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Biogen reported Phase Ib data for its antisense oligonucleotide salanersen, showing substantial neurofilament light chain reductions and new motor milestones in children with spinal muscular atrophy (SMA) who had previously received onasemnogene abeparvovec. The study of 24 participants demonstrated a roughly 75% biomarker drop and functional gains in all patients, with half achieving at least one new WHO‑defined milestone. Salanersen was well tolerated, with only mild to moderate adverse events. Biogen also unveiled a global Phase III program that will test once‑yearly dosing across presymptomatic infants, post‑gene‑therapy infants, and older treatment‑naïve patients.
Lonza has broadened its agreement with Genetix Biotherapeutics to increase manufacturing capacity for ZYNTEGLO, the sole FDA‑approved gene therapy for transfusion‑dependent beta‑thalassemia. Production will continue at Lonza’s Houston, Texas, dedicated cell‑and‑gene therapy site, with provisions to scale up for future...
The article surveys seven early‑stage programmes tackling KRAS, each proposing a distinct therapeutic angle. While many firms still chase the classic model of deeper, longer, or more selective pathway blockade, these initiatives span elegant biochemical tricks to counterintuitive concepts that...
The U.S. Food and Drug Administration approved leucovorin, a synthetic vitamin B9, solely for cerebral folate deficiency, a rare genetic disorder. Earlier this year, President Trump and HHS Secretary Robert Kennedy Jr. promoted the drug as an autism cure, prompting a surge in...
In this episode, host Ashley Vance talks with Nabiha Saklayen, co‑founder and CEO of Celino, about induced pluripotent stem cells (iPSCs) and their rapid evolution from a Nobel‑winning discovery to emerging therapies. They discuss how iPSCs can be reprogrammed from...
Researchers have engineered an antibody‑drug conjugate that simultaneously triggers HIV’s entry‑facilitating conformational shift and binds the newly exposed epitopes. In vitro tests showed the dual‑action molecule achieved seven‑ to ten‑fold greater neutralization than the antibody alone and outperformed separate mixtures...
Cambridge, Massachusetts remains a premier life‑sciences hub, anchored by giants like Intellia Therapeutics and Moderna and supported by the MassBio trade group. While BioSpace job postings fell 4% year‑over‑year in February, they surged 17% month‑over‑month, reflecting renewed hiring momentum. Six...
Veristat announced an expansion of its regulatory and clinical‑trial services aimed at Chinese pharmaceutical and medical‑device companies seeking faster entry into European markets, as well as the United Kingdom, Switzerland, the United States, Canada and Australia. The CRO highlighted recent...
Parabilis Medicines announced that the FDA has granted both Orphan Drug and Fast Track designations to zolucatetide, the first direct inhibitor of the β‑catenin:TCF interaction, for desmoid tumors. Early Phase 1/2 data showed a 100 % disease‑control rate in ten patients and...
Life Biosciences has secured FDA IND approval for ER-100, the first partial epigenetic reprogramming therapy to enter human trials. The gene‑therapy delivers OCT‑4, SOX‑2 and KLF‑4 to retinal ganglion cells via a single intravitreal injection, aiming to reset age‑related epigenetic...
Astellas Pharma leveraged the Trump administration’s push for lower U.S. drug prices to argue for higher reimbursement for its eye drug Izervay in Japan, winning a more generous price level. Meanwhile, a review of FDA adverse event reports found that...
The FDA is rolling out the Adverse Event Monitoring System (AEMS), replacing the legacy FAERS platform and consolidating reporting across drugs, biologics, devices, cosmetics, food, tobacco, and veterinary products. AEMS introduces standardized electronic submissions, AI‑driven redaction and analytics, and a...
Researchers at ETH Zurich have introduced a boron‑based ligation strategy that overcomes the concentration barrier in chemical protein synthesis. By masking potassium acyltrifluoroborates (KATs) with chiral zwitterionic complexes, the team achieved efficient peptide coupling at micromolar levels, far lower than...
Chiesi Group and inhalation CDMO Bespak have expanded their long‑standing partnership by increasing pressurized metered‑dose inhaler (pMDI) manufacturing capacity at Bespak’s Holmes Chapel, UK site. The move supports Chiesi’s Carbon Minimal Inhaler (CMI) program, which targets up to a 90 %...
Researchers at Johns Hopkins University demonstrated that the extremophile bacterium Deinococcus radiodurans can survive simulated asteroid‑impact pressures up to 2.4 gigapascals, with roughly 60% of cells remaining viable. The study identified the bacterium’s crystalline membrane protein arrangement and robust DNA‑repair...
Aplantex has partnered with Investissement Québec to use its state‑of‑the‑art demonstration plant for green biotechnology extraction. The collaboration gives Aplantex access to advanced equipment and technical experts, enabling faster refinement of plant‑based molecules with antioxidant, anti‑inflammatory, antimicrobial and anti‑aging properties....
As a medical school professor, I teach students that vitamin D does far more than build bones. This new study shows just how much more. Researchers found that vitamin D supplementation significantly improved glucose tolerance, lowered fasting blood sugar, and reduced...
Researchers published in JAMA Neurology identified high‑level EBNA‑1 peptide antibody titers as a robust biomarker that distinguishes relapsing‑remitting multiple sclerosis from MOGAD and seronegative NMOSD. In a cohort of over 1,300 neuroinflammatory patients, persistent EBNA‑1 positivity in at least two...
Biotech Science at Less Than Zero | Ep. 955 https://t.co/4DXKsaKXdO [00:49] Biotech capital winter deep dive [01:17] Scientific leaps vs frozen markets [02:31] The 2020–2021 biotech bubble [03:42] Crossover funds fuel IPO surge [04:56] Interest rates freeze biotech funding [07:58] Negative enterprise value phenomenon [10:37] Why Big Pharma...
The KRAS niche has been a deluge of new selective and pan/multi inhibitors. Now there are combos to watch out for – we may be making progress. Here are insights on 7 of them: https://t.co/F9HmM8A4ud https://t.co/IqWw6n2m3k
UCB announced results from its Phase III BE BOLD trial comparing Bimzelx (bimekizumab) to risankizumab in 553 adults with active psoriatic arthritis. The study met its primary endpoint, showing Bimzelx superior in achieving ACR50 responses at week 16. Bimzelx is...
Another potential GLP-1 drug benefit seen in people with diabetes and brain metastases for improved survival; a retrospective study that needs further assessment but intriguing https://t.co/nZm3xEYQMH https://t.co/V76S7o1MJb
The blood neuromarkers p-tau181 and p-tau217 that can predict and diagnose Alzheimer's disease are also abnormal in certain types of amyloidosis, indicating both some non-specificity and potential help in diagnosis of these conditions https://t.co/jodvMY6TNK
Genethon presented two‑year data from its dose‑escalation phase showing that GNT0004, administered at 3 × 10¹³ vg/kg, produced sustained functional gains and biomarker improvements in ambulatory DMD boys. Motor scores rose 9 points on the NSAA, the 6‑minute walk distance improved by...
A causal link between severe Covid or flu and lung cancer risk by impairing immunity and promoting inflammation, as shown in multiple murine models https://t.co/NvE6uIYFZh
DNA damage repair gene alterations influence the tumor immune microenvironment in advanced non-small cell lung cancer [Feb 14, 2025] @KamyaSankar et al. @ReckampK Lung Cancer https://t.co/mXZ80zYke3 #lcsm #ImmunoOnc #PrecisionMedicine @TempusAI https://t.co/UJwZnw4qnc
Zinereo Pharma is preparing to launch Otibiome, a clinically evaluated probiotic derived from *Ligilactobacillus salivarius* PS7, aimed at preventing recurrent acute otitis media (AOM) in children. A pilot study of 61 kids showed an 84% drop in AOM episodes over...
Real-world clinical genomic analysis of Pts w/ BRAF mutated cancers identifies BRAF class II & III as a population of unmet medical need [3/7/22] Severson et al #ESMO2240P @TempusLabs https://t.co/s5PEBEczlv @Annals_OncologyDOI: https://t.co/c3wxWyUuX0 #PrecisionMedicine #caxtx
Mitochondria and Aging: Redox Balance Modulation as a New Approach to the Development of Innovative Geroprotectors (Fundamental and Applied Aspects) https://t.co/TcG9GjMVg7 @IJMS_MDPI https://t.co/gxCC9SsO7z
An international team led by the Turku Bioscience Centre discovered that applying sound‑wave vibration to vocal‑fold cancer cells restores cellular movement and markedly reduces tumor aggressiveness. The mechanical stimulation lowered levels of the oncogenic protein YAP, both in cultured cells...
With $100M, Vima pursues an oral drug for movement disorders https://t.co/gLrhlYDMSy by @gwendolynawu #biotech #startups
Genomic Landscapes of Early-Onset vs Average-Onset Colorectal Cancer Populations [Feb 28, 2025] Storandt et al. @Cancers_MDPI https://t.co/as7Jolal12 #crcsm #PrecisionMedicine most significant difference b/w eoCRC & aoCRC was higher rates of BRAF mutation among Pts w/ aoCRC https://t.co/7qB4b3A1WK
Scientists at UC Santa Cruz trained a mouse‑derived brain organoid to solve the classic cart‑pole balancing problem, boosting success from 4.5% with random stimulation to over 46% using adaptive electrical cues guided by an AI algorithm. The tiny, pepper‑corn‑sized tissue,...
New nonprofit launches with at least $500 million to modernize scientific process for AI era https://t.co/yuypno6rVY via @ADeAngelis_bio
Oncology is moving toward combination regimens, and Merck's Keytruda paired with Pfizer/Astellas' Padcev has emerged as a leading duo. A phase 3 trial in muscle‑invasive bladder cancer showed the combo cut the risk of recurrence, progression and death roughly in half,...
The MIT professor who has been appointed by Robert F Kennedy Jr to review the safety of Covid-19 vaccines has failed to meet basic scientific standards in his own research on the topic, according to more than a dozen scientists...
An emulated target trial using the TriNetX database compared tocilizumab and rituximab in 1,194 rheumatoid arthritis‑associated interstitial lung disease (RA‑ILD) patients each over a five‑year follow‑up. The analysis found no statistically significant difference in all‑cause mortality (15.9% vs 17.7%) or...
Capricor Therapeutics announced that the FDA has scheduled an August 22 decision on its investigational Duchenne muscular dystrophy cell therapy, deramiocel, after lifting a prior complete response letter. The biotech resubmitted an enhanced package that includes robust Phase III HOPE‑3 data showing...
ARTHEx Biotech announced that its RNA‑based drug ATX‑01 has received U.S. FDA Fast Track designation for treating Myotonic Dystrophy Type 1 (DM1). The therapy works by inhibiting miR‑23b, thereby increasing free MBNL protein, correcting splicing errors and reducing toxic DMPK mRNA...
UK health agencies and the Wellcome charity have launched a joint initiative to fund high‑quality infectious‑disease clinical trials in Africa, South Asia and Southeast Asia. The program, co‑led by the NIHR, the Foreign Commonwealth Development Office and Wellcome, will prioritize...
Researchers from Harbin Institute of Technology and Stanford University found that the tropical cockroach Blaptica dubia can rapidly degrade polystyrene. In a 42‑day experiment, 50 roaches consumed about 6 mg of plastic per day, removing 54.9% of the material and achieving...
RA Capital, Forbion, and Canaan have collectively invested in Solstice Therapeutics, the CTLA-4 antibody partner of Harbour Therapeutics. The funding round, reportedly a multi‑million dollar Series B, will support Solstice's pre‑clinical and early clinical programs. By backing Solstice, the investors...
Eurofins Biomnis has developed and validated a new LC‑MS/MS method to detect and quantify cereulide toxin in human stool samples. The assay meets ISO 15189 requirements, accounting for matrix effects, and delivers turnaround times suitable for routine clinical labs. Cereulide, linked...
Researchers have identified the erectile‑dysfunction drug sildenafil as a potential therapy for Leigh syndrome, a fatal mitochondrial disorder affecting roughly one in 40,000 births. In cell models, the compound corrected mitochondrial membrane potential and normalized gene expression, while treated mice...
Early data presented at the San Antonio Breast Cancer Symposium indicate that GLP‑1 receptor agonists, long used for diabetes and obesity, are linked to markedly lower breast cancer recurrence and mortality. Retrospective analyses of thousands of patients showed up to...
The FDA granted its first approval for a therapy targeting cerebral folate transport deficiency, an ultra‑rare neurological disorder, with Wellcovorin (leucovorin calcium) showing meaningful neurological improvements in 89% of patients. Meanwhile, over‑the‑counter naloxone prices have slipped by roughly $0.49 each...
NICE has issued final draft guidance recommending fezolinetant 45 mg once daily for moderate to severe menopause‑related vasomotor symptoms when hormone replacement therapy is unsuitable. The recommendation enables the drug to be supplied through the NHS, providing a new non‑hormonal option...
Researchers report that fenofibrate, a PPARα agonist approved for hyperlipidemia, extends lifespan and reverses cellular senescence in several mouse models, including D‑galactose‑induced, 18‑month‑old, and SAMP8 strains. The drug reduces age‑related lipid accumulation and restores mitochondrial function by up‑regulating CPT1C, a...
Researchers at Oregon State University have developed a DNA‑based barcoding assay that measures, in living mice, which lipid nanoparticles successfully deliver gene‑editing cargo to target cellular compartments. The technique identified that many particles are degraded in lysosomes, while a newly...
Researchers at Brown University, Rhode Island Hospital, and VA Providence demonstrated that simultaneous electrical stimulation above and below a spinal cord lesion can restore both leg movement and spatial sensory feedback in people with complete spinal cord injuries. In a...
