#rna-therapeutics

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fromTNW | Artificial-Intelligence
17 hours ago

OpenAI launches GPT-Rosalind, an AI model for life sciences research

GPT-Rosalind is designed to support evidence synthesis, hypothesis generation, experimental planning, and multi-step scientific workflows across biochemistry, genomics, and protein engineering.
Medicine
#peptides
fromwww.scientificamerican.com
2 days ago
Public health

The Trump administration is looking to experts to weigh in on peptides

Peptides are gaining popularity despite being largely unproven and risky, with increasing use among influencers and athletes.
fromwww.theguardian.com
1 week ago
Medicine

Traceability is vital': labs test thousands of unregulated substances amid peptide craze

The underground market for injectable peptides in the UK has surged, with thousands of unregulated substances being tested for safety and efficacy.
Medicine
fromwww.theguardian.com
1 week ago

Traceability is vital': labs test thousands of unregulated substances amid peptide craze

The underground market for injectable peptides in the UK has surged, with thousands of unregulated substances being tested for safety and efficacy.
#cancer-research
fromNature
3 days ago
Cancer

Four rising stars shaping the future of cancer research

A new generation of cancer researchers is focused on improving diagnostics and treatments to enhance survival rates for cancer patients.
fromenglish.elpais.com
1 month ago
Medicine

The very long road from a cancer cure' in mice to one in humans

Promising mouse cancer cures often fail to become safe, effective human drugs; premature media claims can create false patient expectations and hinder responsible research progress.
Cancer
fromNature
3 days ago

Four rising stars shaping the future of cancer research

A new generation of cancer researchers is focused on improving diagnostics and treatments to enhance survival rates for cancer patients.
fromwww.nature.com
3 days ago

Editorial Expression of Concern: Creation of human tumour cells with defined genetic elements

Concerns were raised regarding a potential duplication of two bands in Fig. 1b. Due to the age of the article, the raw data were not available.
Science
fromNature
2 days ago

Quantum computers take on health care: light-sensitive cancer drugs win US$2 million contest

A team won a $2-million prize for using quantum computing to develop light-sensitive cancer drugs, but no grand prize was awarded.
Artificial intelligence
fromFast Company
4 days ago

AI is rewriting the rules of biological experiments, but safety regulations aren't keeping up

AI is autonomously designing and running biological experiments, outpacing current governance systems meant to regulate these capabilities.
fromTNW | Startups-Technology
3 days ago

Helical closes $10M seed to turn bio foundation models into systems

Helical's thesis is that bio foundation models, AI systems trained on vast genomic, transcriptomic, and proteomic datasets, have already crossed a quality threshold that makes computational hypothesis-testing meaningful in pharma research.
Venture
Medicine
fromThe Atlantic
1 day ago

The DNA Fix for Aging

Michael Prescott diagnosed himself with Werner syndrome after suffering multiple organ failures and rapid aging.
fromTNW | Amazon
1 day ago

Amazon launches AI Bio platform to accelerate early-stage drug discovery

Amazon Bio Discovery enables scientists to run complex computational workflows through more than 40 AI-specialized foundational models, trained on a wide range of biological datasets. These models generate and evaluate potential drug molecules, alongside AI agents that help scientists to select models, optimize inputs and evaluate candidates according to their research.
Public health
Cancer
fromNature
3 days ago

Improving cancer survival rates will require hard policy choices

Global cancer incidence is rising, necessitating early detection strategies and public education on risk factors.
#autoimmune-diseases
fromNature
1 week ago
Science

Daily briefing: CAR-T-cell therapy keeps a trio of autoimmune diseases at bay

Science
fromNature
1 week ago

Daily briefing: CAR-T-cell therapy keeps a trio of autoimmune diseases at bay

Engineered immune cells successfully treated a woman with three autoimmune diseases, resulting in no symptoms or medication needed after fourteen months.
Cancer
fromwww.independent.co.uk
1 week ago

Cell therapy helps woman with three autoimmune diseases make remarkable' recovery

A woman with severe autoimmune diseases achieved treatment-free remission after innovative cell therapy at University Hospital Erlangen.
Medicine
fromwww.theguardian.com
1 week ago

Woman with three deadly diseases has remarkable' recovery after cell therapy

A woman with three autoimmune diseases achieved remission after CAR T-cell therapy, marking a significant breakthrough in treatment options.
#genetics
fromThe Washington Post
1 week ago
Health

One way to live longer: Win the genetic lottery

Genetic factors account for about 50% of human lifespan, significantly higher than the previously estimated 20%.
fromNature
1 week ago
Science

Daily briefing: A treatment to reverse cellular ageing is about to be tested in people

Partial reprogramming may enter clinical trials soon, and a DNA tweak can induce sex reversal in female mice.
Health
fromThe Washington Post
1 week ago

One way to live longer: Win the genetic lottery

Genetic factors account for about 50% of human lifespan, significantly higher than the previously estimated 20%.
Science
fromNature
1 week ago

Daily briefing: A treatment to reverse cellular ageing is about to be tested in people

Partial reprogramming may enter clinical trials soon, and a DNA tweak can induce sex reversal in female mice.
Medicine
fromwww.bbc.com
1 day ago

'Breakthrough' Alzheimer's drugs unlikely to benefit patients, report suggests

Breakthrough Alzheimer's drugs are unlikely to significantly benefit patients despite slowing cognitive decline.
Cancer
fromwww.scientificamerican.com
4 days ago

Person functionally cured of HIV after bone marrow transplant from sibling

A 63-year-old man achieved functional HIV cure through a bone marrow transplant from his brother with a rare genetic mutation.
#car-t-therapy
Medicine
fromInsideHook
4 days ago

CAR T Therapy Shows Promise Against Autoimmune Diseases

CAR T therapy shows promise in treating autoimmune conditions, providing significant relief for patients previously unresponsive to traditional treatments.
OMG science
fromNature
2 weeks ago

'Treasure trove' of antiviral proteins could inspire powerful molecular tools

Bacteria possess a vast array of antiviral proteins, identified through machine-learning algorithms, which could lead to innovative biotechnologies.
Cancer
fromNews Center
4 days ago

Targeting Novel Long Non-Coding RNA May Improve Glioblastoma Treatment - News Center

Increased expression of a novel long non-coding RNA drives glioblastoma cell growth and may inform new therapeutic strategies.
fromwww.scientificamerican.com
3 weeks ago

Why you should keep getting mRNA vaccines

mRNA vaccines have demonstrated their ability to prevent approximately eight million COVID infections within the first six months of their rollout, showcasing their effectiveness in combating the pandemic.
Coronavirus
from24/7 Wall St.
2 weeks ago

5 Biotechs That Big Pharma Could Snap Up as Oncology M&A Heats Up

Incyte tops this list due to its rare combination of commercial scale, cash generation, and pipeline depth. The company posted FY2025 revenue of $5.14 billion, up 21.2% YoY, anchored by Jakafi generating $828.2 million in Q4 2025 alone (+7% YoY) and Opzelura delivering $207.3 million (+28% YoY). With $3.58 billion in cash and 14 pivotal clinical trials underway, Incyte offers an acquirer immediate revenue, margin expansion potential, and a deep oncology pipeline spanning KRASG12D, CDK2 inhibition, and mutCALR.
Venture
Business
from24/7 Wall St.
3 weeks ago

Why Barclays Thinks Ionis Is Headed for a 45% Surge From $73 to $106

Ionis Pharmaceuticals has seen significant stock performance, with Barclays predicting a price target of $106 based on the potential of its sHTG treatment.
fromComputerWeekly.com
3 days ago

Novo Nordisk partners with OpenAI to AI-power drug development | Computer Weekly

Novo Nordisk plans to deploy advanced artificial intelligence capabilities to analyze complex datasets, identify promising drug candidates, and reduce the time required to move from research to patient.
Medicine
#gene-editing
Medicine
fromArs Technica
1 week ago

Clinical trial shows gene editing works for -Thalassaemia, too

An improved gene editing system reactivates a fetal hemoglobin gene to treat β-Thalassaemia, building on CRISPR's success with sickle-cell anemia.
Medicine
fromArs Technica
1 week ago

Clinical trial shows gene editing works for -Thalassaemia, too

An improved gene editing system reactivates a fetal hemoglobin gene to treat β-Thalassaemia, building on CRISPR's success with sickle-cell anemia.
Cancer
fromNews Center
1 week ago

Long Non-Coding RNA May be a Promising Therapeutic Target for Cancer - News Center

A long non-coding RNA, IGF1R-AS1, activates oncogenic pathways in prostate cancer, highlighting its potential as a therapeutic target.
Science
fromFuturism
2 weeks ago

A Startup Has Been Quietly Pitching Cloned Human Bodies to Transfer Your Brain Into

Cloning efforts have evolved from animals to controversial human embryo models, with ambitions for brainless human clones for organ transplants.
#ai-in-healthcare
fromTNW | Opinion
6 days ago
Medicine

AI health tech is booming. The cures are not.

AI in drug discovery shows promise but has not yet delivered significant breakthroughs for patients.
fromFast Company
2 weeks ago
Medicine

The AI drug revolution is real but the hype around it isn't

AI may revolutionize drug discovery, but it cannot simplify the complexities of human biology or guarantee successful treatments.
Medicine
fromTNW | Opinion
6 days ago

AI health tech is booming. The cures are not.

AI in drug discovery shows promise but has not yet delivered significant breakthroughs for patients.
Medicine
fromFast Company
2 weeks ago

The AI drug revolution is real but the hype around it isn't

AI may revolutionize drug discovery, but it cannot simplify the complexities of human biology or guarantee successful treatments.
Cancer
fromNature
1 week ago

New drugs take aim at one of cancer's deadliest mutations

Researchers are developing innovative strategies to target the cancer-causing KRAS protein, previously deemed 'undruggable', showing promising results in clinical trials.
Medicine
fromNature
1 week ago

Saturation editing of RNU4-2 reveals distinct dominant and recessive disorders - Nature

De novo variants in RNU4-2 cause ReNU syndrome, a neurodevelopmental disorder characterized by developmental delays and other severe symptoms.
fromFast Company
1 week ago

AI is coming for superbugs

Antibiotics are essential for modern medicine, but bacteria are evolving and developing resistance, turning routine infections into life-threatening conditions. A global analysis estimates that antibiotic-resistant infections could cause over 39 million deaths by 2050.
Medicine
Science
fromNature
1 month ago

Synthetic circuits for cell ratio control - Nature

Synthetic biology enables artificial cell differentiation and division of labor by engineering genetic and epigenetic circuits that mimic natural stem cell asymmetric division processes.
Silicon Valley
fromKqed
1 month ago

How South San Francisco Became the Birthplace of Biotechnology | KQED

South San Francisco transformed from an industrial meatpacking and steel manufacturing hub into the world's biotechnology capital, hosting over 250 biotech companies including Genentech.
#car-t-cell-therapy
#biotech
Medicine
from24/7 Wall St.
2 weeks ago

MRNA, SRPT, and KRYS Phase 3 Data Will Shape XBI's 2026 Performance

The SPDR S&P Biotech ETF offers equal-weight exposure to biotech companies, presenting unique opportunities and risks influenced by FDA regulatory changes.
Medicine
from24/7 Wall St.
2 weeks ago

MRNA, SRPT, and KRYS Phase 3 Data Will Shape XBI's 2026 Performance

The SPDR S&P Biotech ETF offers equal-weight exposure to biotech companies, presenting unique opportunities and risks influenced by FDA regulatory changes.
Medicine
fromNature
3 weeks ago

Eye drops made from pig semen deliver cancer treatment to mice

Pig semen-derived eye drops can halt retinal tumor growth and preserve vision in mice, offering a potential treatment for retinoblastoma in children.
Science
fromNature
1 month ago

From cancer to Alzheimer's: could a renewed focus on energy transform biomedicine?

Energy flow, governed by universal physics principles, provides a more fundamental understanding of biological processes and disease than molecular mechanisms alone.
fromFortune
1 month ago

An Australian tech entrepreneur used AI to help create the first-ever bespoke cancer vaccine for a dog to treat his beloved pet Rosie | Fortune

I went to ChatGPT and came up with a plan on how to do this. He also used AlphaFold, an AI tool from Google's DeepMind, to find mutated proteins that could be potential targets for treatment. While an immunotherapy treatment that looked like a good fit for Rosie was identified, the drugmaker wouldn't provide it.
Cancer
Venture
from24/7 Wall St.
1 month ago

Forget AI. This Biotech Stock's Taking Off Right Now

AI stocks face correction risk due to rising capital expenditures without proportional profits, making biotech an undervalued alternative for AI-driven growth exposure.
Medicine
from24/7 Wall St.
3 weeks ago

Sarepta Therapeutics Zooms 20% Higher on Promising Clinical Data: Is SRPT Ready for a Bigger Breakout?

Sarepta Therapeutics' stock surged 20% after releasing promising clinical data for two siRNA programs targeting rare neuromuscular diseases.
US news
from24/7 Wall St.
2 months ago

Biotech Turnaround: How Moderna Went From Revenue Collapse to YTD Rally Leader

Moderna stock rose 37% YTD in 2026 driven by earnings beats, $2B cost cuts, international manufacturing deals, and pipeline monetization despite regulatory headwinds.
Tech industry
from24/7 Wall St.
2 months ago

NVIDIA Just Made a Bigger Push Into AI Drug Discovery

Nvidia's stock has traded sideways for six months despite strong AI demand and strategic deals that may enable an eventual breakout.
#ai-drug-discovery
fromFortune
2 months ago
Science

AI drug startup Insilico Medicine launches an AI 'gym' to help models like GPT and Qwen be good at science | Fortune

fromFortune
2 months ago
Science

AI drug startup Insilico Medicine launches an AI 'gym' to help models like GPT and Qwen be good at science | Fortune

Business
from24/7 Wall St.
1 month ago

Wall Street Thinks These 4 Biotech Stocks Will Double--At Least--This Year

Wall Street analysts assign unanimous or near-unanimous Buy ratings to four clinical-stage biotech companies, implying potential gains up to 384% driven by late-stage pipelines and upcoming catalysts.
Medicine
fromTNW | Health-Tech
1 month ago

Kupando raises 10M more to take its immunity drug into the clinic

Kupando raised €10 million in Series A extension funding to advance KUP101, a dual TLR agonist, toward first human trials for solid tumors and drug-resistant infections.
fromNature
2 months ago

This AI has chemical expertise - and helps synthesize 35 new drugs and materials

Now, researchers have created an artificial-intelligence system that vastly simplifies and accelerates the process of chemical synthesis. The system, which is called MOSAIC and is described in a study published in Nature on 19 January, recommended conditions that researchers were able to use to generate 35 compounds with the potential to become products like pharmaceuticals, agrochemicals or cosmetics without needing to do any further trawling or tweaking.
Artificial intelligence
fromNature
4 weeks ago

Masked mitochondria slip into cells to treat disease in mice

When mitochondria are exposed to tissue or blood, they lose the electrical gradient across their outer membrane. Mitochondria that lack such a gradient are recognized by a cell's internal machinery as damaged and quickly destroyed. The vast majority of previous studies involved injecting 'naked' mitochondria directly into the bloodstream or tissue sites, but the approach isn't very efficient, so researchers often have to use 'ridiculous' doses of mitochondria.
Medicine
OMG science
fromHarvard Gazette
2 months ago

Why did that cancer cell become drug-resistant? - Harvard Gazette

TimeVault records and stores cellular gene-expression history inside living cells, enabling retrieval of past gene-activity information to study differentiation, stress responses, adaptation, and drug resistance.
Cancer
fromHarvard Gazette
1 month ago

Unlocking hidden pocket on a billiondollar drug target - Harvard Gazette

Researchers discovered a hidden binding pocket on cereblon protein that enables more selective and safer cancer drug design through targeted protein degradation.
Medicine
fromBusiness Matters
1 month ago

UK biotech Ternary raises 3.6m to scale AI platform for next-generation drugs

Ternary Therapeutics secured £3.6 million in seed funding to develop an AI-driven platform for engineering molecular glues, a new class of medicines that bring proteins together to destroy disease-causing targets.
OMG science
fromNature
2 months ago

Regulatory grammar in human promoters uncovered by MPRA-based deep learning - Nature

Massively parallel reporter assays provide cell-type-specific causal training data enabling more direct inference of DNA sequence effects on promoter activity than epigenomic maps.
Medicine
fromWIRED
1 month ago

Japan Approves the World's First Treatment Made With Reprogrammed Human Cells

ReHeart and Amusepri represent breakthrough cell transplant therapies addressing severe heart failure and Parkinson's disease by replacing damaged tissue with functional cells derived from iPS cells.
Science
fromNews Center
2 months ago

New Underlying Mechanisms May Support Proper Transcriptional Regulation and Improve Targeted Therapies - News Center

BET proteins, particularly BRD4, regulate transcription initiation and elongation independently of bromodomains, with implications for targeted therapeutic development.
Cancer
fromNature
1 month ago

Cancer blood tests are everywhere. Do they really work?

Multi-cancer early detection blood tests show promise but lack regulatory approval and rigorous trial evidence, with initial results indicating limited effectiveness in improving cancer outcomes.
Medicine
fromBusiness Matters
1 month ago

Flavia Pichiorri: Turning Cancer Research Into Real Therapies

Dr. Flavia Pichiorri bridges laboratory discoveries and clinical applications in blood cancer research, focusing on therapeutic targets and radiation strategies to accelerate patient treatment outcomes.
Science
fromwww.nature.com
2 months ago

Scalable and multiplexed recorders of gene regulation dynamics across weeks

CytoTape enables multiplexed, genetically encoded, spatiotemporally scalable recording of gene regulation dynamics in single cells for up to three weeks with minute-scale resolution.
fromNews Center
2 months ago

Identifying Mechanisms Supporting Nanoparticle Therapy for Autoimmune Diseases - News Center

We knew that if you inject these nanoparticles into an animal model, the nanoparticles get taken up by antigen presenting cells and this resulted in increased regulatory T-cells and decreased inflammatory disease. However, we did not know how this happens,
Science
Cancer
fromFuturism
1 month ago

Bacteria Engineered to Eat Tumors From the Inside

Researchers engineered Clostridium sporogenes bacteria to consume tumor cells from inside, offering a potential alternative to traditional cancer treatments.
fromArs Technica
1 month ago

Have we leapt into commercial genetic testing without understanding it?

Martschenko's argument is largely that genetic research and data have almost always been used thus far as a justification to further entrench extant social inequalities. But we know the solutions to many of the injustices in our world-trying to lift people out of poverty, for example-and we certainly don't need more genetic research to implement them. Trejo's point is largely that more information is generally better than less.
Science
Science
fromMail Online
2 months ago

Scientists use AI to create a virus never seen before

Scientists used AI and gene-assembly tools to create Evo-Φ2147, a novel 11-gene virus designed to kill pathogenic E. coli.
fromNews Center
2 months ago

AI Model May Improve RNA Sequencing Research - News Center

Scientists in the laboratory of Rendong Yang, PhD, associate professor of Urology, have developed a new large language model that can interpret transcriptomic data in cancer cell lines more accurately than conventional approaches, as detailed in a recent study published in Nature Communications. Long-read RNA sequencing technologies have transformed transcriptomics research by detecting complex RNA splicing and gene fusion events that have often been missed by conventional short-read RNA-sequencing methods.
Cancer
fromNature
1 month ago

AI tools can design genomes. Will they upend how life evolves?

Biology is undergoing a transformation. After centuries of studying life as it evolves naturally, researchers are now using a combination of computation and genome engineering to intervene, generating new proteins and even whole bacteria from scratch. The use of artificial-intelligence tools to design biological components, an approach known as generative biology, is set to turbocharge this area of research. Just last year, scientists used AI-assisted design to produce artificial genes that can be expressed in mammalian cells.
Science
Science
fromArs Technica
2 months ago

Tiny, 45 base long RNA can make copies of itself

A 45-base RNA ribozyme can self-replicate, supporting RNA-based origin-of-life scenarios where RNA carried both genetic information and catalytic functions.
Medicine
fromwww.scientificamerican.com
1 month ago

Pioneering gene therapy may treat a deadly seizure disorder

Gene therapy drug zorevunersen significantly reduces seizures in Dravet syndrome patients by targeting the underlying SCN1A gene mutation, offering hope for treatment-resistant cases.
fromHarvard Gazette
2 months ago

How COVID-era trick may transform drug, chemical discovery - Harvard Gazette

Laboratories turned to a smart workaround when COVID‑19 testing kits became scarce in 2020. They mixed samples from several patients and ran a single test. If the test came back negative, everyone in it was cleared at once. If it was positive, follow-up tests would zero in on who was infected. That strategy, known as group testing, saved valuable time, money, and resources.
Science
Cancer
fromenglish.elpais.com
1 month ago

Douglas Hanahan, biologist: We don't necessarily need a cure, what we really need is cancer without disease'

Cancer cells acquire hallmarks: uncontrolled proliferation, evasion of growth barriers, resistance to programmed death, and relative immortality, driving tumor diversity and treatment variability.
Science
fromNature
2 months ago

Construction of complex and diverse DNA sequences using DNA three-way junctions - Nature

DNA writing remains limited by short oligo synthesis and two-way junction assembly methods, hindering affordable, scalable construction of large, complex synthetic DNA.
fromwww.scientificamerican.com
2 months ago

Lung cancer hijacks the brain to trick the immune system

For years, scientists have viewed cancer as a localized glitch in which cells refuse to stop dividing. But a new study suggests that, in certain organs, tumors actively communicate with the brain to trick it into protecting them. Scientists have long known that nerves grow into some tumors and that tumors containing lots of nerves usually lead to a worse prognosis.
Science
fromTNW | Deep-Tech
2 months ago

Aerska raises $39M to help RNA medicines reach the brain

For families living with neurodegenerative disease, the hardest part is not always the diagnosis. It is the slow erosion that follows: memory fading, personality shifting, independence shrinking. It unfolds quietly. First, forgotten appointments. Then repeated questions. Then moments when a familiar face no longer feels familiar. The illness does not isolate itself to one body. It rearranges the lives around it.
Medicine
Medicine
fromLGBTQ Nation
2 months ago

These 4 promising breakthroughs are bringing HIV researchers closer to a cure - LGBTQ Nation

Significant scientific advances have produced promising combination therapies and experimental approaches that have eliminated HIV in rare cases but no widely scalable cure exists yet.
fromArs Technica
2 months ago

mRNA cancer vaccine shows protection at 5-year follow-up, Moderna and Merck say

As for side effects, the companies reported that little had changed from previous analyses; adverse events were similar between the two groups. The top side effects linked to the vaccine were fatigue, injection site pain, and chills. The results "highlight the potential of a prolonged benefit" of the vaccine combined with Keytruda in patients with high-risk melanoma," Kyle Holen, a senior vice president at Moderna, said. They also "illustrate mRNA's potential in cancer care," he said, noting that the company has eight more Phase 2 and Phase 3 trials going for mRNA vaccines against a variety of other cancers, including lung, bladder, and kidney cancers.
Medicine
Medicine
fromNature
2 months ago

China's biotech boom: why the nation must collaborate to stay ahead

China leads in drug manufacturing and biotech innovation, but geopolitical scrutiny and moves toward a closed biotech ecosystem threaten scientific collaboration and global medicine access.
Medicine
fromThe Atlantic
2 months ago

Their Mutated Genes Were Supposed to Be Harmless

People who carry single-gene mutations for disorders like thalassemia can experience real health effects, including lethargy and fainting, despite being labeled asymptomatic.
Medicine
fromwww.theguardian.com
2 months ago

Simple blood test can predict which breast cancer treatment will work best, study finds

A blood test measuring circulating tumour DNA predicts breast cancer treatment response before or within four weeks, enabling alternative therapies and avoiding ineffective drugs.
Medicine
fromIntelligencer
3 months ago

Did AI Alter the Course of This Baby's Life?

A newborn, Jorie, was diagnosed with DeSanto-Shinawi syndrome, a rare, incurable genetic disorder causing neurodevelopmental and physical challenges, with limited treatment options.
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