The company uses a mobile manufacturing system to produce autologous stem cell therapy for patients with dyskeratosis congenita and other telomere biology disorders.
An FDA rare pediatric disease designation recently pushed extra wind into the sails of Elixirgen Therapeutics, an RNA cell therapy company.
With a Phase 1/2 trial to treat dyskeratosis congenita (DC) and other telomere biology disorders underway at Cincinnati Children’s Hospital, the Baltimore-based company aspires to rewrite some of the most patient-taxing parts of stem cell therapy — including extended manufacturing windows and unpleasant patient preconditioning, which is typical of cell therapy.