Neurenati Therapeutics Inc.’s NEU-001 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Hirschsprung disease.

Ensoma Inc.’s lead program, EN-374, has been granted orphan drug and rare pediatric disease designations by the FDA for the treatment of X-linked chronic granulomatous disease. Ensoma anticipates filing an IND application for EN-374 in the first half of this year.

Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).