Beam Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302.

Beam Therapeutics宣布，美国食品药品监督管理局（FDA）已授予BEAM-302再生医学先进疗法（RMAT）认定。

AATD is caused by mutations in the SERPINA1 gene, most commonly the PiZ variant, which leads to low circulating levels of functional AAT and accumulation of misfolded protein in the liver. This results in lung damage due to unregulated neutrophil elastase activity and liver damage due to toxic build-up of the defective protein. .

AATD 是由 SERPINA1 基因突变引起的，最常见的 PiZ 变异型会导致功能性 AAT 的循环水平降低，并在肝脏中积累错误折叠的蛋白质。这会引起肺部损伤，由于中性粒细胞弹性蛋白酶活性未受抑制，以及肝脏损伤，由于缺陷蛋白质的毒性积累。

BEAM-302 aims to offer a one-time treatment by directly correcting the underlying genetic mutation. By restoring production of functional AAT protein and reducing harmful protein aggregates, the therapy could address both lung and liver complications of AATD. Unlike current therapies, which rely on protein replacement and do not prevent disease progression, BEAM-302 may offer a more comprehensive and lasting solution..

BEAM-302 旨在通过直接纠正潜在的基因突变提供一次性治疗。通过恢复功能性 AAT 蛋白的生产并减少有害的蛋白聚集体，该疗法可以同时应对 AATD 的肺部和肝脏并发症。与当前依赖蛋白替代且无法阻止疾病进展的疗法不同，BEAM-302 可能提供更全面和持久的解决方案。

Current treatment options are limited and non-curative, highlighting the need for innovative approaches such as BEAM-302.

目前的治疗选择有限且无法治愈，突显了对 BEAM-302 等创新方法的需求。

This investigational therapy is aimed at treating alpha-1 antitrypsin deficiency (AATD), a genetic condition affecting the lungs and liver.

这种研究性疗法旨在治疗α-1抗胰蛋白酶缺乏症（AATD），这是一种影响肺部和肝脏的遗传性疾病。

BEAM-302 is a liver-targeted lipid nanoparticle (LNP) formulation containing a guide RNA and an mRNA that encodes a base editor. It is designed to correct the PiZ mutation in patients with AATD. Individuals with two copies of this mutation (PiZZ genotype) represent the majority of those suffering from severe forms of the disease..

BEAM-302是一种靶向肝脏的脂质纳米颗粒（LNP）制剂，包含一个引导RNA和一个编码碱基编辑器的mRNA。它旨在纠正AATD患者中的PiZ突变。携带该突变两个拷贝（PiZZ基因型）的个体占患有严重形式疾病的大多数。

RMAT designation is intended to support the development of regenerative therapies for serious or life-threatening diseases where there is an unmet medical need. It offers benefits such as early and frequent interactions with the FDA, potential for accelerated approval based on surrogate endpoints, rolling review of regulatory submissions, and priority review eligibility..

RMAT指定旨在支持为存在未满足医疗需求的严重或危及生命的疾病开发再生疗法。它提供的好处包括与FDA早期和频繁的互动、基于替代终点的加速批准潜力、监管提交的滚动审查以及优先审查资格。

The company also plans to begin enrolling AATD patients with mild to moderate liver disease in the second half of 2025.

公司还计划在 2025 年下半年开始招募轻度至中度肝病的 AATD 患者。