Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia or phenylketonuria.

The cardiomyositis that is a rare adverse effect of mRNA-based COVID vaccines is due to immune cell activity as a result of increased levels of the chemokines CXCL10 and interferon-γ (IFN-γ). Blocking CXCL10 and IFN-γ could prevent muscle cell damage in cell culture, and cardiomyositis in animal models. The findings, reported in the Dec. 10, 2025, issue of Science Translational Medicine, suggest a way of mitigating the risk of cardiomyositis.

Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.

Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy.

The UK Health Security Agency (UKHSA) has identified a new recombinant strain of mpox (formerly monkeypox) that contains elements of clade Ib and clade IIb of the virus, in a traveler who recently returned from Asia. In a paper describing the new strain, the researchers at UKHSA say it is not possible to determine from a single genome how long the recombinant virus has been in circulation, or whether it will have a fitness benefit over currently circulating lineages.

Researchers at the Institut Pasteur have developed a vaccine that spurred the production of autoantibodies to immunoglobulin E antibodies, protecting vaccinated mice from anaphylaxis.

The UK Health Security Agency has identified a new recombinant strain of mpox (formerly monkeypox) that contains elements of clade Ib and clade IIb of the virus, in a traveler who recently returned from Asia.

Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy.

The first phase of the U.K. synthetic human genome project has successfully completed, realizing key steps in chromosome synthesis. The work has demonstrated a multistep method for transfecting mouse stem cells with native human chromosomes, where they are stably maintained and can be manipulated to replace native DNA with synthetic DNA. The engineered chromosomes can then be transferred into a human cell in place of the native chromosomes.

On Dec. 2, 2025, the FDA released draft guidance that could reduce the use of nonhuman primates (NHPs) in preclinical testing of monoclonal antibodies. According to the guidance, which the FDA released for the purpose of soliciting comments, “In general, studies longer than 3 months in nonrodent species (e.g., NHPs, dogs, and mini-pigs) are not warranted to evaluate toxicities … when data from 3-month studies are supplemented with a weight-of-evidence (WoE) risk assessment.”