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# Beam Gets Regenerative Medicine Status for Gene Therapy
- URL: https://www.fdaweb.com/beam-gets-regenerative-medicine-status-for-gene-therapy/
- Published: 2025-05-12T12:00:00.000Z
- Updated: 2026-09-14T15:11:30.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5159163

FDA has granted Beam Therapeutics a Regenerative Medicine Advanced Therapy designation for BEAM-302, a liver-targeting gene therapy designed to correct a disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). The inherited genetic disorder affects the lungs and/or liver, leading to early-onset emphysema and liver disease, according to the company.

The designation is based on initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 that were previously [reported](https://www.globenewswire.com/Tracker?data=DA7vCfMfeWFXeIxzmWuCQbq57-hOQDq3lYcM01eYzAB2DDZYKNsjZwr%5Fiq5%5FTDmR93QBOR24G6ICtFMNBz4iR0AAgLewn-IQvVD3cuv0TM1xUJyrh4OIAmqse6MMY42kX6v-%5FEFDrgneDyjxRqX56dJMQ7rYyGNbYI9Q0FsN60LtxsUV4SKfM6WCVgfyqPQg1icVyi4ZHOANTZ4Tk3sS5w6sgMl7aBvX-wWLvbIdaeg=&ref=fdaweb.com) in March, establishing clinical proof of concept as a potential treatment for AATD and *in vivo* base editing, the company says. “Preliminary results from the first three single-ascending dose cohorts in Part A of the study demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort,” it adds.