Intellia Therapeutics and Regeneron are developing NTLA-2001, a therapeutic that is designed to target liver cells and disable a gene that is responsible for the production of misfolded transthyretin (TTR) protein…more [Intellia Therapeutics]
https://www.genengnews.com/insights/crispr-based-therapeutics-blaze-an-in-vivo-path-to-the-clinic/