After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies, ...
A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing mutations. The technology could advance treatments for cystic fibrosis and ...
Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...
RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...
TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable ...
The promise of genome editing to help understand human diseases and create new therapies is vast, but technological limitations have limited advancement of the field. While existing editing ...
DALLAS--(BUSINESS WIRE)--In an extraordinary achievement of advanced multiplexed genome engineering, Colossal Biosciences announces the birth of the Colossal Woolly Mouse—mice engineered to express ...
A Yale research team has designed a system to modify, or edit, multiple genes in the genome simultaneously, while also minimizing unintended effects. The gene-editing "toolbox" provides a ...
Artificial intelligence (AI) is known for enabling deeper insights into drug development, identifying patterns and molecules that may otherwise go unnoticed. Now it is poised to make similar ...
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...
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