Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) [Yahoo! Financ...
Beam Therapeutics Inc. (BEAM)
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Source: Yahoo! Finance
medicines through base editing, today announced that the United States (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 (AATD). 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. “Just weeks after the clearance of our U.S. investigational new drug application, we are pleased to report that the FDA has now granted RMAT designation to BEAM-302, recognizing its potential as a transformative, one-time treatment for patients living with AATD,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “This designation
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BEAM
News
- BEACON Trial Data Makes Beam Therapeutics (BEAM) an Attractive Buy [Yahoo! Finance]Yahoo! Finance
- Beam Therapeutics to Present at 44th Annual J.P. Morgan Healthcare ConferenceGlobeNewswire
- Beyond Traditional Drugs: Novel Platforms Targeting Hard-to-Treat ConditionsPR Newswire
- Beam Therapeutics (BEAM) Is Up 6.9% After New BEACON Trial Data Spotlight Base-Editing Potential [Yahoo! Finance]Yahoo! Finance
- Beam Therapeutics Reports Updated Data from BEACON Phase 1/2 Trial of ristoglogene autogetemcel (risto-cel) Highlighting Durable, Differentiated Profile in Sickle Cell Disease (SCD) at American Society of Hematology (ASH) Annual MeetingGlobeNewswire
BEAM
Earnings
- 11/4/25 - Miss
BEAM
Sec Filings
- 12/11/25 - Form 8-K
- 11/5/25 - Form SCHEDULE
- 11/4/25 - Form 10-Q
- BEAM's page on the SEC website