SAN FRANCISCO--(BUSINESS WIRE)--Kamau Therapeutics ('Kamau' or 'the Company'), a clinical-stage gene correction company, announced today its emergence from stealth, following a strategic transaction with Graphite Bio (“Graphite”). Under the terms of the agreement, Kamau received an option to acquire all genome editing assets, including a platform technology that integrates precision DNA repair, using homology directed repair (HDR) and CRISPR/Cas9..

旧金山--（商业新闻短讯）--临床阶段基因校正公司Kamau Therapeutics（“Kamau”或“该公司”）今天宣布，在与Graphite Bio（“Graphite”）进行战略交易后，它将从隐形公司崛起。根据协议条款，卡莫获得了收购所有基因组编辑资产的选择权，包括使用同源定向修复（HDR）和CRISPR/Cas9整合精确DNA修复的平台技术。。

The HDR platform technology, developed by gene-editing pioneers Drs. Matthew Porteus and Maria Grazia Roncarolo at Stanford, received U.S. Food and Drug Administration (FDA) clearance for the Investigational New Drug (IND) application of nulabeglogene autogedtemcel (nula-cel). This technology now forms the basis for Kamau's lead program, nula-cel, previously developed by Graphite.

HDR平台技术由斯坦福大学的基因编辑先驱Matthew Porteus博士和Maria Grazia Roncarolo博士开发，已获得美国食品和药物管理局（FDA）批准用于nulabeglogene autogedtemcel（nula cel）的研究性新药（IND）应用。这项技术现在成为卡莫领先项目nula cel的基础，nula cel先前由Graphite开发。

The newly established company will retain control of all intellectual property (IP) related to HDR-based CRISPR gene correction as well as the IND application..

新成立的公司将保留与基于HDR的CRISPR基因校正以及IND应用相关的所有知识产权（IP）的控制权。。

Nula-cel is an investigational, potentially first-in-class hematopoietic stem-cell therapy engineered to restore normal adult hemoglobin (HgbA) by precisely correcting the mutation that causes sickle hemoglobin (HgbS), which may provide a cure for patients with this life-threatening genetic disease.

Nula cel是一种研究性的，可能是一流的造血干细胞疗法，旨在通过精确纠正导致镰状血红蛋白（HgbS）的突变来恢复正常的成人血红蛋白（HgbA），这可能为这种危及生命的遗传病患者提供治疗。

The therapy has established proof of concept in the first patient treated for sickle cell disease (SCD). The data will be presented during a poster session (Abstract #5000) on Monday, December 11, at the 2023 Annual Meeting of the American Society of Hematology (ASH)..

该疗法已在第一名接受镰状细胞病（SCD）治疗的患者中建立了概念验证。这些数据将在12月11日（星期一）美国血液学会（ASH）2023年年会的海报会议（摘要#5000）上发布。。

Redefining Genetic Disease Treatment

重新定义遗传病治疗

Kamau's HDR gene correction technology holds immense promise in enhancing human health outcomes through curative cell therapies. The versatile and robust platform corrects disease-causing DNA mutations, offering unmatched precision, template-based gene correction, and minimal off-target effects with the capacity to replenish the bloodstream with healthy red blood cells..

卡莫的HDR基因校正技术在通过治愈性细胞疗法改善人类健康方面具有巨大的前景。该多功能且强大的平台可纠正引起疾病的DNA突变，提供无与伦比的精确度，基于模板的基因校正，以及最小的脱靶效应，并具有用健康红细胞补充血流的能力。。

Kamau co-founders Dr. Porteus, previously a scientific co-founder of CRISPR Therapeutics and academic co-founder of Graphite Bio, and Dr. Roncarolo, will also serve as the company’s scientific leadership to expand the use of the platform HDR technology in addressable genetic diseases beyond SCD. Jane Gorgan, Ph.D., previously the chief scientific officer at Graphite, and Jerry Cacia, previously the chief technical officer at Graphite, will serve as strategic advisors to Kamau..

Kamau联合创始人Porteus博士（之前是CRISPR Therapeutics的科学联合创始人和Graphite Bio的学术联合创始人）和Roncarolo博士也将担任该公司的科学领导者，以扩大平台HDR技术在SCD以外的可解决遗传病中的应用。曾任Graphite首席科学官的Jane Gorgan博士和曾任Graphite首席技术官的Jerry Cacia将担任Kamau的战略顾问。。

Dr. Porteus commented, “Our HDR technology brings to life the concept of genome editing. Kamau's approach surpasses first-generation technologies, which often introduce new errors in the genome. Instead, our technology can correct, add, replace, and rearrange disease-causing genes with functional or new ones.

Porteus博士评论道：“我们的HDR技术为基因组编辑的概念带来了活力。卡莫的方法超越了第一代技术，后者经常在基因组中引入新的错误。相反，我们的技术可以用功能性或新的基因纠正、添加、替换和重排致病基因。

In our lead program, nula-cel, the technology transforms the sickle cell gene into a non-pathogenic one, addressing the root cause of the illness at the genomic level. This results in a significant reduction of pathogenic sickle hemoglobin and the production of non-pathogenic adult hemoglobin. Kamau aims to achieve the gold standard in genome engineering: directly correcting mutations that cause diseases, and we are making strides toward this ambitious goal.”.

在我们的主导项目nula cel中，该技术将镰状细胞基因转化为非致病性基因，从基因组水平解决了疾病的根本原因。这导致致病性镰状血红蛋白的显着减少和非致病性成人血红蛋白的产生。卡莫的目标是实现基因组工程的黄金标准：直接纠正导致疾病的突变，我们正在朝着这一雄心勃勃的目标迈进。”。

One Year Follow-up on the First Patient Treated with Nula-Cel

第一例接受Nula-Cel治疗的患者的一年随访

At the 2023 ASH Annual Meeting, Dr. David Shyr, Clinical Assistant Professor in Pediatric Stem Cell Transplantation from Stanford Medicine, will present a poster detailing outcomes of the first patient treated with investigational nula-cel during a Phase 1/2 clinical trial.

在2023年ASH年会上，斯坦福医学院儿科干细胞移植临床助理教授David Shyr博士将展示一张海报，详细介绍第一位在1/2期临床试验中接受研究性nula-cel治疗的患者的结果。

The abstract is available here, and the poster with one-year data will be available on the Kamau website on December 11. Key highlights from the six-month follow up include the following:

这里提供了摘要，12月11日将在Kamau网站上提供一年数据的海报。六个月随访的主要亮点包括：

Prior to joining the trial, the patient experienced six painful blood vessel blockages known as vaso-occlusive crises and had been hospitalized four times each year for the past two years.

在参加试验之前，该患者经历了六次痛苦的血管阻塞，称为血管闭塞性危机，并且在过去两年中每年住院四次。

The patient underwent meticulous procedures to harvest and store CD34+ cells, with treatment initiated in August 2022 post-chemotherapy.

该患者接受了细致的程序来收获和储存CD34+细胞，并于化疗后2022年8月开始治疗。

While early stages showed promise, subsequent tests revealed a lower-than-expected cell survival rate, necessitating platelet and red blood cell transfusions due to low platelet counts.

虽然早期阶段显示出前景，但随后的测试显示细胞存活率低于预期，由于血小板计数低，需要输注血小板和红细胞。

The introduction of eltrombopag, not initially part of the trial protocol, temporarily posed a significant adverse event but did not interrupt the trial.

最初不属于试验方案的eltrombopag的引入暂时造成了严重的不良事件，但没有中断试验。

Upon gradual discontinuation of eltrombopag, the patient's blood counts improved, alongside positive changes in other markers. Blood tests indicated a favorable impact of nula-cel treatment on reducing sickle hemoglobin and increasing healthier types (HgbA and HgbF).

逐渐停用eltrombopag后，患者的血细胞计数得到改善，其他标志物也发生了积极变化。血液测试表明，nula-cel治疗对减少镰状血红蛋白和增加更健康的类型（HgbA和HgbF）有良好的影响。

Dr. Shyr explained, “Current sickle cell treatments focus on managing acute symptoms such as pain crises and avoiding severe complications like stroke, without curing the patients. Bone marrow transplant has been the only curative option, but carries significant risks for transplant related complications, some of which can be life threatening.

Shyr博士解释说：“目前的镰状细胞治疗侧重于管理急性症状，如疼痛危机，避免中风等严重并发症，而没有治愈患者。骨髓移植是唯一的治疗选择，但存在移植相关并发症的重大风险，其中一些可能危及生命。

Kamau's gene-editing technology can offer a potential cure using the patient's cells. Further study of nula-cel is eagerly anticipated.”.

卡莫的基因编辑技术可以利用患者的细胞提供潜在的治疗方法。人们热切期待着对nula-cel的进一步研究。”。

Dr. Roncarolo added, “Nula-cel represents a groundbreaking approach to target the underlying mutation in sickle cell disease. These proof of concept data offer important insights and hope to those grappling with this debilitating disease. We look forward to treating additional patients in the clinical trial with the goal of curing this disease.”.

Roncarolo博士补充道：“Nula-cel代表了一种针对镰状细胞病潜在突变的开创性方法。这些概念验证数据为那些患有这种衰弱性疾病的人提供了重要的见解和希望。我们期待着在临床试验中治疗更多患者，以治愈这种疾病。”。

ASH Poster Presentation Details

ASH海报展示细节

Title: One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

标题：第一例接受Nula-Cel治疗的患者的一年随访：一种自体CRISPR/Cas9基因校正的CD34+细胞产物，用于治疗镰状细胞病

Abstract #: 5000

摘要#：5000

Session: 801. Gene Therapies: Poster III

课程：801。基因疗法：海报III

Hematology Disease Topics & Pathways: Biological therapies, Sickle Cell Disease, Hemoglobinopathies, Gene Therapy, Diseases, Therapies

血液病主题和途径：生物疗法，镰状细胞病，血红蛋白病，基因疗法，疾病，疗法

Presenter: David Shyr, MD, Stanford Medicine

主持人：斯坦福医学博士David Shyr

Advancing Manufacturing Capabilities with Strategic Agreement

通过战略协议提升制造能力

Kamau has reached a significant milestone in its commitment to advancing its manufacturing processes. The company recently entered into a Contract Development and Manufacturing Organization (CDMO) agreement, marking a strategic move toward enhanced capabilities.

卡莫在致力于推进其制造流程方面取得了重要的里程碑。该公司最近签署了合同开发和制造组织（CDMO）协议，标志着向增强能力的战略举措。

Through this collaboration, Kamau is actively initiating technology transfer and is focused on elevating the HDR technology, increasing cell yield, and improving overall cell health. These advancements aim to optimize production efficiency, ensuring a more robust and sustainable manufacturing framework..

通过这种合作，卡莫正在积极启动技术转让，并专注于提高HDR技术，提高细胞产量，改善整体细胞健康。这些进步旨在优化生产效率，确保更强大和可持续的制造框架。。

About Sickle Cell Disease

关于镰状细胞病

Sickle Cell Disease (SCD) is a common genetic condition affecting over 500,000 people worldwide every year. In the U.S., more than 100,000 individuals have SCD, and around 20% of them have a severe form of the disease. SCD occurs when a gene called HBB undergoes a specific change, which changes one substance in its genetic code to another.

镰状细胞病（SCD）是一种常见的遗传病，每年影响全球50多万人。在美国，超过100000人患有SCD，其中约20%患有严重的疾病。SCD发生在称为HBB的基因发生特定变化时，这种变化将其遗传密码中的一种物质改变为另一种物质。

This genetic alteration leads to the creation of a variant known as HbS. The irregular shape of red blood cells resulting from this mutation causes serious health issues, including anemia, blockages in blood flow, severe pain, an increased risk of stroke, damage to multiple organs, and ultimately a shorter lifespan, representing a significant medical need..

这种基因改变导致产生一种称为HbS的变体。这种突变导致的红细胞形状不规则会导致严重的健康问题，包括贫血，血流阻塞，剧烈疼痛，中风风险增加，多器官受损，最终寿命缩短，这是一个重大的医疗需求。。

About Kamau Therapeutics

关于Kamau Therapeutics

Kamau Therapeutics is a clinical-stage, gene correction company dedicated to transforming the lives of patients with devastating genetic diseases through best-in-class, curative stem-cell therapies. Kamau stands apart through the unique capabilities of its next-generation gene correction platform that directly corrects genetic mutations with unprecedented precision: removing the pathologic mutation and replacing it with the healthy version..

Kamau Therapeutics是一家临床阶段的基因校正公司，致力于通过一流的治愈性干细胞疗法改变患有毁灭性遗传疾病的患者的生活。Kamau凭借其下一代基因校正平台的独特功能而脱颖而出，该平台以前所未有的精度直接校正基因突变：去除病理突变并用健康版本替代。。

The company’s platform technology is based on the pioneering research of company co-founder Dr. Matthew Porteus, MD, PhD, and offers broad potential for transforming human health. Kamau’s lead clinical program, nulabeglogene autogedtemcel (nula-cel), previously developed by Graphite Bio, is the only hematopoietic stem-cell based therapy in clinical development for sickle cell disease (SCD) that precisely corrects the mutation in the beta-globin gene found in the sickled hemoglobin (HgbS) of SCD patients, restoring patient’s stem cells to normal adult hemoglobin (HgbA)..

该公司的平台技术基于公司联合创始人Matthew Porteus博士的开创性研究，为改变人类健康提供了广阔的潜力。Kamau的主要临床项目nulabeglogene autogedtemcel（nula-cel）先前由Graphite Bio开发，是镰状细胞病（SCD）临床开发中唯一一种基于造血干细胞的疗法，它可以精确纠正SCD患者镰状血红蛋白（HgbS）中发现的β-珠蛋白基因突变，将患者的干细胞恢复为正常成人血红蛋白（HgbA）。。

Kamau plans to enroll additional patients with SCD in the Phase 1/2 clinical trial of nula-cel, for which it has received both fast track and orphan drug designations from the U.S. Food and Drug Administration (FDA).

Kamau计划在nula-cel的1/2期临床试验中招募更多SCD患者，该试验已获得美国食品和药物管理局（FDA）的快速通道和孤儿药指定。

For more information, please visit www.kamautx.com and follow the company’s progress on our LinkedIn page.

有关更多信息，请访问www.kamautx.com，并在我们的LinkedIn页面上关注公司的进展。