The Cas9 protein together with RNA and DNA (orange; artist's illustration). A new therapy uses the CRISPR-Cas9 gene editing system to treat a rare genetic disease called hereditary angioedema.Credit: Alfred Pasieka/Science Photo Library The therapy, named lonvoguran ziclumeran (lonvo-z), was designed to treat one form of a genetic disorder that causes episodes of painful and potentially dangerous swelling in the throat and other parts of the body. Lonvo-z deploys the CRISPR-Cas9 gene-editing system to disable the gene that produces prekallikrein, a protein made in the liver that promotes blood-vessel leakage. Intellia, headquartered in Cambridge, Massachusetts, has applied for approval of lonvo-z to the US Food and Drug Administration (FDA), which is expected to issue a decision by 10 March. If approved, the therapy will become the world's first CRISPR medicine to be delivered directly inside the body, or in vivo, John Leonard, Intellia's chief executive, told investors on 12 November. Currently, the only CRISPR therapy approved by the FDA is performed on cells that have first been removed from the body and are subsequently re-infused....

The milestone treatment is part of a clinical trial of prime editing, a technique that can alter DNA with more precision than standard CRISPR'Cas9 methods can. In this case, prime editing was used to treat a condition called Wilson disease by rewriting DNA in liver cells, to erase a mutation that prevents them from eliminating excess copper. Prime Medicine, a biotechnology company in Cambridge, Massachusetts, announced that it had treated the first person in its trial earlier this month. It expects to announce the first results from the study next year. 'I never dreamed that in my lifetime I would see this applied to my rare disease,' says Rhonda Rowland, president of the Wilson Disease Association in Atlanta, Georgia. 'It's going to be a really exciting year.' In launching the trial, Prime Medicine joins a host of companies aiming to engineer cells directly in the body, rather than using the standard method of extracting a person's cells, editing their DNA in a laboratory and then reinjecting the altered cells. Rewriting DNA in the body, or in vivo, is appealing because manufacturing and administering the treatment might be much cheaper and faster than making a bespoke therapy for each person from their own cells....

Regardless of the answer, artificial intelligence titan Anthropic has launched a biology 'wet lab' where human scientists and AI agents will work together to design and conduct experiments. This week, the company announced one of the team's first finds: a peculiar pattern of DNA in the genomes of several giant viruses. Similar patterns are found in DNA encoding the CRISPR immune systems of some microbes. Scientists have developed those microbial systems into powerful genome-editing tools. But researchers at Anthropic, which is headquartered in San Francisco, California, have not yet determined what the newfound viral sequences do, and thus whether their function is similar to that of the parallel sequences in microbial CRISPR systems. Even so, the finding, posted online on the alphaXiv platform, offers a first glimpse into Anthropic's new life sciences research group and its associated lab, as well as how its AI tools could be used to mine petabytes of genomic data in search of novel molecular tools. The company announced the new lab and released the preprint on 23 September. The preprint has not yet been peer reviewed....

The DNA-shredding approach, reported in two papers in Nature1,2, could provide a way for researchers to kill cancer cells that express 'undruggable' mutant proteins that have been difficult to target using conventional medicines. 'It's a molecular kill switch that recognizes a particular RNA,' says Yang Liu, a molecular biologist at the University of Utah School of Medicine in Salt Lake City, and an author of one of the papers. 'This is basically a programmable chemotherapy.' A therapy that uses this approach to target head and neck cancers caused by human papillomavirus (HPV) is already in early development at Akribion Therapeutics, a biotechnology company in Zwingenberg, Germany. The goal is to produce the first clinical-trial data by 2030, says company co-founder Paul Scholz, who is head of research and development at Akribion and a co-author of one of the papers. CRISPR systems occur naturally in bacteria and other microorganisms, in which they act as a protective immune mechanism. Some CRISPR systems use RNAs that direct CRISPR-associated (Cas) enzymes to target stretches of DNA in viruses and other invaders. The Cas enzyme then cuts the DNA, destroying the interloper. For more than a decade, researchers have harnessed and modified such systems to edit genomes, creating their own guide RNAs to direct the Cas enzymes to a desired site for editing....