CRISPR-associated transposases (CASTs) are an attractive candidate for genome editing applications, as they enable the insertion of large DNA cargoes without creating double-strand breaks. However, CAST systems have shown limited activity in human cells. In a paper published in Science, Witte et al. apply phage-assisted continuous evolution (PACE) to direct the rapid evolution of new CAST variants

Base and prime editors promise to correct nearly all known pathogenic variants, but their therapeutic development is restricted to a handful of recurrent mutations owing to the high cost of bringing gene therapies to market. Writing in the New England Journal of Medicine, Musunuru et al. report on the rapid development of a customized base-editing therapy, provided to an infant born with a rare disease

Although spatial RNA organization is central to cellular functions and disease mechanisms, its functional consequences remain poorly understood owing to a lack of tools for manipulating RNA localization within cells. Writing in Nature, Han et al. introduce CRISPR-mediated transcriptome organization (CRISPR-TO), a method that uses the RNA-guiding properties of nuclease-dead dCas13 to transport endogenous

Recent patents relating to the production, culture and use of organoids.

Recent moves of note in and around the biotech and pharma industries.

To those considering a change in careers from academia to the pharmaceutical industry, here are the keys to a successful transition.

The UK government is close to approving rules for precision-bred plants in England. In May, Parliament published draft legislation that will enable a new regulatory system, as set out in the Genetic Technology (Precision Breeding) Act 2023.

Pigs genetically modified to resist a devastating virus are now approved for human consumption in the United States. British animal genetics company Genus, based in Basingstoke, announced in April that the US Food and Drug Administration (FDA) had approved its gene-edited pigs after a lengthy review through the agency’s Investigational New Drug regulatory pathway.

A new law proposed by the Federal Council would ease restrictions on plants developed through new genomic techniques such as CRISPR. Currently in Switzerland, such plants are treated similarly to those developed through conventional genetic engineering, including an ongoing moratorium on cultivation. If passed, the Breeding Technologies Act would enact a risk-based authorization system that reflects

The University of California and the University of Vienna have convinced a US appeals court to revive their bid for patent rights to the CRISPR–Cas9 gene-editing technology created by their scientists Jennifer Doudna and Emmanuelle Charpentier. The case was sent back to the Patent Office’s Patent Trial and Appeal Board for reconsideration after the court found fault with a previous patent tribunal’s

The gutting of US research funding represents a once-in-a-generation opportunity for Europe to boost world-class research deprioritized by the Trump administration.

The first cancer vaccines matched to a person’s unique tumor neoantigens are forging ahead, with expectations running high as the field awaits results from the first pivotal trial.

More precise base editors make mitochondrial DNA editing efficient enough to model disease and correct pathogenic mutations in rodents, but they are slow to move into clinical trials.

Merck’s new antibody clesrovimab and other long-lasting shots promise to both expand and simplify care of newborns across the world to combat respiratory syncytial virus.

Site-specific retroelements, such as R2 retrotransposons, have potential as programmable genome-editing systems, but so far have only been used for targeted insertion at genomic safe-harbor loci. In a paper now published in Nature, Fell et al. profile the evolution of site-specific retrotransposon families to gain insight into target site preferences, which they then used to engineer reprogramming

Huanglongbing is a devastating bacterial disease of citrus trees with no known cure. An emerging strategy to provide resistance to plant pathogens is disrupting genes that increase disease susceptibility. A paper in Science by Zhao et al. uses this approach in citrus trees, and identifies a regulatory circuit that can be modulated to alleviate Huanglongbing disease symptoms. Huanglongbing can infect

Recent patents relating to systems, methods and compositions for bioprinting.

Regulatory DNA sequences orchestrate cell-type-specific gene expression by facilitating transcription factor binding, yet their precise effects and reprogrammability remain challenging to delineate. Now, in Cell, Martyn et al. develop a method called variant effects from flow-sorting experiments with CRISPR targeting screens (Variant-EFFECTS), which measures the quantitative effects of changes to regulatory

Polybion creates this next-generation material by feeding a mango fruit waste slurry to Acetobacter bacteria genetically modified to speed up their metabolism. The bacteria feed on the sugar and turn it into cellulose to form a gel-like mat on the surface of the fermentation tank. Bacteria employ this strategy to survive in harsh environments. “It’s a shelter they create [against] the radiation of

Recent moves of note in and around the biotech and pharma industries.

Big pharma and investors are piling into the precision radiation space as the next generation of increasingly potent targeted α-particle therapies promises to destroy cancerous cells with minimum damage to healthy tissues.

In late March, the US Food and Drug Administration’s (FDA) director of the Center for Biologics Evaluation and Research, Peter Marks, resigned under pressure from Health and Human Services Secretary Robert F. Kennedy. In his resignation letter Marks said he had been asked to hand in data on measles vaccine-related deaths and brain swelling that do not exist.

Brazil is the first country in Latin America to produce its own cut-price chimeric antigen receptor (CAR)-T cell therapies. Now, Caring Cross, a US-based non-profit focused on making advanced therapies affordable, plans to expands Brazil’s model to Turkey and the Middle East.

As part of its Dare2eraD TB initiative to combat the challenge of drug-resistant tuberculosis, Indian researchers sequence the genomes of 10,000 Mycobacterium tuberculosis isolates. Sequencing will aid in personalizing antibiotic prescription, allowing faster, more effective treatment. India has set the goal of eliminating TB by 2025, five years ahead of the World Health Organization’s global target

The US Food and Drug Administration plans to introduce computational modeling and other innovative methods to replace animal testing in preclinical drug safety studies, according to a roadmap released in April. By encouraging drug sponsors to embrace organ-on-a chip, in silico modeling, organoid and other in vitro assays, the FDA aims to reduce animal testing to the extent that it becomes “the exception

Correction to: Nature Biotechnology https://doi.org/10.1038/s41587-024-02447-1, published online 18 October 2024.