Transthyretin amyloidosis (ATTR) is a rare disease caused by the abnormal accumulation of amyloid deposits composed of misfolded transthyretin (TTR) proteins in multiple organs of the body, including the retina, the vitreous, the kidney, and the brain.

Transthyretin amyloid polyradiculoneuropathy (ATTR-PN) and transthyretin amyloid cardiomyopathy (ATTR-CM) are prominent subtypes.

GemPharmatech replaces the coding region and regulatory sequences of the murine TTR gene with the corresponding human gene fragments to generate B6-hTTR and B6-hTTR V50M models, which can be used for the study of ATTR  and the in vivo efficacy evaluation of human TTR-targeted gene therapies.