By Roshan Fernandez
Beam Therapeutics said the Food and Drug Administration has granted an orphan drug designation to its treatment for a genetic disorder that affects the lungs and or liver.
The Cambridge, Mass.-based biotechnology company's drug, BEAM-302, is a liver-targeting lipid nanoparticle formulation that is designed to correct for a mutation in patients with alpha-1 antitrypsin deficiency. AATD, an inherited genetic disorder, leads to early onset emphysema and liver disease.
The FDA's orphan drug designation is to support the development and evaluation of treatments for rare diseases that affect fewer than 200,000 people. It includes benefits such as exemption from user fees and seven years of market exclusivity after approval.
"Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options," said Beam Therapeutics' President Giuseppe Ciaramella.
The company said it reported Phase 1 and 2 trial data in March.
Write to Roshan Fernandez at roshan.fernandez@wsj.com
(END) Dow Jones Newswires
May 29, 2025 17:51 ET (21:51 GMT)
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