This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.

The newfound DNA-cutting enzyme, called Fanzor, can be programmed to edit the human genome and could prove easier to deliver to cells than current CRISPR tools.

The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.

Here is a roundup of news and research for the week of 26 June.

This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.

The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.

The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.

This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.

The approach removes methyl tags from the gene and shields it from other silencing factors without changing the gene itself, raising hopes for a new treatment.

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.