Transthyretin amyloidosis (ATTR) is a rare systemic disorder caused by amyloid deposition of misfolded transthyretin (TTR) protein. TTR is a 55 kDa protein consisting of 127 amino acids, mainly present in plasma and cerebrospinal fluid (CSF). It transports thyroxine (T4)…
Duchenne muscular dystrophy (DMD) is a rare X-linked recessive genetic disorder caused by mutations in the DMD gene, leading to the loss of dystrophin protein. Dystrophin is critical for maintaining the stability of muscle cell membranes. The incidence of DMD…
Deafness is one of the most common congenital birth defects. Worldwide, the incidence of hearing loss in newborns is 1.86‰, and more than 60% of deafness cases are attributed to genetic factors. Hereditary deafness is classified into two categories: Syndromic…
On June 9, 2025, Xinya Gene announced that GEN6050X, its proprietary in vivo base-editing therapeutic candidate, had obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for DMD treatment. This recognition marks a landmark milestone for…
