A Next-Generation RNA Therapy Targets Telomere Disorders

Telomere biology disorders are a set of rare genetic diseases caused by a shortening of the protective DNA that appears at the ends of chromosomes. Between 80 and 90 percent of people with these conditions will suffer from bone marrow failure by age 30, the leading cause of mortality for people with these disorders. The only available treatment today is transplantation of donor human stem cells. Elixirgen is developing what it calls self-replicating RNA therapies to treat telomere biology disorders and other conditions. We spoke to Akihiro Ko, CEO Elixirgen Therapeutics, about telomere biology disorders, the company’s self-replicating RNA therapies, and the advantages this new therapeutic approach offers over more traditional mRNA therapies.