. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268, Sanofi's one-time AAV gene therapy for the treatment of non-congenital (juvenile and adult onset) DM1 myotonic dystrophy type 1 (DM1). This designation process aims to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need.

美国食品药品监督管理局（FDA）已授予赛诺菲公司一次性AAV基因疗法SAR446268快速通道资格，用于治疗非先天性（青少年和成人起病）的1型肌强直性营养不良（DM1）。该指定程序旨在促进用于治疗严重疾病并满足未满足医疗需求的药物的开发和加快审查进程。

The FDA created this process to help deliver important new medicine to patients earlier and it covers a broad range of serious illnesses..

FDA创建了这一流程，以帮助患者更早获得重要的新药，且该流程涵盖多种严重疾病。

SAR446268 employs a vectorized RNA interference (RNAi) approach to silence

SAR446268 采用矢量化的 RNA 干扰 (RNAi) 方法进行沉默处理

DMPK

DMPK

expression through a single administration. By reducing

通过单一管理表达。通过减少

DMPK

药物代谢动力学

transcripts, the gene therapy aims to eliminate the abnormal and toxic RNA foci responsible for splicing defects in muscle tissue, thereby restoring normal splicing and improving muscular function. This approach has the potential to address key symptoms of the disease, including progressive muscle weakness, difficulty relaxing muscles (myotonia), and effects on multiple body systems including heart, lungs, and endocrine functions.

转录本，基因治疗旨在消除导致肌肉组织剪接缺陷的异常和有毒的RNA焦点，从而恢复正常的剪接并改善肌肉功能。这种方法有潜力解决该疾病的关键症状，包括进行性肌肉无力、肌肉松弛困难（肌强直）以及对心脏、肺和内分泌功能等多系统的影响。

SAR446268 is the only investigational therapy in clinical development for this disease, and there are no currently approved therapies for DM1. .

SAR446268 是目前唯一处于临床开发阶段的 DM1 疾病研究性疗法，目前尚无获批的 DM1 治疗方法。

SAR446268 is currently under investigation in a first-in-human, phase 1-2 study to evaluate the safety, tolerability, and efficacy (clinical study identifier:

SAR446268目前正在一项首次人体、1-2期研究中进行评估，以评价其安全性、耐受性和有效性（临床研究标识符：

NCT06844214

NCT06844214

). The first patient is planned for enrolment in late 2025. Sanofi has already been granted orphan designations for SAR446268 in both the US (July 2024) and EU (October 2024).

). 首位患者计划于2025年底入组。赛诺菲已在美国（2024年7月）和欧盟（2024年10月）获得SAR446268的孤儿药资格认定。

About myotonic dystrophy type 1

关于1型肌强直性营养不良

Myotonic dystrophy type 1, also known as Steinert's disease, is an inherited, progressive, rare disorder affecting approximately 1 in 2,300 people worldwide. The condition is caused by mutations in the DMPK gene and is characterized by progressive muscle weakness, difficulty relaxing muscles (myotonia), and effects on multiple body systems including heart, lungs, and endocrine functions.

1型肌强直性营养不良，也被称为Steinert病，是一种遗传性、进行性、罕见的疾病，全球大约每2300人中就有1人受其影响。该病由DMPK基因突变引起，其特征为进行性肌肉无力、肌肉松弛困难（肌强直），并对包括心脏、肺和内分泌功能在内的多个身体系统产生影响。

DM1 can manifest at any age with varying severity, from mild adult cases to severe congenital forms, and has a profound impact on quality of life, affecting patients' ability to perform daily activities, maintain independence, and in severe cases, sustain vital functions. There are no currently approved treatments for DM1..

DM1可以在任何年龄出现，严重程度各异，从轻度成人病例到重度先天形式不等，并对生活质量产生深远影响，影响患者执行日常活动、保持独立性以及在严重情况下维持生命功能的能力。目前尚无获批的DM1治疗方法。

About Sanofi

关于赛诺菲

Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time..

赛诺菲是一家以研发为驱动、以人工智能为助力的生物制药公司，致力于改善人们的生活并实现引人注目的增长。我们凭借对免疫系统的深刻理解，研发药物和疫苗，为全球数百万人提供治疗与保护，同时通过创新的研发管线，有望使数百万人进一步受益。我们的团队秉持一个目标：追逐科学奇迹以改善人们的生活；这激励我们推动进步，并通过应对当今最紧迫的医疗、环境和社会挑战，为我们服务的人群和社区带来积极影响。

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

赛诺菲在 EURONEXT（欧洲证券交易所）上市，代码为 SAN；在 NASDAQ（纳斯达克）上市，代码为 SNY。