Ractigen Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, a novel small interfering RNA (siRNA) therapy.RAG-21 is an RNA interference therapy designed to lower FUS mRNA levels, preventing the production of toxic proteins linked to FUS-ALS, a severe form of amyotrophic lateral sclerosis.

Ractigen Therapeutics宣布，美国食品和药物管理局（FDA）已将孤儿药命名（ODD）授予RAG-21，这是一种新型的小干扰RNA（siRNA）疗法。。

Delivered via the SCAD™ platform, it ensures precise and sustained gene knockdown within the central nervous system.ALS is a neurodegenerative disease with no cure, often causing death within 2–5 years of diagnosis. FUS mutations lead to aggressive ALS, characterised by early onset, rapid progression, and toxic protein build-up in neurons.

通过SCAD™平台提供，它确保了中枢神经系统内精确而持续的基因敲除。ALS是一种无法治愈的神经退行性疾病，通常在诊断后2-5年内导致死亡。FUS突变导致侵袭性ALS，其特征是早期发作，快速进展和神经元中有毒蛋白质的积累。

Existing treatments offer limited benefits, but RNA-based therapies like RAG-21 target the root cause, providing new hope.This therapy is designed to target the FUS gene in amyotrophic lateral sclerosis (ALS), a rare and severe neurological condition.RAG-21 addresses FUS-ALS, a particularly aggressive form of the disease.

现有的治疗方法提供的益处有限，但基于RNA的疗法（如RAG-21）针对根本原因，提供了新的希望。该疗法旨在针对肌萎缩侧索硬化症（ALS）中的FUS基因，这是一种罕见且严重的神经系统疾病。RAG-21解决了FUS-ALS，一种特别具有侵略性的疾病。

Using RNA interference (RNAi) technology, the therapy reduces levels of FUS protein, which is linked to motor neuron damage in this ALS subtype. Preclinical research has shown that RAG-21 effectively mitigates the harmful effects of FUS mis-localisation and aggregation, offering a potential solution where no curative treatments currently exist.The Orphan Drug Designation programme recognises therapies targeting rare conditions affecting fewer than 200,000 individuals in the United States.

使用RNA干扰（RNAi）技术，该疗法降低了FUS蛋白的水平，FUS蛋白与这种ALS亚型的运动神经元损伤有关。临床前研究表明，RAG-21有效减轻了FUS错误定位和聚集的有害影响，为目前尚无治疗方法的情况下提供了潜在的解决方案。孤儿药物指定计划认可针对影响美国不到20万人的罕见疾病的疗法。

It provides benefits to developers, such as tax credits for clinical trials, seven years of market exclusivity, and a waiver of certain regulatory fees.RAG-21 represents the company’s second orphan designation for ALS therapies, following an earlier approval for RAG-17 ta.

它为开发商提供了好处，例如临床试验的税收抵免，七年的市场排他性以及某些监管费用的豁免。RAG-21代表了该公司在早期批准RAG-17 ta后，针对ALS治疗的第二个孤儿指定。