Beam Therapeutics (BEAM) announced that the FDA has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle 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. 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.
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Read More on BEAM:
- Beam Therapeutics treatment of alpha-1 antitrypsin deficiency gets orphan status
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