Intellia Therapeutics ' CRISPR - based treatment could help treat rare progressive condition : amyloidosis ( ATTR ) . The patient sees abnormal transthyretin ( TTR ) proteins build up in a patient 's organs and tissues , potentially affecting their nervous system and heart . Working at a European Society of Gene and Brain Therapy trials , Intellia demonstrated that its anti - Cas9 treatment may combat the effects of ATTR . Working with Regeneron , the company conducted primate testing showed a " high correlation " between liver editing and a reduction in TTR .
Intellia Therapeutics' CRISPR-based treatment could help treat rare progressive condition transthyretin amyloidosis (ATTR). The disease sees misfolding transthyretin (TTR) proteins build up in a patient's organs and tissues, potentially affecting their nervous system and heart. Speaking at a European Society of Gene and Cell Therapy meeting, Intellia demonstrated that its CRISPR-Cas9 treatment may combat the effects of ATTR. Working with Regeneron, the company said primate testing showed a "high correlation" between liver editing and a reduction in TTR.