CTX110 is a donor-derived, gene-edited, allogeneic CAR T investigational therapy that targets B-lymphocyte antigen CD19. It is the subject of the ongoing CARBON clinical trial, a phase I, single-arm, multicenter, open-label clinical trial designed to assess safety and efficacy, according to the company.

The RMAT designation expedites the drug development and review processes for promising cell therapies that are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition that has unmet medical or therapeutic need. Similar to the FDA's Breakthrough Therapy designation, the RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, potential accelerated approval and post-approval requirements, as well as potential priority review of the biologics license application.

CRISPR Therapeutics develops genetically engineered therapies for serious diseases in the fields of hemoglobinopathies, oncology, regenerative medicine, and rare diseases, using its proprietary CRISPR-Cas9 gene-editing platform. CRISPR Therapeutics has partnerships with Bayer, Vertex Pharmaceuticals, and ViaCyte, according to CRSPR Therapeutics. CRISPR Therapeutics' headquarters is in Zug, Switzerland, with its U.S. subsidiary and R&D operations based in Cambridge, MA.