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 Ionis 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.
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.