WASHINGTON (dpa-AFX) - Beam Therapeutics Inc. (BEAM) announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency.
Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant unmet need for effective therapies that can treat the entire spectrum of disease.
RMAT designation provides opportunities for early interactions with the FDA to discuss potential surrogate or intermediate endpoints to support accelerated approval, organizational commitment from senior staff at the agency, opportunities to participate in novel review and development programs, and the potential for a rolling review and priority review of a products future biologics license application.
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