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Artan-101 is a preclinical gene therapy candidate developed by ArtanBio (also known as VITARNA). It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a suppressor tRNA designed to recognize and override arginine (CGA) nonsense mutations. These mutations result in premature stop codons that truncate essential proteins, such as the tumor suppressor p53, which are critical for DNA repair, tumor suppression, and neurological health. By facilitating the insertion of arginine at the premature stop site, Artan-101 restores the production of full-length, functional proteins. While Artan-101 demonstrated proof-of-concept in restoring p53 levels in initial studies, ArtanBio has designated Artan-102 as its lead candidate for further development toward clinical trials.
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