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Every morning
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Gene therapy for sickle cell disease has moved from experimental promise to an actionable clinical reality, with early CRISPR‑based treatments already cutting crises and hospital stays. A recent Sanius Health survey of 94 patients shows strong interest—71% want more information—yet nearly half lack basic awareness and cite safety and cost as major concerns. The article argues that the next growth phase hinges on robust real‑world data, longitudinal monitoring, and transparent patient‑centric ecosystems. Industry leaders must pair scientific breakthroughs with new reimbursement models and infrastructure to sustain long‑term value.
$QURE AMT-130 update: Still blocked The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for...
$MRK claims a survival benefit in ovarian cancer irrespective of PD-L1 status, but FDA & EMA say BS. Via @ApexOnco -> https://t.co/Cld1O7Uuvp #ESGO26
Shine Technologies announced a $240 million financing round led by Patrick Soon‑Shiong’s NantWorks, bringing its total capital raised to over $1 billion. The infusion will accelerate the company’s commercial fusion initiatives and expand its Lu‑177 isotope production, a cornerstone of targeted cancer...
Researchers identified that the aging gut microbiome harbors increased Klebsiella aerogenes, which elevates histamine production and compromises intestinal barrier integrity. The excess histamine suppresses Nlrp6 expression, disrupting LC3‑mediated autophagy and intensifying inflammation in septic models. Experiments showed that lowering histamine...
A new donor‑derived cell‑free DNA (dd‑cfDNA) blood test is transforming post‑transplant surveillance by providing real‑time, noninvasive insight into graft health. The assay quantifies donor DNA fragments released when a transplanted organ is injured, offering a high negative predictive value that...
The U.S. FDA granted accelerated approval to Ascendis’ Yuviwel (navepegritide) for increasing linear growth in children aged two years and older with achondroplasia and open epiphyses. Approval rests on three placebo‑controlled trials and up to three years of open‑label extension...
Cell therapy manufacturing is vulnerable to contamination because living cells cannot be terminally sterilized. Regulatory bodies such as EU GMP Annex 1 and the FDA now require risk‑based contamination control strategies that prioritize closed, sterile, and automated processes. Closing open steps...
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending Dupixent (dupilumab) for paediatric chronic spontaneous urticaria (CSU) in children aged 2‑11. The recommendation is based on robust Phase III data from the LIBERTY‑CUPID...
Opus Genetics presented early Phase I/II data for its OPGx‑BEST1 gene therapy targeting best vitelliform macular dystrophy and autosomal‑recessive bestrophinopathy. In a sentinel 63‑year‑old participant, the treatment was well tolerated and delivered a 12‑letter gain in best‑corrected visual acuity after...
BioMarin’s enzyme substitution therapy Palynziq received FDA approval for use in adolescents aged 12 and older with phenylketonuria (PKU). The decision was based on Phase III PEGASUS trial results showing 44.4% of participants lowered blood phenylalanine below guideline levels, with a...
The article profiles 18 UK biotech firms spanning oncology, immunology, regenerative medicine and AI‑driven drug design, highlighting recent milestones such as Amphista’s $30 million upfront deal with BMS, Autolus’ FDA and EU approvals for obe‑cel, and Isomorphic Labs’ $600 million raise to...
Researchers have developed a monolithic 3D nanoelectrode array (HD‑NEA) that integrates 26,400 vertical nanowire electrodes directly onto commercial CMOS chips. The low‑temperature wafer‑scale post‑fabrication process maintains circuit functionality while delivering uniform, high‑yield electrode performance across 4‑inch wafers. In vitro recordings...
X4 Pharmaceuticals’ Xolremdi (mavorixafor) received a positive opinion from the European CHMP, recommending approval under exceptional circumstances with an EC decision expected in Q2 2026. The recommendation is based on the global Phase III 4WHIM trial involving 31 patients aged 12 and...
European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending conditional approval of Ipsen’s Ojemda (tovorafenib) as monotherapy for pediatric low‑grade glioma with BRAF fusions, rearrangements or V600 mutations. The recommendation is based on...
Researchers have engineered a sulfur‑vacancy‑rich Bi2S3‑x@PDA/CuS (BPC) nanocomposite that forms a Z‑scheme heterojunction and is further functionalized with hyaluronic acid (BPC@HA). The defect‑rich structure enhances charge separation, enabling efficient singlet‑oxygen generation under ultrasound and Fenton‑like hydroxyl‑radical production for chemodynamic therapy....
The FDA will render decisions this March on five high‑profile drug applications, including two delayed from last year. Bristol Myers Squibb seeks a psoriatic arthritis label for Sotyktu, backed by Phase 3 data showing a 54.2% ACR20 response. Aldeyra, Rhythm, GSK...
Quanterix (QTRX) reported FY 2025 revenue of $16.1 million, down $11 million from the prior year, driven by steep declines in collaboration and contract‑manufacturing income. R&D spending fell modestly to $140.7 million, while SG&A rose to $65.5 million due to higher professional fees for...
Cerus Corp reported record total revenue of $233.8 million in 2025, a 16% increase year‑over‑year, driven by strong product sales and robust growth in EMEA. Product revenue rose 14% for both the quarter and full year, with IFC kit sales...
Researchers integrated inflammatory cytokines, kynurenine pathway balance, and amygdala reactivity with the IDEA‑RS sociodemographic risk score to predict adolescent depression over three years. The composite model (IDEA‑BIO‑RS) raised predictive accuracy from a 0.715 AUC to 0.889, correctly classifying 82.2% of...
Xeris Biopharma reported record Q3 2025 product revenue of $74.1 million, a 40% year‑over‑year increase driven primarily by Recorlev’s 109% revenue jump. The company posted its first quarterly net income and an adjusted EBITDA of $17.4 million, reflecting strong operating leverage and...
UniQure reported a regulatory setback as the FDA now deems the Phase 1/2 external‑control data for AMT‑130 insufficient for a Biologics License Application, creating uncertainty around the U.S. filing timeline. Despite this, the high‑dose AMT‑130 achieved a statistically significant 75% slowdown...
Nuvation Bio reported $7.7 million net product revenue for Iptrozi in Q3 2025, driven by 204 new patient starts and payer coverage expanding to over 80% of U.S. lives. Clinical data showed an 89% overall response rate and a median...
A team of researchers from Singapore and China has engineered a rigid mRNA folding architecture that markedly improves intracellular processing and protein output. By integrating all‑atom and coarse‑grained molecular dynamics simulations with lipid‑nanoparticle (LNP) formulation, they created stiffened mRNA structures...
In a recent Unsupervised Learning episode, Razib Khan interviews Washington University genetics professor Mike White about his lab’s work on the biophysical architecture of regulatory DNA. White’s interdisciplinary approach combines functional genomics, synthetic biology, computational biology and deep‑learning to predict...
According to ITOA, NAD declines with age, causing epigenetic changes & disease susceptibility In 2010, we showed NAD-dependent enzyme, SIRT3, protects the heart 🐁 New study says NAD precursor NMN protects the heart via SIRT3 in mice fed a Western diet https://www.sciencedirect.com/science/article/pii/S0022316626000842
OK. Here it is. Over the last year, the @OctantBio and OpenADMET team have been hard at work developing scalable, quantitative, data-rich, and low-cost methods for assessing CYP reactivity and inhibition. The interplay between building a data engine and building...
Researchers at the Innovation Center of NanoMedicine unveiled PL‑display, a cell‑free platform that immobilizes individual peptides on magnetic beads for rapid screening. The method delivers over ten‑fold efficiency gains versus traditional cell‑based displays and can operate under high‑temperature, high‑salt, or...
Ginkgo reported full year 2025 revenue of $170M at the end of last week. When it went public at a $15B valuation in 2021, they guided to investors that they’d be adding $7.6B in new business PER YEAR in 2025...
But also it’s not only measles, pertussis (whooping cough) is also returning, and based on latest ACIP (or what used to be ACIP) stunts, we’ll see rotavirus, meningococcal meningitis, HBV liver cancer, return after that. A public health disaster built...
The article proposes designating green fluorescent protein (GFP) as a model protein to standardize protein research, much like fruit flies or mice serve as model organisms. It argues that GFP’s visual read‑out makes it ideal for benchmarking AI‑generated protein designs...
A pan-cancer compendium of 1,294 plasma cell-free DNA methylomes and fragmentomes enabling multicancer detection https://t.co/A6gHwcEzqq https://t.co/mSMcR0a833
This week’s coverage juxtaposes longevity hype with emerging science, highlighting David Sinclair’s bold claim that aging could be reversed within 10‑20 years, a new Nature paper confirming adult hippocampal neurogenesis, and research showing men lose the Y chromosome as they...
Researchers at Adelaide University conducted the first in‑vivo study of salcaprozate sodium (SNAC), the absorption enhancer used in oral semaglutide tablets such as Ozempic and Wegovy. Over a 21‑day period in rats, repeated SNAC exposure reduced fibre‑degrading gut bacteria, lowered...
Penn State researchers have built a bio‑hybrid memristor that couples synthetically produced DNA with a perovskite semiconductor. The device stores data in the molecular lattice of DNA while processing signals through the perovskite layer, effectively merging memory and compute. Laboratory...
$MRK Welireg could be about to expand its reach at last. #GU26 data via @ApexOnco with relevance to $RCUS -> https://t.co/k59284INdK
Looks like Miami is getting ready for biotech week (the week after next). Note: BiotechTV will be broadcasting from the Barclays conference March 10 & 11. https://t.co/ToZH0EgOVz
A phase‑2 trial of relmacabtagene autoleucel (relma‑cel), a CD19‑directed CAR‑T therapy, enrolled 59 Chinese patients with relapsed/refractory mantle‑cell lymphoma after BTK‑inhibitor failure. The study reported a 71.2% overall response rate and a 59.3% complete response rate, with median time to...
But note IL11 knockout mice are infertile (females) and have low body weight and some osteoporosis. Still interesting angle though.
A new review synthesizes recent advances that clarify the biology of Richter transformation (RT), the aggressive lymphoma that develops in 2%‑10% of chronic lymphocytic leukemia (CLL) patients. The analysis confirms that 70%‑80% of RT cases are clonally related to the...
Some very good news about engineering our cells vs cancer (accelerating the CAR). A short thread 1. For background, a new 5★ review on cancer immunotherapy @Cancer_Cell https://t.co/1Qz2rCs8I5 https://t.co/sJ6BwaXyjk
A single-cell atlas characterizes dysregulation of the bone marrow immune microenvironment associated with outcomes in multiple myeloma https://t.co/5t0M2eX0fB https://t.co/p3kv8LEzrC
A new analysis quantifies the operational and financial benefits of using Genmab’s epcoritamab, a dual‑indication bispecific antibody, for both relapsed/refractory diffuse large B‑cell lymphoma (DLBCL) and follicular lymphoma (FL). In a community‑practice model of 100 patients, the study projects 3,110...
Scientists at the University of Edinburgh's Wallace Lab engineered E. coli to generate hydrogen from simple sugars, including stale bread, and paired it with a palladium surface catalyst to hydrogenate organic molecules. The biocatalytic system achieved up to 99% conversion efficiency,...
A phase‑2b trial enrolling 94 adults with chronic hand eczema (CHE) without etiologic pre‑selection uncovered a mixed immune signature spanning type 2, type 3 and type 1 pathways. Dupilumab, an IL‑4Rα antagonist, delivered a 59.8% mean improvement in modified Total Lesion Symptom Score...
Lemnisca, a Bengaluru startup, is launching a digital‑twin platform that models fermentation at industrial scale, merging microbial biology with physical process conditions. The hybrid model, paired with an in‑house wet lab, creates a feedback loop that refines simulations and cuts...
This week’s Endpoints Weekly highlighted a surge of GLP‑1 developments, including new trial data and expanded indications that reinforce the class’s dominance in obesity and diabetes treatment. The newsletter also released its annual Top 100 venture investors list, showing a notable...
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended six new medicines, including three orphan drugs already approved by the FDA—Ojemda for pediatric low‑grade glioma, Palsonify for acromegaly, and Xolremdi for WHIM syndrome. The agency also gave a...
There are a few known chemical exposures in early pregnancy that interact with autism genes. But not Tylenol. I’ve spoken to RFK Jr about them but he had no interest, and he can’t process scientific information. Here’s my article https://t.co/pxLaDLInlf
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