Resvita Bio Inc.’s RVB-003 has been awarded orphan drug designation by the FDA for the skin disorder Netherton syndrome. RVB-003 was previously granted rare pediatric disease designation.

Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.

Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits.

Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits.

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).

Ariceum Therapeutics GmbH’s radiopharmaceutical 225Ac-SSO110 ([225Ac]satoreotide tetraxetran) has been awarded orphan drug designation by the FDA for the treatment of small-cell lung cancer (SCLC). [225Ac]Satoreotide is a first-in-class Actinium-labeled somatostatin SST2 receptor antagonist.

Seed Therapeutics Inc.’s ST-01156 has been awarded orphan drug designation for the treatment of Ewing sarcoma as well as rare pediatric disease designation by the FDA.

Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.