The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with hereditary amyloidosis and polyneuropathy, according to the results of a ...

Despite setbacks and funding cuts — and a quieting of the hype blaring its arrival — multiple CRISPR-based trials are ...

In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...

Intellia Therapeutics NTLA has made meaningful progress with the development of its in vivo programs. The company is currently advancing two late-stage in vivo candidates — lonvo-z (or, NTLA-2002) for ...

A one-time gene-editing therapy, exagamglogene autotemcel (exa-cel), has demonstrated sustained and clinically meaningful ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct gene-editing experiments. The device, CRISPR-GPT, is an artificial intelligence lab ...

It's not the size of your accelerator, it's how you use it Gene editing startup Metagenomi has tapped AWS's Inferentia 2 ...

MIT scientists have found a way to make gene editing far safer and more accurate — a breakthrough that could reshape how we ...

In early October 2025, Intellia Therapeutics presented new clinical results at the European Society of Gene & Cell Therapy Congress, highlighting long-term safety and efficacy data for its ...

Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile--The AATD ...