biotechnology

Harvard researchers find new gene pathway for sickle cell treatment

A team of researchers at Boston Children's Hospital, Dana-Farber Cancer Institute, and the Broad Institute has discovered a previously unknown genetic pathway that activates fetal hemoglobin, opening a potential new front in the treatment of sickle cell disease and beta…

First targeted therapy for rare genetic ALS succeeds in Phase 3 trial

Ionis Pharmaceuticals and Otsuka Pharmaceutical announced on Tuesday that ulefnersen, an investigational RNA-targeted therapy, met the primary endpoint of the Phase III FUSION trial in patients with a rare genetic form of amyotrophic lateral sclerosis, marking a first for this…

Syngenta unveils microRNA crop technology it calls a biological first in 20 years

Syngenta on Tuesday unveiled EXORT technology, a new biological platform that uses naturally occurring plant microRNAs to help crops increase yields by optimizing their own internal resources — what the company calls a first new mode of action in biologicals…

Novo Nordisk CEO says company is open to larger M&A deals

Novo Nordisk CEO Mike Doustdar told CNBC on Tuesday that the company is actively considering mergers and acquisitions to fill gaps in its drug portfolio, a day after the Danish drugmaker's Capital Markets Day in London left investors underwhelmed.

Chinese biopharma stocks surge on U.S. drug deal rules

Chinese biopharma stocks rallied sharply in Hong Kong on Monday after reports that the U.S. Treasury Department is drafting rules that would preserve American pharmaceutical companies' ability to license most drugs from Chinese firms. The Hang Seng Biotech Index rose…

Anthropic built a wet lab to push AI into drug science, Reuters reports

Anthropic on Wednesday unveiled the Life Sciences Verification Program, a beta initiative that for the first time gives vetted researchers access to its most advanced AI models — including its Mythos line — with relaxed biosafety guardrails tailored for biology…

Stanford’s AI ‘biotech company’ designed a lung cancer drug later validated by GSK

Researchers at Stanford University have built an artificial intelligence system that mimics an entire drug development company — complete with a chief scientific officer, specialized departments, and tens of thousands of AI employees — and published their findings Thursday in…

New ‘prime assembly’ method can fix whole genes in one step

A new genome engineering method called prime assembly now allows researchers to stitch long DNA fragments into precise, programmable locations within living human cells, a development that could open the door to universal gene therapies applicable across many patients. The…

Columbia study finds gene editing in embryos effective but too risky for clinical use

A team of researchers led by Columbia University has demonstrated that base editing can modify genes in human embryos with remarkable precision, achieving 100% accuracy in some experiments, but the study published Wednesday in Nature also exposed a range of…

Google DeepMind launches AlphaGenome Atlas mapping 9 billion DNA variants

Google Alphabet Inc. DeepMind on Tuesday released AlphaGenome Atlas, a database containing predictions for the molecular effects of all 9 billion possible single-letter DNA changes in the human genome — an effort the company describes as "the most comprehensive catalogue…

Gene-editing therapy hits 100% remission across 30-plus sickle cell and thalassemia patients

A base-editing therapy developed by Shanghai-based CorrectSequence Therapeutics has achieved transfusion independence or freedom from painful vaso-occlusive crises in every patient treated to date — more than 30 across four continents — according to clinical data published September 7 in…

Novartis, Bristol Myers face scrutiny over delayed CAR-T trial disclosures

Novartis and Bristol Myers Squibb Bristol-Myers Squibb Company have paused multiple clinical trials testing CAR-T cell therapies in autoimmune diseases, following three patient deaths and other serious safety incidents that have sent shockwaves through a field once seen as one…

Ivonescimab tops Keytruda in overall survival for first time in lung cancer trial

Akeso and Summit Therapeutics announced on September 2 that their bispecific antibody ivonescimab achieved a statistically significant overall survival improvement over Merck's blockbuster Keytruda in a Phase 3 trial treating PD-L1-positive non-small cell lung cancer, marking the first time a…

Novartis signs $3.2B deal for Alteogen’s injection technology

Novartis announced Wednesday it has signed a licensing and option agreement with South Korean biotech Alteogen worth up to $3.22 billion, gaining access to technology that converts intravenous drugs into simpler under-the-skin injections.

Novartis, Bristol Myers halt CAR-T autoimmune trials over safety concerns

Novartis and Bristol Myers Squibb have temporarily halted multiple clinical trials testing CAR-T cell therapies in autoimmune diseases after observing serious inflammatory side effects, the companies confirmed Monday.

CAR T-cell therapy drives severe arthritis into remission in first trial

Researchers at Charité—Universitätsmedizin Berlin have demonstrated for the first time that CAR T-cell therapy can drive severe rheumatoid arthritis into sustained remission, according to results from the COMPARE trial published in Nature Medicine on August 27, 2026. Disease activity decreased…

Engineered bacteria speed up ocean carbon removal in Harvard study

Researchers at Harvard University's Wyss Institute have genetically engineered a marine bacterium to speed up rock weathering in seawater, removing more carbon dioxide from the atmosphere in the process. The findings, published Friday in Nature Biotechnology, demonstrate that the modified…

BioNTech halts mRNA colorectal cancer vaccine trial

BioNTech shares fell sharply on Friday after the German biotech company announced it would terminate its Phase 2 clinical trial of autogene cevumeran, an individualized mRNA cancer vaccine developed with Roche's Genentech unit, in patients with high-risk colorectal cancer. The…

Inhaled RNA therapy restores lung protein in cystic fibrosis models

Researchers at the University of Toronto have demonstrated that chemically modified transfer RNA molecules, delivered to the lungs via inhaled nanoparticles, can restore production of a critical protein in models of cystic fibrosis — offering a potential treatment path for…

AI-designed antibody outperforms lab results in first blinded global benchmark

An AI-designed antibody has outperformed the best result from months of traditional laboratory experiments, according to the results of the first international blinded benchmark for AI antibody design, published in Nature Biotechnology on August 25, 2026.

Robotic platform runs two genetic codes simultaneously

Scientists have developed a way to run two separate genetic codes at the same time within a single biological system, bypassing one of the most fundamental constraints in synthetic biology. The research, published in Nature on August 26, 2026, introduces…

Scientists use engineered yeast to turn plastic into edible cookies

Researchers at Southern Illinois University Carbondale have used gene-editing technology to program yeast strains that transform discarded plastic bottles and crop waste into protein-rich, 3D-printed cookies — a development presented Monday at the American Chemical Society's fall meeting in Chicago.

Singapore unveils data center powered by living human neurons

The National University of Singapore has unveiled what it describes as the world's first independently operated biological data center prototype, a system that uses 16 million living human neurons grown from stem cells to perform computational tasks alongside conventional silicon…

Anthropic says Claude AI designed protein binders for 14 of 15 targets in lab tests

Anthropic announced Monday that its Claude AI models autonomously designed protein binders that succeeded against 14 of 15 targets in wet-lab testing, marking one of the most comprehensive demonstrations yet of an AI system executing end-to-end drug design work without…

MIT engineers build living circuit boards from bacteria

Researchers at the Massachusetts Institute of Technology have engineered bacteria to function as transistors, creating "living circuit boards" that can be printed onto growth medium in Petri dishes and perform basic computational logic using chemical signals instead of electricity.

Nobel laureate’s startup unveils first full virtual human cell model

GenBio AI on Tuesday unveiled AIDO Cell, a system it describes as the first virtual cell "world model" capable of simulating human cell behavior across its full biological hierarchy — from DNA and RNA through proteins to whole-cell dynamics —…

South Korean researchers build gene switch controlled by electromagnetic fields

Researchers at Dongguk University in Seoul have created a gene switch that can be turned on and off inside living animals using electromagnetic fields, a development that could reshape how gene therapies are delivered. The study, published in Cell on…

AI ’tissue clocks’ can estimate biological age of individual organs

Researchers have built artificial intelligence systems that can estimate how old individual human organs are biologically by reading microscopic patterns invisible to the naked eye, offering a new way to track aging and disease that may eventually require nothing more…

Scientists publish blueprint for AI ‘digital organism’

A team of researchers led by Eric Xing published a paper in Nature Medicine on Thursday laying out a roadmap for building an AI-driven "digital organism" — a system of connected AI models that could simulate how drugs or genetic…

Stanford researchers use AI to design 16 working viruses from scratch

Researchers at Stanford University and the Arc Institute have used generative AI to design complete viral genomes that function in the laboratory, producing 16 novel bacteriophages capable of infecting and killing *Escherichia coli* — including strains resistant to natural phages.…