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Biom Therapeutics Receives Orphan Drug Status for BIO017 from the U.S. FDA for the treatment of Angelman Syndrome
– There is currently no effective treatment for the 25,000 children diagnosed in the U.S. with Angelman syndrome, a rare pediatric epilepsy. BIO017 showed efficacy in preclinical Angelman animal models and preliminary clinical studies.
Biom Therapeutics, a clinical stage biotech company, announced today it has received orphan drug designation status for BIO017—which is designed to treat Angelman syndrome—from the U.S. FDA’s Office of Orphan Product Development. Biom anticipates commencing clinical trials for BIO017 in Q4 of 2021, following submission of an Investigational New Drug (IND) application with the FDA in Q3 of this year. Read More