Sarepta Therapeutics Inc. (formerly AVI Biopharma Inc., before that Antivirals Inc.) is developing applications of Morpholino oligos for therapeutics and is pursuing clinical trials with several Morpholinos. In their literature, Morpholino oligos are often referred to as PMOs or Neugenes. Sarepta Therapeutics has received FDA approval for three of its drugs targeting various exons of the human dystrophin gene to treat Duchenne muscular dystrophy: eteplirsen, casimersen, and golodirsen.
Another notable approval is the Morpholino viltolarsen from NS Pharma, also for treatment of Duchenne muscular dystrophy.
Morpholinos from several other companies are also entering clinical trials.
The research program at Gene Tools to develop effective in vivo delivery technology for Morpholino oligos is intended to enhance the efficacy of Morpholino oligos for therapeutic applications as well as to simplify the use of Morpholinos in vivo for fundamental biological research and pharmaceutical target validation.
The research program at Gene Tools to develop effective in vivo delivery technology for Morpholino oligos is intended to enhance the efficacy of Morpholino oligos for therapeutic applications as well as to simplify the use of Morpholinos in vivo for fundamental biological research and pharmaceutical target validation.