RTC14

RTC14 is a small molecule read-through compound (RTC) identified through high-throughput screening for its ability to suppress nonsense mutations. It functions by inducing ribosomal read-through of premature termination codons (PTCs) in mRNA, thereby allowing the translation of full-length, functional proteins. RTC14 has been investigated for the treatment of genetic disorders caused by nonsense mutations, including Duchenne muscular dystrophy (DMD) and Ataxia-telangiectasia (A-T). In preclinical studies, RTC14 demonstrated the ability to restore dystrophin expression in the *mdx* mouse model and ATM protein function in A-T cell lines. However, in vivo studies have indicated that RTC14 may have lower potency compared to its related compound, RTC13, particularly when administered systemically.