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Every morning
Weekly
Tuesday recap
A 56‑year‑old Chinese patient became the first living person to be connected to a genetically modified pig liver via extracorporeal perfusion, keeping him alive until a human liver transplant could be performed. The operation, carried out at Xijing Hospital, used a pig liver engineered with six genetic edits to lower rejection risk. The bridge therapy lasted several days, after which the patient received a human liver and is now recovering. The case marks a milestone for xenotransplantation as a viable interim solution for organ‑failure patients.
UT San Antonio has been given a $38biin ARPA-H grant to study the health benefit effects of three drugs in humans, namely, rapamycin, dapagliflozin and semaglutide …🧵 https://t.co/fAX14ngoyD
![[Comment] Pimicotinib: A New Agent for an Orphan Disease](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/5b160e8a2b2821c82aa66047ca629679.webp)
Tenosynovial giant cell tumour (TGCT) is a rare, locally aggressive neoplasm driven by CSF‑1 over‑expression. The phase 3 MANEUVER trial evaluated pimicotinib, a selective CSF1R kinase inhibitor, against placebo in patients with inoperable or relapsed diffuse‑type TGCT. Results demonstrated statistically significant...
Asian biotech firms outside China announced 72 deals in 2024‑25, emphasizing first‑in‑class assets over me‑better or me‑too programs. At least four transactions involve novel targets such as CNTN4, GPR52, MTARC1 and OK‑1, spanning new modalities and indications. Global pharmaceutical companies...
Biotech firms are cutting staff as venture‑capital inflows dry up, clinical‑trial setbacks mount, and operating costs climb. While breakthroughs in gene therapy, RNA therapeutics and precision medicine continue, companies are trimming non‑core programs to preserve cash. Strategic restructurings aim to...
Scientists delivered naked Cldn11 messenger RNA directly into the testes of genetically infertile male mice, restoring Sertoli cell function and enabling spermatogenesis. The treatment produced viable sperm that generated healthy offspring via in‑vitro fertilization, without permanent germline alteration. The approach...
Researchers at NYU Langone Health discovered that herpes simplex virus 1 (HSV‑1) deploys its early protein ICP4 to partially liquefy the densely packed human cell nucleus. This fluidization creates a more permissive environment for viral replication compartments, accelerating virus production....
Schrödinger’s stock has plunged 88% over five years, leaving a sub‑$1 billion market cap. The company’s AI‑driven software segment posted 11% revenue growth in 2025 and aims for modest 12% growth in 2026, while shifting customers to cloud contracts that compress...
The FDA released a comprehensive Patient‑Focused Drug Development (PFDD) glossary to standardize terminology across its guidance documents mandated by the 21st Century Cures Act and PDUFA VI. The glossary defines key concepts such as attributes, benefit‑risk assessment, clinical outcome assessments, patient‑reported...
Cognito Therapeutics closed an oversubscribed $105 million Series C round, led by Morningside Ventures with new investors such as Apollo Health Ventures. The capital will fund the readout of its HOPE pivotal study, FDA submission preparation, and commercialization of its Spectris neurostimulation...
British biotech Vesalic reports a breakthrough that links amyotrophic lateral sclerosis (ALS) to a systemic metabolic dysfunction detectable in blood extracellular vesicles (EVs). The company says the EVs carry a toxic lipid cargo that harms motor neurons, and its blood‑based...
At the recent ASCO‑GU meeting, industry leaders highlighted that the greatest threat to emerging bladder‑cancer programs is strategic, not clinical. Phase‑2 candidates are poised to enter Phase‑3 trials, but shifting control arms, evolving endpoints, and changing patient demographics risk rendering...
Researchers at RIKEN discovered that the pond microalga Euglena agilis processes the majority of its introns using a non‑canonical splicing code, bypassing the typical GT‑AG splice site signals. Over 70 % of its introns lack the standard motifs, instead following a...
Psychedelic antidepressants are poised for FDA review this year, driven by strong investor and patient interest. William Blair analysts note that while Johnson & Johnson’s Spravato generated $1.7 billion in 2025 sales, psychedelics are unlikely to capture the entire treatment‑resistant depression market. Companies...
Alnylam Pharmaceuticals has signed a deal with Tenaya Therapeutics, providing $10 million upfront and the potential for up to $1.13 billion in milestones to discover up to 15 new genetic targets for heart disease. Tenaya will apply its modality‑agnostic platform to validate...
UniQure’s one‑time gene therapy AMT‑130 showed a 75% slowdown in Huntington’s disease progression in its Phase 1/2 trial, prompting expectations for a BLA filing in early 2026. The FDA, however, reversed its earlier stance and now requires a sham‑controlled Phase 3 study,...
Amazing quote in this NYT story on the FDA from @By_CJewett. Janet Woodcock, a top drug regulator for decades, said reversals for a promising therapy send shock waves through the industry. “The Huntington’s refusal I thought was truly evil,” she told NYT....
Thermo Fisher Scientific has opened a Cryo‑Electron Microscopy Drug Discovery Center in South San Francisco, offering pharmaceutical and biotech firms direct, hands‑on access to cutting‑edge cryo‑EM instrumentation. The facility is designed to accelerate structural insight generation, enabling faster, more cost‑effective...
The CNBC Cures Summit opened with Becky Quick urging families and innovators to accelerate rare‑disease research. Speakers highlighted a widening gap between rapid scientific breakthroughs—gene therapies, AI‑driven diagnostics, and modular “nodal biology”—and an aging regulatory framework. Leaders from Biogen, the...
My friend @adamfeuerstein delivers some great reporting and smart commentary on the FDA's direction that $QURE conduct a new randomized trial for its Huntington's drug. A senior FDA official and the company disagree on the basic question of whether the...
PepGen’s Phase 2 FREEDOM2 trial in myotonic dystrophy type 1 received a partial FDA clinical hold due to concerns over a sub‑chronic mouse study that showed blood‑pressure changes. The agency did not question the Phase 1 human data, and the company continues dosing...
Randomized trials for non‑small cell lung cancer often miss real‑world nuances that affect patients' quality of life. Real‑world data (RWD) and social determinants of health (SDOH) expose hidden barriers such as transportation gaps and limited molecular testing access. Pharma can...
Roche announced a $480 million, five‑year investment in South Korea to build a national clinical‑trial ecosystem, fund R&D infrastructure, and train specialized personnel. The pledge also includes support for domestic biotech startups aiming for global markets. This move aligns with a...
A new BMJ meta‑analysis of over 600,000 U.S. veterans with type‑2 diabetes found that patients prescribed GLP‑1 agonists such as semaglutide or tirzepatide were 14% less likely to develop substance‑use disorders (SUD) than those on SGLT2 inhibitors. Over a three‑year...
TECregen, a biotech firm developing thymus‑rejuvenating biologics, announced Dr Klaas Zuideveld as its new chief executive officer. Zuideveld brings more than two decades of experience in pharmaceutical and biotech leadership, spanning translational strategy, clinical development, and global regulatory affairs. He has overseen...
Late 2025 saw the FDA grant traditional approval to pirtobrutinib, an oral, reversible BTK inhibitor targeting multiple B‑cell malignancies. The drug demonstrated robust efficacy in BTK‑resistant chronic lymphocytic leukemia and small lymphocytic lymphoma, backed by positive Phase 3 data and early...
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE...
Ribosomes as Drug Targets. The Quiet Rewrite of Psychiatry and Beyond. | Ep. 950 https://t.co/gKlWpzO1Ci https://t.co/ImGOmUVrSO
Beiersdorf’s AI‑driven Skin Age Clock, patented in 2021, reads epigenetic markers to estimate the biological age of skin. The technology underpins the Epicelline ingredient, now featured in Nivea and Eucerin lines, which claims to reverse cellular aging. To refine the...
Neuropathic pain affects 7-10% of all people globally. New 🐁 study finds resveratrol alleviates neuropathic pain associated with restoration of mitochondrial dynamics 👏 https://t.co/zJ8LvwZ9UP https://t.co/hdCK7UgqBj
stunning week in cell and gene therapy: in vivo base editing of PCSK9 delivered via LNP, w clinical data (Nat Med), preclinical POC of CAR-astrocytes engineered w AAV (Science) and of self-amplifying RNA delivered IM (also Science), Japan approves gene...
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to consume solid tumors from the inside out. The spores infiltrate the oxygen‑free tumor core, proliferate and break down cancerous tissue. By inserting an oxygen‑resistant gene and a quorum‑sensing...
Doesn’t look like $LLY and $NVO have much to worry about from $RHHBY obesity 🤷🏻♂️
Will we give a shot of protective, self-amplifying RNA in patients with a heart attack in the future? @ScienceMagazine an intriguing innovation https://t.co/FcJme1ElyU https://t.co/lABFRkR3qg
The UK government announced a new package of measures to accelerate space‑based pharmaceutical manufacturing, offering regulatory clarity and a sandbox for companies developing drugs in microgravity. The initiative, led by the Department for Science, Innovation and Technology, brings together the...
When cancer niches are rapidly changing they create both a vacuum of indecision and fresh opportunities for smart players. Here we explore bladder cancer and look at where the field is headed... https://t.co/cf93Qb8dhe https://t.co/fx5V1MVwYN
China competition is coming for the US's multimillion-dollar gene therapies -- a must-read from @Jared_Whitlock about what's happening there and how it will affect access around the world: https://t.co/F7KaMv3Kkp
Targeted therapies are reshaping oncology, yet chemotherapy remains essential. Immune checkpoint inhibitors like Keytruda generate $31.7 bn sales in 2025 and cover more than 40 indications. The FDA granted 63 antibody‑drug conjugate review designations in 2024, nearly double the previous peak....
Can an AI model be a generalist to work and perform well for all types of medical images? Just published, our MedVersa paper @NEJM_AI Led by @pranavrajpurkar Free access https://t.co/5llNf94hAF https://t.co/Bf01zUZ1sk
20 years of Yamanaka stem cell factor research culminates in 2 conditional approvals for heart disease and Parkinson's disease in Japan https://t.co/8oxKxXYafu by Shinya Yamanaka @CellStemCell https://t.co/U878IxSfSi @ScienceMagazine https://t.co/HCCPaTEP7e
The FDA has begun reviewing Hansa Biopharma’s imlifidase, a drug that desensitises highly sensitised kidney‑transplant patients, with a decision expected by December 19, 2024. If approved, it would be the first U.S. therapy to improve transplant odds for the 10‑15 % of...
I speculated last month that HMPL-A580 was an anti-EGFR ADC https://t.co/XFDtZfWWkH and $HCM just confirmed that this is the case. Relevant for $ALXO
Erythrocyte–anti-PD1 conjugates in persons with advanced solid tumors resistant to anti-PD1/PDL1: preclinical characterization and results of a phase 1 trial https://t.co/1GQMlUHn4Y https://t.co/0it5nTvUm0
Xtalks has spotlighted Giorgia Palano, PhD, a Life Science Consultant at Knightec Group, highlighting her expertise in validation strategies and regulatory compliance. Palano works with cross‑functional teams to ensure quality documentation and continuous improvement across complex life‑science operations. She emphasizes...
When you drew the sample matters as much as what you measured. Biological data has nuances that get buried if you don't think about timing. https://t.co/eWzEu8FDfc
Researchers identified mitochondrial circular RNA MT‑RNR2 as abundant in young cells but depleted in older individuals and senescent fibroblasts. The RNA‑binding protein GRSF1 binds both linear and circular MT‑RNR2, linking it to TCA‑cycle enzymes and glucose metabolism. Loss of GRSF1...
Researchers discovered that boosting mitochondrial metabolism in neurons enhances long‑term memory formation in both fruit flies and mice. By reducing expression of the mitochondrial calcium exporter Letm1, calcium accumulates in the mitochondrial matrix, over‑activating metabolic pathways and increasing ATP production...
Akeso presented Phase‑II COMPASSION‑03 data for cadonilimab in patients with recurrent or metastatic cervical cancer who progressed after platinum chemotherapy. The trial reported a median overall survival of 17.5 months across the cohort, with 24‑month OS rates of 40.9% irrespective...
Researchers at WEHI and the Burnet Institute have mapped the human immune response to Plasmodium vivax, revealing how antibodies neutralize the parasite. The study, published in Immunity, shows that protective immunity depends on antibody function and simultaneous targeting of multiple...
HUTCHMED has launched a first‑in‑human Phase I/IIa trial of HMPL‑A580, its second antibody‑targeted therapy conjugate, in the United States and China. The multi‑centre, open‑label study will evaluate safety, tolerability, immunogenicity, pharmacokinetics and early efficacy across dose‑escalation and expansion cohorts. HMPL‑A580...
