ENCell's umbilical cord-derived mesenchymal stem cell therapy candidate EN001 received FDA orphan drug designation for Duchenne muscular dystrophy, marking its second designation after Charcot-Marie-Tooth disease and positioning it as a potential platform therapy for rare neuromuscular diseases.

Long-term follow-up suggests ProTrans may help preserve insulin production, with results varying by dose.

Based on Phase IIa trial data where 85% of patients had resolution of chronic fatigue over 20% with placebo, the company will now offer its cord blood cell therapy outside of the Phase III soon.