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當?shù)貢r間2024年7月20日，專注于新型基因編輯技術(shù)的創(chuàng)新生物醫(yī)藥科技企業(yè)正序生物（上海）宣布蚓再，與廣西醫(yī)科大學(xué)第一附屬醫(yī)院合作開展的針對重型β-地中海貧血癥的堿基編輯藥物CS-101的臨床研究成功治愈首位外籍患者爆捞，達到持續(xù)擺脫輸血依賴超過兩個月咽瓷，總血紅蛋白濃度穩(wěn)定至120 g/L以上逗爹，并已回歸到正常生活中诚啃，實現(xiàn)了中國首次基因編輯治愈外籍患者鞭玩。截至目前粤唤，已有多位β-地貧患者經(jīng)過CS-101治療后擺脫輸血依賴啊嘁，其中有兩位患者已經(jīng)擺脫輸血依賴超過6個月朵聪，最長的已經(jīng)超過8個月。該療法采用了自主知識產(chǎn)權(quán)的堿基編輯技術(shù)干际，僅需數(shù)周時間制備亮花，即可實現(xiàn)“一次治療，終身治愈”的效果亚侠，為全球β-血紅蛋白病患者的徹底治愈帶來曙光曹体。（廣西醫(yī)科大學(xué)第一附屬醫(yī)院：基因編輯治愈首位外籍患者，老撾18歲地貧女孩獲新生）

中國首例接受堿基編輯治療（正序生物CS-101）的外籍β-地中海貧血癥患者已經(jīng)擺脫輸血依賴超過兩個月并已經(jīng)回歸正常生活

此次接受正序生物堿基編輯治療的老撾患者為輸血依賴型β-地貧患者硝烂，在接受CS-101治療前箕别，輸血頻率達到每月2單位紅細胞≈托唬患者在接受CS-101治療后兩個月內(nèi)串稀，總血紅蛋白濃度升高至120 g/L以上并且保持穩(wěn)定，成功實現(xiàn)脫離輸血依賴的目標狮杨。

截至目前母截，這項由正序生物與廣西醫(yī)科大學(xué)第一附屬醫(yī)院共同合作開展的針對重型β-地中海貧血癥的臨床研究中，已有多名β-地中海貧血患者順利接受CS-101治療橄教，并均已成功擺脫輸血依賴清寇。正序生物CS-101注射液也在今年4月獲得NMPA的IND批準，正式啟動了臨床I期試驗护蝶。

正序生物CS-101臨床研究的順利開展华烟，得益于該療法所用基因編輯技術(shù)的安全性和靶點的有效性。正序生物CS-101療法的原理青烙，是通過采集患者自體造血干細胞径瘪，利用上海科技大學(xué)自主研發(fā)的高精準變形式堿基編輯器tBE（transformer Base Editor）（Wang et al., Nat Cell Biol, 2021）冕店，對患者自體造血干細胞中的HBG1/2啟動子區(qū)域進行精準堿基編輯患敢，模擬健康人群中天然存在的有益堿基突變，重新激活γ-珠蛋白的表達嘁压，重建血紅蛋白的攜氧功能（Han et al., Cell Stem Cell, 2023）邦孽。進一步已堆，將編輯后的造血干細胞回輸至患者體內(nèi)，使得患者自身血紅蛋白濃度達到健康人水平揣喻，從而徹底擺脫輸血依賴圈机。

相較于傳統(tǒng)的輸血療法和異體造血干細胞移植法，正序生物CS-101注射液來自于患者的自體干細胞悠怕，制備周期短偏戳，患者不需要長時間等待，只需要數(shù)周即可實現(xiàn)“一次治療隶谒，終身治愈”的效果攻泼；相較于其他基于CRISPR技術(shù)的β-地貧基因編輯療法，正序生物CS-101注射液具有更好的有效性和安全性鉴象，采用的tBE技術(shù)不會造成DNA雙鏈斷裂忙菠，有效避免了DNA大片段缺失、染色體異位纺弊、脫靶突變等風(fēng)險牛欢，同時所編輯的靶點可更高效地激活胎兒血紅蛋白表達，使患者迅速完成造血重建淆游，更快地達到健康人血紅蛋白水平傍睹，更早擺脫輸血依賴。

正序生物CS-101注射液可以更安全犹菱、快速拾稳、有效的徹底治愈β-血紅蛋白病，將為全球β-血紅蛋白病患者帶來福音腊脱。血紅蛋白病是全世界最普遍的單基因遺傳病访得，其中，β-地中海貧血癥是一種流行于我國南方地區(qū)的遺傳性血紅蛋白病陕凹，在地中海沿岸國家左启、南亞和東南亞各國也較為多見。據(jù)統(tǒng)計贸薇，我國地中海貧血癥突變基因攜帶者約有3000萬人技腻，其中，重型和中間型地貧患者約為30萬人痕豺；東南亞地區(qū)地中海貧血癥突變基因攜帶者約有3億人亏铃，占總?cè)丝诘?4.60%闪妓；另一種常見的遺傳性血紅蛋白病是鐮刀型貧血癥努辛，在全球多個國家也廣泛存在，全球鐮刀型貧血癥突變基因攜帶者約占總?cè)丝诘?.5%甚庇，每年大約有30萬名嬰兒出生時患有鐮刀型貧血癥腿扑。

目前没赔，正序生物CS-101注射液針對β-地中海貧血癥的臨床I期試驗正在穩(wěn)步推進。同步陆拐，關(guān)于CS-101 注射液治療鐮刀型貧血癥的臨床試驗也正在籌備中取铃，針對鐮刀型貧血癥患者的全球招募計劃已經(jīng)啟動（詳情請聯(lián)系CT@correctsequence.com）。

正序生物將快速推進中國原創(chuàng)的基因編輯技術(shù)轉(zhuǎn)化為臨床藥物丛楚，開發(fā)出全球首創(chuàng)（First-in-Class）的堿基編輯治療藥物和同類最好（Best-in-Class）的基因編輯治療藥物族壳，為全球患者帶來安全可靠、費用更優(yōu)的選擇趣些。

（特別感謝廣西醫(yī)科大學(xué)第一附屬醫(yī)院仿荆、上海科技大學(xué)及上海臨床研究中心）

English Version

The First Clinical Gene Editing Therapy to Treat An Overseas Patient in China

CorrectSequence Therapeutics’ CS-101 Cures One Laos Patient with β-Thalassemia

On July 20, 2024, Shanghai, China, CorrectSequence Therapeutics Co., Ltd. (Correctseq) announced a significant milestone in their base editing therapy CS-101 for transfusion-dependent β-thalassemia. Utilizing their pioneering transformer Base Editor (tBE), Correctseq has successfully cured the first overseas patient with transfusion-dependent β-thalassemia in a clinical trial in collaboration with the First Affiliated Hospital of Guangxi Medical University. The patient has achieved a sustained transfusion-free status for over two months, with the hemoglobin level stabilized at above 120 g/L. As a result, the patient has been able to resume a normal life. (The First Affiliated Hospital of Guangxi Medical University: 基因編輯治愈首位外籍患者坏平，老撾18歲地貧女孩獲新生 The First Overseas Patient Cured by the Gene Editing Therapy, An 18-Year-Old Girl with β-Thalassemia from Laos is Given A New Life)

This milestone marks China's first documented report of an overseas patient being clinically cured through gene editing therapy. As of the reporting date, the clinical trial for CS-101 has yielded remarkable results. Several patients with transfusion-dependent β-thalassemia who have received CS-101 treatment have all achieved transfusion independence following treatment. Notably, two patients have remained transfusion-free for over six months, with the longest duration exceeding eight months.

CS-101, a pioneering cell and gene therapy based on tBE, holds the promise of a "single treatment, lifetime cure" for patients with β-hemoglobinopathies worldwide. This groundbreaking advancement brings hope for a complete cure to those enduring these health challenges.

The first overseas patient who received CS-101 treatment is from Laos. The patient was diagnosed with transfusion-dependent β-thalassemia and required 2 units of red blood cells (RBC) per month prior to receiving CS-101 treatment. Following the therapy, the patient’s hemoglobin level has reached at above 120 g/L within two months after treatment and maintained at such level as of the report date. As a result, the patient successfully achieved independent of blood transfusions.

In April 2024, CS-101 received IND (Investigational New Drug) approval from the National Medical Products Administration (NMPA) of China. This approval has allowed the Phase I clinical trial to commence, marking a significant milestone in the development of CS-101 as a promising treatment for transfusion-dependent β-thalassemia.

The successful implementation of the CS-101 clinical trial is a testament to the safety of the base editing technology employed and the selection of effective targets. The underlying principle of CS-101 involves the collection of the patient's own hematopoietic stem cells, followed by the utilization of tBE. Pioneered by scientists at ShanghaiTech University (Wang et al., Nat Cell Biol, 2021), this technology enables the precise editing of the DNA sequence within the promoter region of the gene responsible for encoding γ-globin (HBG1/2).

By mimicking a naturally occurring beneficial single-nucleotide variant found in individuals with hereditary persistence of fetal hemoglobin, the tBE reactivates γ-globin expression, resulting in the production of functional HbF (Han et al., Cell Stem Cell, 2023). The edited stem cells are then reintroduced into the patient's body, enabling continuous production of blood cells with functional hemoglobin. This approach eliminates the need for frequent blood transfusions, offering a significant advancement in the treatment of transfusion-dependent β-thalassemia.

Compared to traditional blood transfusion therapy and allogeneic hematopoietic stem cell transplantation, CS-101 offers several compelling advantages. One of the key benefits is its short preparation period. Another is eliminating the need for long waiting time for a matching donor since it utilizes the patient’s own hematopoietic stem cells. CS-101 has the potential to achieve a "single treatment, lifetime cure" within a few weeks.

In comparison to other CRISPR-based β-thalassemia gene editing therapies, CS-101 does not carry safety risks associated with large DNA fragment deletions, chromosomal translocation, or off-target mutations. This enhances the overall safety profile of the treatment and reduces potential complications.

Overall, CS-101 offers a promising approach for the treatment of transfusion-dependent β-thalassemia, providing faster and safer hematopoietic reconstruction, quicker restoration of hemoglobin levels, and a shorter path to achieving transfusion independence compared to other therapies.

CS-101 offers a potential complete cure for β-hemoglobinopathy in a safer, faster, and more efficient manner, instilling new hope for patients worldwide. Hemoglobinopathies encompass the most prevalent group of monogenic diseases globally. Among them, β-thalassemia is particularly prevalent in southern China, as well as in Mediterranean countries, South Asian and Southeast Asian countries. In China alone, there are approximately 30 million individuals carrying the thalassemia mutant gene, with around 300,000 severe and intermediate thalassemia patients. In Southeast Asia, approximately 300 million people carry the thalassemia mutant gene, accounting for 44.60% of the total population in the region.

Another common genetic hemoglobinopathy is sickle cell disease (SCD), which is prevalent in various countries worldwide. Approximately 3.5% of the global population carries the mutant gene for SCD, and around 300,000 newborns worldwide are affected by SCD each year.

The Phase I clinical trial for the treatment of β-thalassemia with CS-101 is proceeding steadily. In tandem, a clinical trial targeting SCD with CS-101 are in active preparation, which is an important step towards addressing the needs of SCD patients. A global recruitment program for SCD patients is recently launched, underscoring Correctseq’s commitment in providing care and resolution for patients worldwide.

Correctseq is dedicated to expediting the clinical translation of pioneering gene editing technologies. By developing best-in-class gene editing therapy and first-in-class base editing therapy, Correctseq aims to provide safe, reliable, and cost-effective treatment options for patients worldwide.

For more detailed information about the clinical trials and recruitment program, it is recommended to contact CT@correctsequence.com.

Acknowledgements: The First Affiliated Hospital of Guangxi Medical University, ShanghaiTech University, Shanghai Clinical Research and Trial Center.

About CorrectSequence Therapeutics

CorrectSequence TherapeuticsTM (CorrectseqTM), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrate its superior performance. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for hereditary angioedema (HAE), hypercholesterolemia, and hypertriglyceridemia. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. We are developing multiple pipelines targeting genetic diseases, cancer immunotherapy, metabolic disorders, and cardiovascular diseases.

Please refer to our homepage for more information about the tBE and its therapeutics applications at www.correctsequence.com

關(guān)于正序生物

正序生物（CorrectSequence TherapeuticsTM）是一家專注于新型基因編輯技術(shù)拢操、處于臨床階段的的生物醫(yī)藥科技公司，致力于利用世界先進的堿基編輯體系舶替，為罹患嚴重疾病的人們開發(fā)突破性精準療法令境，造福人類健康。

公司基于以變形式堿基編輯器tBE為代表的自主知識產(chǎn)權(quán)堿基編輯系統(tǒng)搭建了融合多治療領(lǐng)域的新藥發(fā)現(xiàn)平臺顾瞪，可長期開發(fā)和篩選針對多種遺傳性疾病或罕見病的有效治療靶點舔庶。所創(chuàng)建的多種精準療法，在動物體內(nèi)實現(xiàn)了疾病治療靶點上的高效的編輯效率和未檢出脫靶的安全性陈醒。目前栖茉，已經(jīng)針對遺傳疾病、腫瘤孵延、代謝疾病狭缰、心血管疾病等布局了近10條管線，首條管線CS-101已使多位β-地中海貧血癥患者擺脫輸血依賴茸例，正處于IND臨床試驗階段罪靠。

正序生物孵化自上海科技大學(xué)偷逆，擁有世界一流的創(chuàng)新技術(shù)平臺和管線研發(fā)能力季糜。在中國科學(xué)院上海高等研究院和上海細胞和基因產(chǎn)業(yè)園擁有先進的研發(fā)和生產(chǎn)中心，在北京華貿(mào)中心設(shè)立有臨床注冊和運營中心卤索。目前恰除，公司匯聚了數(shù)十位來自全球基因治療技術(shù)開發(fā)、藥物研發(fā)斯泥、工藝開發(fā)與生產(chǎn)诵藐、臨床開發(fā)和質(zhì)量與合規(guī)等領(lǐng)域的優(yōu)秀生物醫(yī)藥專家，管理團隊和科研團隊擁有平均十年以上工業(yè)界經(jīng)驗，碩士及以上學(xué)位比例超過40%册血，核心技術(shù)人員畢業(yè)于國內(nèi)外頂尖名校扑轮。

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