. Major players such as AstraZeneca are launching dedicated health‑tech units like Evinova to accelerate clinical‑trial design, while the...
North Carolina’s Holly Springs is becoming a hub for next‑generation obesity drugs as Amgen and Roche commit more than $3.5 billion to build manufacturing capacity. Amgen’s $1.55 billion rollout includes a new drug‑substance plant and a second facility slated for 2032, while Roche’s...
