1.1. 概述 Introduction

(本章節內容的可下載PDF版檔 A downloadable PDF version of the content in this section aCGT Intro-20241205.pdf)

aCGT專注于細胞與基因治療新藥的研發、全球申報、海内外臨床試驗樣品生產和人體臨床試驗。

aCGT亞洲細胞與基因治療集團Asia Cell & Gene Therapeutics Group (開曼群島)下設三個全資業務板塊①Asia Cell & Gene Therapeutics (H.K.) Co., Ltd.亞洲細胞與基因(香港)有限公司(成立於2014年)、②上海泉生生物科技有限公司(成立於2016年)、③aCGT USA Inc.(註冊中)。

aCGT focuses on the research and development of new drugs for cell and gene therapy, global submissions, domestic and overseas production of clinical trial materials, and human clinical trials.

aCGT / Asia Cell & Gene Therapeutics Group (Cayman Islands) has three wholly-owned subsidiaries: ① Asia Cell & Gene Therapeutics (Hong Kong) Co., Ltd. (established in 2014), ② Asia Cell & Gene Therapeutics (Shanghai) Co., Ltd. (established in 2016) and ③ aCGT USA Inc. (in the process of registration).

aCGT全部產品均是“貨架式”異體細胞製劑。截止目前，aCGT有3項異體臍帶間充質幹細胞靜脈注射液(allogenic umbilical-cord mesenchymal stem cell intravenous injection)國家1類新藥處於中國藥監局II期臨床試驗；另2項處於I期臨床試驗；其中1項(治療強直性脊柱炎ankylosing spondylitis)正在申請美國FDA的直接II期臨床試驗，並已獲美國FDA積極肯定性pre-IND meeting回復函。

同時，aCGT“貨架式”的①iPSC分化iOPC(少突膠質細胞前體細胞oligodendrocyte precursor cells)製劑治療脊髓損傷spinal injury、② iPSC分化iNPC(神經前體細胞neural precursor cells)製劑治療腦卒中stroke等、③ CAR-iNK製劑治療系統性紅斑狼瘡systemic lupus erythematosus (SLE )等 計畫分別於今年12月、明年3月、11月向中、美藥監局提交preIND meeting申請。其臨床前數據優於美國同產品，並正在積極開展IIT臨床試驗。

aCGT臨床前“貨架式”產品管線還包括iPSC分化iRPE(視網膜色素上皮細胞retinal pigment epithelial cells)治療黃斑病變、iPSC分化beta細胞治療糖尿病、功能增強的iOPC治療脊髓損傷等。

All products of aCGT are off-the-shelf allogenic cell preparations. As of now, aCGT has three new drugs of off-the -shelf allogenic umbilical-cord mesenchymal stem cell intravenous injection in Phase II clinical trials with the National Medical Products Administration of China; another two are in Phase I clinical trials. Among these, one (for the treatment of ankylosing spondylitis) is applying for a direct Phase II clinical trial with the US FDA and has received a positive response letter from the FDA regarding the pre-IND meeting.

At the same time, aCGT plans to submit pre-IND meeting applications to China NMPA and the US FDA for the following off-the-shelf new drugs: ① iPSC-derived iOPC (oligodendrocyte precursor cells) for spinal injury in December of this year, ② iPSC-derived iNPC (neural precursor cells) for stroke etc. in March next year, and ③ CAR-iNK for systemic lupus erythematosus (SLE) etc.  in November next year. Their preclinical data outperform similar products in the US. They are now actively undergoing IIT clinical trials.

aCGT's preclinical off-the-shelf product pipeline also includes iPSC-derived iRPE (retinal pigment epithelial cells) for treating macular degeneration, iPSC-derived beta cells for diabetes treatment, and function-enhanced iOPC for spinal injury.

(注：自2017年中國藥監局頒佈法規正式確立幹細胞是藥物，中國臍帶間充質幹細胞製劑新藥研發方興未艾。aCGT目前是中國臍帶間充質幹細胞製劑新藥獲批進入藥監局臨床試驗數量最多的公司。近日北京藥監局半年工作會議核發了中國第一張幹細胞、也是第一張臍帶間充質幹細胞《藥品生產許可證》(重磅！北京藥監局核發全國第一張幹細胞《藥品生產許可證》)，結合aCGT截至目前已經獲得的積極性臨床試驗資料，該第一張臍帶幹細胞《藥品生產許可證》極大地增大了aCGT多個在研臍帶幹細胞藥物未來獲批NDA/BLA及《藥品生產許可證》的把握。

Note: Since the Chinese National Medical Products Administration (NMPA) issued regulations in 2017 officially establishing stem cells as drugs, the research and development of new drugs using human umbilical cord mesenchymal stem cell preparations has been rapidly advancing. Currently, aCGT is the company with the highest number of approved clinical trial applications for umbilical cord mesenchymal stem cell preparations in China. Recently, the Beijing Medical Products Administration issued the first stem cell drug production license in China, which is also the first license for umbilical cord mesenchymal stem cells (Major! Beijing Medical Products Administration issues the first stem cell drug production license in the country). Combined with the positive clinical trial data aCGT has obtained so far, this first umbilical cord stem cell drug production license greatly enhances aCGT's chances of obtaining NDA/BLA approvals and drug production licenses for several of its ongoing umbilical cord stem cell drugs in the future.)

請點擊下圖中各產品管線連結進入該具體產品介紹頁面   Please click on the links for each product pipeline in the image below to enter the specific product introduction page:

aCGT目前已建成①通用型(基因工程低免疫原性)iPSC技術平臺、②iPSC分化技術平臺、③功能基因設計與篩選技術平臺、④生物反應器工程技術平臺、⑤高穩定性製劑技術平臺。

aCGT臨床級製劑生產基地有 ①位於上海古愛路9號使用中的B級和C級潔淨4000平米GMP車間、②位於浙江安吉裝修中的近3萬平米的“中國NMPA.美國FDA.歐盟EMA國際cGMP認證的細胞製劑生產基地”、③contracted香港HKIB的PIC/S GMP車間。

aCGT has established the following technical platforms: ① universal (genetically-engineered low immunogenicity) iPSC technology platform, ② iPSC differentiation technology platform, ③ functional gene design and screening technology platform, ④ bioreactor engineering technology platform, and ⑤ high stability formulation technology platform.

aCGT has clinical-grade formulation production facilities, including: ① a 4000 m² cGMP pilot plant with B and C-level clean rooms currently in use at No. 9 Guai Road, Shanghai; ② a nearly 30,000 m² cGMP production plant in Anji, Zhejiang, under renovation and designed to be certified by the China NMPA, US FDA, and EU EMA; and ③ a PIC/S GMP pilot plant contracted in Hong Kong (Hong Kong Institute of Biotechnologies).

aCGT公司建立了先进水平的“细胞与基因药物研发标准化流程化在线云管理系统GRCP(Good R&D and Clinical-trial Practices)”；并基于GRCP，建立了强大的各高校课题组合作研发中心及公司兼职研发团队，保障了雄厚的公司研发储备项目；并与公司全职研发团队衔接，形成了aCGT新药研发流程：非GMP车间早研(各合作高校课题组) → GMP车间药学CMC研发 → 毒理动物安评实验 → 药理动物药效实验 → 中.美.欧药监局IND申报 → 全球人体临床试验。

aCGT has established an advanced-level 'Online Cloud Management System for Standardized Processes in Cell and Gene Drug R&D' (GRCP: Good R&D and Clinical-trial Practices). Based on GRCP, aCGT has created a robust collaborative R&D center with various university research teams and a part-time R&D team, ensuring a strong pipeline of research projects. This is connected with the company's full-time R&D team, forming the new drug development process at aCGT: early research in non-GMP workshops (various collaborating university research teams) → pharmaceutical CMC R&D in GMP workshops → toxicological animal safety assessment experiments → pharmacological efficacy experiments on animals → IND submissions to regulatory agencies in China, the U.S., and Europe → global human clinical trials.

aCGT於2022年9月完成三億元人民幣股權融資；並已於2024年7月啟動海外上市公司架構搭建。計畫以2025年6月30日作為基準日申報並於2025年第四季度美國納斯達克IPO上市。

In September 2022, aCGT completed a RMB 300 million (US$ 42 million) equity financing; and in July 2024, initiated the construction of an overseas listing company structure. aCGT plans to be listed on NASDAQ in the fourth quarter of 2025, with a filing reference date of June 30, 2025.

1.2. 公司架构 Company Structure

(商務部 國家衛生健康委 國家藥監局《關於在醫療領域開展擴大開放試點工作的通知(商資函[2024]568號)》.pdf 在中國(北京)自由貿易試 驗區、中國(上海)自由貿易試驗區、中國(廣東)自由貿易試驗區和海南自由貿易港允許外商投資企業從事人體幹細胞、基因診斷與治療技術開發和技術應用，以用於產品註冊上市和生產。所有經過註冊上市和批准生產的產品，可在全國範圍使用。

Ministry of Commerce, National Health Commission, National Medical Products Administration 'Notice on Expanding the Pilot of Opening Up in the Medical Field (Document No. [2024] 568)'.pdf In the China (Beijing) Pilot Free Trade Zone, China (Shanghai) Pilot Free Trade Zone, China (Guangdong) Pilot Free Trade Zone, and Hainan Free Trade Port, foreign-invested enterprises are allowed to engage in the development and technical application of human stem cells and gene diagnosis and therapy technologies for product registration, market launch, and production. All products that have been registered, approved for production, and marketed can be used nationwide.)

1.3. 竞争优势 Competitive Advantages

充分利用中国CGT新药研发领域 1)人力资源、2)上下游配套资源、3)临床试验资源 丰富且较欧美成本低的优势，在ICH体系下严格按照中国NMPA/美国FDA/欧盟EMA规范，遵循CGT新药工艺过程技术门槛高，但终产品人体细胞本身无像化药结构直接专利限制的竞争方式，积极建立aCGT自己工艺过程专利或技术秘密，包括合同有偿获取授权如iPSC主库等具专利的基础性技术，对欧美临床试验II期后数据优秀的CGT在研产品以及部分创新产品，予以大面积覆盖式me-better和innovative研发和全球申报。

截至目前，aCGT已具竞争优势如下：

1.3.1. 同时拥有①间充质干细胞MSC、②多能干细胞iPSC、③基因编辑 三大技术平台和产品管线；

1.3.2. 利用中国研发资源丰富且成本低(美国FDA/欧盟EMA接受其结果数据)优势，研发产品管线丰富；

1.3.3. 整合大学资源，产品储备雄厚；

1.3.4. fast-follow/me-better产品研发数据优于欧美同产品；同时具有部分first-in-class/innovative产品；

1.3.5. 工业规模化封闭连续生物反应器细胞扩增培养和分化工艺，保障高质量低成本(上市销售的绝对竞争优势)；

1.3.6. 中国、美国、欧洲同步申报，市场潜力大。

Make full use of the advantages in China's CGT (Cell and Gene Therapy) new drug R&D field, including 1) human resources, 2) upstream and downstream supporting resources, and 3) clinical trial resources, which are abundant and comparatively much low-cost compared to Europe and the United States. Strictly adhere to the ICH system and the regulations of China’s NMPA, the U.S. FDA, and the EU’s EMA. Although the technical threshold for CGT new drug processes is high, the end product, which is human cells, does not face direct patent restrictions like traditional drugs. Actively establish patents or know-how technologies for our own aCGT process, including compensated contracts for obtaining licenses for patented foundational technologies such as iPSC master cells. For CGT products under clinical trials that have excellent post-Phase II data in Europe and the U.S., as well as some innovative products, implement large-scale me-better and innovative R&D and global submissions.

As of now, aCGT has the following competitive advantages:

1.3.1. Three Major Technology Platforms: aCGT simultaneously possesses three major technology platforms and product pipelines: ① Mesenchymal Stem Cells (MSC), ② Induced Pluripotent Stem Cells (iPSC), and ③ Gene Editing.

1.3.2. Rich R&D Resources: Utilizing abundant and low-cost R&D resources in China (where results are accepted by the U.S. FDA and EU EMA), aCGT has a diverse product pipeline.

1.3.3. University Integration: The company integrates university resources, resulting in a strong product reserve.

1.3.4. Superior Product Data: Fast-follow/me-better product R&D data surpass those of similar products in Europe and the U.S., alongside some first-in-class and innovative products.

1.3.5. High-Quality, Low-Cost Production: The industrial-scale, closed, continuous bioreactor for cell expansion and differentiation processes ensures high quality at low cost, providing an absolute competitive advantage in market sales.

1.3.6. Simultaneous Global Submissions: aCGT applies for approvals in China, the U.S., and Europe simultaneously, tapping into significant market potential.

1.4. 投资要素表Teaser、商业计划书Business Plan(PPT) & etc.

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