TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)--Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883, CEO: Haru Morita), a leading company pioneering treatments for rare genetic diseases using its proprietary CRISPR-based epigenome editing technology, CRISPR-GNDM®, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD)..

东京和沃尔瑟姆，马萨诸塞州。-（商业新闻短讯）--Modalis Therapeutics Corporation（东京证券交易所：4883，首席执行官：Haru Morita），一家领先的公司，使用其专有的基于CRISPR的表观基因组编辑技术CRISPR-GNDM®，率先治疗罕见遗传疾病，今天宣布美国食品和药物管理局（FDA）已将罕见儿科疾病（RPD）命名为MDL-101，MDL-101是一种用于治疗先天性肌营养不良症1a型（LAMA2-CMD）的新型精密药物。。

The FDA grants RPD designations to treatments for serious and life-threatening diseases that primarily affect children under 18 years old and affect fewer than 200,000 people in the U.S. If a product with RPD designation receives marketing approval, the company may be awarded a Priority Review Voucher (PRV), allowing for faster FDA review of another product.

FDA 授予 RPD 称号，以治疗主要影响 18 岁以下儿童且影响美国不到 200,000 人的严重和危及生命的疾病。如果具有 RPD 名称的产品获得上市批准，该公司可能会获得优先审评券 （PRV），从而可以更快地对其他产品进行 FDA 审查。

Modalis has also submitted an Orphan Drug application for MDL-101, which is currently under separate review by the FDA..

Modalis还提交了MDL-101的孤儿药申请，目前FDA正在对其进行单独审查。。

“We are pleased that the FDA has recognized our development efforts for the rare disease and granted us RPD designation. We have received many requests for our efforts from children and families around the world suffering from this disease for which there is currently no treatment, and we feel a mission to respond to the expectations of patients who are eagerly awaiting the start of clinical trials as soon as possible,” said Haru Morita, CEO of Modalis.

Modalis首席执行官Haru Morita说：“我们很高兴FDA认可了我们对这种罕见疾病的开发工作，并授予了我们RPD的称号。我们收到了世界各地患有这种疾病的儿童和家庭的许多要求，目前尚无治疗方法，我们感到有一项使命，即满足渴望尽快开始临床试验的患者的期望。”。

“This designation from the FDA is proof that our efforts are rational and achieving results, and we hope to further accelerate the development of this world's first product using the cutting-edge technology.”.

“FDA的这一指定证明了我们的努力是合理的，并取得了成果，我们希望进一步加速使用尖端技术开发世界上第一种产品。”。

LAMA2-CMD is a severe, early onset congenital muscular dystrophy caused by the absence of the LAMA2 protein, which is made up of more than 3,000 amino acids. Because it cannot be loaded onto an AAV vector, it is thought that the conventional approach to gene therapy development is difficult. For this reason, there is currently no treatment, including gene therapy, that addresses the underlying cause of LAMA2-CMD..

LAMA2-CMD是一种严重的早发性先天性肌营养不良症，由不存在由3000多个氨基酸组成的LAMA2蛋白引起。由于它不能加载到AAV载体上，因此认为传统的基因治疗开发方法很困难。因此，目前还没有包括基因治疗在内的治疗方法可以解决LAMA2-CMD的根本原因。。

Modalis’ proprietary CRISPR-GNDM® technology allows for precise modulation of gene expression without introducing double-strand DNA breaks. MDL-101, Modalis’ lead candidate for LAMA2-CMD, represents a first-in-class therapeutic that aims to address the unmet need by inducing expression of the sister gene LAMA1 in muscle tissues, thereby compensating for the deficient function of LAMA2..

Modalis专有的CRISPR-GNDM®技术允许在不引入双链DNA断裂的情况下精确调节基因表达。MDL-101是Modalis LAMA2-CMD的主要候选药物，是一种一流的治疗药物，旨在通过诱导肌肉组织中姐妹基因LAMA1的表达来解决未满足的需求，从而弥补LAMA2的功能缺陷。。

Guided by its mission, 'Every Life Deserves Attention,' Modalis is dedicated to bringing life-changing treatments to patients suffering from diseases for which no cure currently exists.

在“每一个生命都值得关注”的使命指导下，Modalis致力于为患有目前无法治愈的疾病的患者提供改变生命的治疗。

About MDL-101

关于MDL-101

MDL-101 is an experimental, epigenetic editing therapy that is being developed for the treatment of LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD). MDL-101 is comprised of a guide nucleotide targeting LAMA1 gene, a highly homologous sister gene of the disease-causing gene LAMA2, enzyme-null Cas9 (dCas9) fused with trans-activating domain driven by a muscle-specific promoter and coded in a muscle-specific AAV vector.

MDL-101是一种实验性的表观遗传编辑疗法，正在开发用于治疗LAMA2先天性肌营养不良症（LAMA2-CMD）。MDL-101由靶向LAMA1基因的指导核苷酸，致病基因LAMA2的高度同源姐妹基因，与肌肉特异性启动子驱动的反式激活结构域融合的酶无效Cas9（dCas9）组成，并编码在肌肉特异性AAV载体中。

MDL-101 upregulates LAMA1 gene products in patients’ muscle tissue to compensate for loss-of-function caused by mutation of LAMA2, and therefore has the potential to provide a one-time, durable treatment to benefit people living with LAMA2-CMD..

MDL-101上调患者肌肉组织中的LAMA1基因产物，以补偿LAMA2突变引起的功能丧失，因此有可能提供一次性，持久的治疗方法，使LAMA2-CMD患者受益。。

About Modalis:

关于Modalis：

Modalis Therapeutics develops precision genetic medicines using epigenome editing technology. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM® technology which enables the gene/locus-specific modulation of gene expression or epigenome editing without the need for DNA cleavage or altering DNA sequence.

Modalis Therapeutics使用表观基因组编辑技术开发精确的遗传药物。Modalis正在使用其专有的CRISPR-GNDM®技术寻求孤儿遗传疾病的治疗方法，该技术可以实现基因表达或表观基因组编辑的基因/基因座特异性调节，而无需DNA切割或改变DNA序列。

Headquartered in Tokyo with all research and development operations in Waltham Massachusetts, the company is listed on the Tokyo Stock Exchange’s Growth market. For additional information, visit www.modalistx.com/en/..

该公司总部位于东京，所有研发业务均位于马萨诸塞州沃尔瑟姆，在东京证券交易所的成长型市场上市。有关更多信息，请访问www.modalistx.com/en/。。