Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...

Scientists had tried to treat diseases by editing genes since the 1990s, but the methods were cumbersome and didn’t pay off. Then in June 2012, the journal Science published a paper by two future ...

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As far back as 2015, scientists have been warning against using CRISPR-Cas9 technology to modify germline genomes. Indeed, a perspective published that year in Science suggested that otherwise ...

A powerful form of DNA-editing machinery discovered in bacteria might allow us to make much bigger changes to genomes than is currently possible with CRISPR-based techniques. However, it isn’t yet ...

Profluent has staked a claim at the intersection of some of the buzziest terms in biopharma. The protein-focused artificial intelligence startup believes that its large language models can help extend ...

For years, the CRISPR-Cas9 genome technology has been reshaping genetic engineering, a precision tool to transform everything from agriculture to medicine. With its incredible efficiency, this ...