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 an Orphan Drug Designation (ODD) 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）已授予MDL-101孤儿药名称（ODD），MDL-101是一种正在开发的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 ODD we have just received is based on the Orphan Disease Act of 1983 and is a system for designating drugs that meet certain conditions, such as having fewer than 200,000 patients in the United States and being particularly in need of medical treatment. This system supports and promotes the development of orphan drugs intending to provide safe, high-quality drugs to the medical community as soon as possible, in light of the situation where research and development of orphan drugs is not progressing sufficiently due to the small number of patients despite the high medical need for such drugs..

我们刚刚收到的ODD是基于1983年的《孤儿疾病法案》，是一个指定符合某些条件的药物的系统，例如在美国只有不到200000名患者，并且特别需要医疗。该系统支持和促进孤儿药的开发，旨在尽快为医学界提供安全，优质的药物，鉴于孤儿药的研究和开发由于患者人数少而进展不足，尽管对此类药物的医疗需求很高。

Haru Morita, CEO and President of Modalis, said, “LAMA2-CMD is a serious, life-threatening rare disease that causes muscle weakness and reduced survival in patients. Currently, there is no approved treatment for LAMA2-CMD in the United States. We are hopeful that MDL-101, which has the potential to activate LAMA1, the sister gene of LAMA2, the causative gene of this disease, and achieve a fundamental cure, will become the first treatment that improves the prognosis of these patients.”.

Modalis首席执行官兼总裁Haru Morita说：“LAMA2-CMD是一种严重的危及生命的罕见疾病，会导致患者肌肉无力和生存率降低。目前，美国还没有批准的LAMA2-CMD治疗方法。我们希望MDL-101能够激活LAMA2的姐妹基因LAMA1（该疾病的致病基因），并实现根本治愈，将成为改善这些患者预后的第一种治疗方法。”

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..

由于无法将其加载到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 altering patient DNA sequence. 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®技术允许在不改变患者DNA序列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 https://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。