CAMBRIDGE, Mass., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene editing medicine, for the treatment of severe sickle cell disease (SCD)..

2023年10月16日（GLOBE NEWSWIRE）-临床阶段基因组编辑公司Editas Medicine，Inc。（纳斯达克股票代码：编辑）今天宣布，美国食品和药物管理局（FDA）授予再生医学高级治疗（RMAT）指定用于治疗严重镰状细胞病（SCD）的研究性基因编辑药物EDIT-301。。

“Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death. Receiving RMAT designation for EDIT-301 for severe sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 can provide life-changing clinical benefits to patients,” Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.

“镰状细胞病是一种破坏性疾病，可导致贫血，疼痛危机，器官衰竭和早期死亡。接受MATT指定EDIT-301治疗严重镰状细胞病突出了患者迫切需要新的治疗方案，并支持我们的信念EDIT-301可以为患者提供改变生活的临床益处，”Gilmore O'Neill，M.B.，M.M.Sc。，Editas Medicine总裁兼首席执行官。

“I would like to thank the participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial. We look forward to sharing further clinical updates including additional data for the trial prior to year-end.”.

“我要感谢参与RUBY试验的合作机构的参与者，他们的家人，临床医生和同事，我们期待分享进一步的临床更新，包括年底之前试验的其他数据。”。

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine therapies. An investigational cell therapy medicine or gene editing medicine is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the experimental medicine has the potential to address unmet medical needs for the disease or condition.

根据“21世纪治愈法”成立，RMAT指定是一项专门的计划，旨在加快有希望的再生医学疗法的开发和审查过程。如果研究细胞治疗药物或基因编辑药物旨在治疗，修改，逆转或治愈严重或危及生命的疾病或病症，则符合RMAT指定的条件，初步临床证据表明实验药物具有潜力解决疾病或病症未满足的医疗需求。

Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including but not limited to intensive FDA guidance on efficient and expedited drug development, possible rolling review, and priority review of the biologics license application (BLA), and FDA’s organizational commitment involving senior managers..

RMAT指定的优势包括快速和突破性治疗指定计划的所有好处，包括但不限于FDA关于有效和快速药物开发的强化指导，可能的滚动审查以及生物制剂许可证申请（BLA）的优先审查，以及FDA与高级经理有关的组织承诺。。

The FDA previously granted Orphan Drug Designation and Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease and beta thalassemia.

FDA以前授予孤儿药名称和罕见儿科疾病名称EDIT-301用于治疗镰状细胞病和β地中海贫血。

About Sickle Cell Disease

关于镰刀型红血球疾病

Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death.

镰状细胞病是由β珠蛋白基因突变导致镰状血红蛋白（HbS）聚合引起的遗传性血液病。在镰状细胞病中，红细胞畸形为镰状而不是典型的盘状。异常形状导致红细胞寿命缩短并阻塞血流，导致贫血，疼痛危机，器官衰竭和早期死亡。

There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling..

目前美国估计有10万人患有镰状细胞病。较高水平的胎儿血红蛋白（HbF）抑制HbS聚合，从而减少镰状疱疹的表现。。

About EDIT-301

关于编辑301

EDIT-301 is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease.

EDIT-301是一种正在研究的用于治疗严重镰状细胞病（SCD）和输血依赖性β地中海贫血（TDT）的实验性基因编辑药物。EDIT-301由患者来源的CD34+造血干细胞和在γ珠蛋白基因（HBG1和HBG2）启动子上编辑的祖细胞组成，其中天然存在的诱导胎儿血红蛋白（HbF）的突变存在于其中，由高度特异性和高效的专有工程AsCas12a核酸酶。

Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT..

来自EDIT-301 CD34+细胞的红细胞表现出胎儿血红蛋白产生的持续增加，这有可能为患有严重SCD和TDT的人提供一次性，持久的治疗益处。。

About the RUBY Trial

关于RUBY试验

The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576)..

RUBY试验是一项单臂，开放标签，多中心的1/2期研究，旨在评估EDIT-301在严重镰状细胞病患者中的安全性和有效性。登记的患者将接受单次EDIT-301管理。更多详细信息请访问www.clinicaltrials.gov（NCT＃04853576）。。

About Editas Medicine

关于Editas医学

As a clinical-stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases.

作为一家临床阶段的基因组编辑公司，Editas Medicine专注于将CRISPR/Cas12a和CRISPR/Cas9基因组编辑系统的力量和潜力转化为针对世界各地严重疾病患者的强大治疗方案。Editas Medicine旨在发现，开发，制造和商业化用于广泛疾病的转化型，耐用，精密基因组药物。

Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com..

Editas Medicine是Broad Institute的Cas12a专利权和Broad Institute以及哈佛大学的Cas9人类药物专利权的独家被许可人。有关最新信息和科学演示文稿，请访问www.editasmedicine.com。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

本新闻稿包含1995年“私人证券诉讼改革法”含义范围内的前瞻性陈述和信息。“预期”，“相信”，“继续”，“可能”，“估计”，“期望”，“打算”，“可能”，“计划”，“潜在”，“预测”，“项目”，“目标”，“应该”，“应该”，以及类似的表达式旨在标识前瞻性语句，尽管并非所有前瞻性陈述都包含这些识别词。

Forward-looking statements in this press release include statements regarding the Company’s expectation to provide further clinical data updates, including additional data for the RUBY trial by year-end. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements.

本新闻稿中的前瞻性声明包括有关公司期望提供进一步临床数据更新的声明，包括年底RUBY试验的其他数据。公司可能实际上未达到这些前瞻性声明中披露的计划，意图或期望，您不应过分依赖这些前瞻性声明。

Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the RUBY trial, and clinical development of the Company’s product candidates, including EDIT-301; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements.  These and other risks are described in greater detail under th.

由于各种重要因素，实际结果或事件可能与这些前瞻性声明中披露的计划，意图和期望有很大不同，包括：启动和完成临床前研究和临床试验（包括RUBY试验）所固有的不确定性，以及本公司产品候选人的临床开发，包括编辑301；临床前研究和临床试验结果的可用性和时间安排；临床试验的中期结果是否可以预测试验的最终结果或未来试验的结果；对监管部门批准进行试验或销售产品的期望以及足以满足公司可预见和不可预见的运营费用和资本支出要求的资金可用性。这些和其他风险将在下面更详细地描述。

Media and Investor Contact:

媒体和投资者联系：

Cristi Barnett

克里斯蒂·巴内特

(617) 401-0113

(617) 401-0113

cristi.barnett@editasmed.com

cristi.barnett@editasmed.com