基因治疗
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西湖大学开发基因递送黑科技,全球首个哺乳动物蛋白凝聚体递送系统丨ASGCT 口头报告精选
生物世界· 2025-05-26 02:52
Core Viewpoint - The EASY system, developed by Westlake University and the Westlake Coacervate Research Team, is a novel non-viral, non-liposomal nucleic acid delivery platform that has been recognized at the American Society of Gene & Cell Therapy (ASGCT) annual meeting, highlighting its potential in gene delivery applications [1][3][19]. Group 1: Introduction to EASY System - The EASY system is the first mammalian coacervate-based delivery system, designed to efficiently deliver various types of nucleic acids, including plasmid DNA, mRNA, and CRISPR-Cas9 tools [1][5]. - The system is inspired by natural cellular coacervates, which are membrane-less organelles formed through liquid-liquid phase separation, playing crucial roles in various biological processes [6]. Group 2: Mechanism and Advantages - The core of the EASY system is the engineered endogenous protein, ProteanFect™, which self-assembles with nucleic acids to form highly ordered nanoparticle structures [7]. - The delivery process involves the formation of stable nanoparticles, efficient cellular uptake through active endocytosis, intracellular release of nucleic acids, and safe degradation of carrier proteins via natural cellular pathways [8]. - The EASY system offers revolutionary advantages, including: 1. Ultra-high loading capacity, approximately 50 times that of lipid nanoparticles (LNP) [9]. 2. Compatibility with various nucleic acids, enabling the delivery of plasmid DNA, mRNA, siRNA, sgRNA, and Cas9 mRNA [10]. 3. High efficiency and low toxicity, achieving comparable or higher gene delivery efficiency while significantly reducing cell mortality [10]. 4. Broad applicability to various primary cells, including NK cells, B cells, and hematopoietic stem cells [10]. Group 3: Commercialization and Clinical Relevance - The ProteanFect series of kits represents the first commercial product of the EASY system, enabling efficient gene delivery and precise gene editing in various primary and hard-to-transfect cells [13]. - The system has demonstrated high gene editing efficiency in primary T cells, with results showing a range of 67%-88% efficiency for gene marking and over 90% efficiency for gene editing [18]. Group 4: Future Outlook - As gene therapy approaches critical clinical applications, the safety and efficacy of delivery systems are paramount. The EASY system provides a new solution by leveraging the natural biological mechanisms of coacervates [19]. - The international recognition of this technology not only affirms China's scientific innovation but also brings new hope to the global gene therapy field, with expectations for the EASY system to play an increasingly important role in clinical applications [19].
科普|儿童肾病40%与遗传相关,细胞疗法后基因治疗曙光初现
Di Yi Cai Jing· 2025-05-23 14:22
沈茜告诉第一财经记者,除了细胞治疗之外,在复旦儿科医院肾脏科和风湿科学科带头人徐虹教授的带 领下,一种目前用于法布雷病的基因疗法未来也有望用于肾病治疗。目前该疗法已经进入IIT(研究者 发起研究)阶段。 除了细胞治疗之外,在复旦儿科医院肾脏科和风湿科学科带头人徐虹教授的带领下,一种目前用于法布 雷病的基因疗法未来也有望用于肾病治疗。目前该疗法已经进入IIT阶段。 近日,浙江大学医学院附属儿童医院肾脏内科公布消息称,一名多药耐药型肾病综合征患儿接受了一种 靶向BCMA/CD70自体CAR-T细胞治疗后,在回输的第3个月,尿蛋白11年来首次转阴。 这一病例的救治成功意味着CAR-T细胞治疗在治疗儿童肾病方面迈出重要的一步,有望帮助更多疑难疾 病患儿实现治愈。 此前,细胞治疗已经用于儿童系统性红斑狼疮以及难治性幼年皮肌炎等疾病。 5月22日,在复旦大学附属儿科医院肾脏科的一场儿童肾病公益项目中,复旦大学附属儿科医院肾脏科 主任沈茜教授对第一财经记者表示:"系统性红斑狼疮会导致狼疮性肾炎,肾炎会影响肾功能,如果不 及时治疗就会发展成为尿毒症。" "法布雷病是一种基因遗传性疾病,会导致包括肾衰竭在内的多种脏器的功能损伤 ...
诊断治疗双突破:环状核酸引领精准医疗新浪潮
生物世界· 2025-05-21 07:35
近年来, 环状寡核苷酸 凭借 其独特的闭环结构和卓越的生物稳定性, 逐渐 成为生物医学领域的研究热 点。这类分子主要包括 环状 RNA (circRNA) 和 环状单链 DNA ( C ssDNA) ,在疾病诊断和治疗中 展现出广阔的应用前景,正推动精准医疗 迈向新高度 。 环状寡核苷酸为何备受 瞩目 ? 1、 疫苗 开发 :利用 circRNA 编码抗原蛋白,并通过脂质纳米颗粒封装后注射到体内。circRNA 进入细 胞后翻译出相应的抗原蛋白,最终由抗原递呈细胞 (APC) 呈递,引发免疫应答。 2、 基因表达调控 : 合成具有特定功能的 circRNA,例如 miRNA 海绵、蛋白海绵、基因沉默等。通过纳 米颗粒递送到靶细胞后,这些 circRNA 可在细胞内执行相应的基因调控功能。 3、 细胞治疗 :利用 circRNA 编码 CAR (嵌合抗原受体) 或 TCR (T 细胞受体) 结构蛋白,并封装于 靶向纳米颗粒中。通过静脉注射,可直接在体内原位生成 CAR-T 或 TCR-T 细胞,无需复杂的体外病毒改 造过程, 能够显著 提高开发效率、降低开发成本。 环状寡核苷酸 ( Circular Oligo ...
2024年十大畅销基因疗法,仅3款销售额超1亿美元
生物世界· 2025-05-17 06:32
Core Viewpoint - The current gene therapy market is characterized by both significant investment opportunities and notable challenges, reflecting a duality in its development landscape [2][3]. Investment Highlights - As of mid-April 2023, five gene therapy companies have collectively secured $534.4 million in venture capital funding, indicating strong investor interest in the sector [2]. - Notable funding rounds include Tune Therapeutics raising over $175 million in January, and Atsena Therapeutics completing a $150 million oversubscribed round in April [2]. Market Challenges - AmplifyBio, a CRO/CDMO focused on gene and cell therapies, shut down in April 2023 after four years of operation, highlighting funding shortages faced by early-stage biotech companies [3]. - Despite over ten gene therapies receiving FDA approval, only one is projected to exceed $1 billion in sales in 2024, with only three therapies expected to surpass $100 million in sales [3]. Top Gene Therapies by Projected Sales 1. **Zolgensma®**: Projected sales of $1.214 billion in 2024, developed by Novartis, for treating spinal muscular atrophy in children under 2 years old [12]. 2. **Elevidys®**: Projected sales of $820.791 million in 2024, developed by Sarepta Therapeutics, for treating Duchenne muscular dystrophy [13]. 3. **Vyjuvek®**: Projected sales of $290.5 million in 2024, developed by Krystal Biotech, for treating epidermolysis bullosa [13]. 4. **Adstiladrin®**: Projected sales of €70 million ($79 million) in 2024, developed by Ferring Pharmaceuticals, for treating high-risk non-muscle invasive bladder cancer [14]. 5. **Zynteglo™**: Projected sales of $62.273 million in 2024, developed by bluebird bio, for treating beta-thalassemia [14]. 6. **Roctavian®**: Projected sales of $26 million in 2024, developed by BioMarin Pharmaceutical, for treating severe hemophilia A [14]. 7. **Lenmeldy™ / Libmeldy™**: Projected sales of $224.54 million in 2024, developed by Orchard Therapeutics, for treating metachromatic leukodystrophy [14]. 8. **Luxturna®**: Projected sales of CHF 18 million ($20.9 million) in 2024, developed by Spark Therapeutics, for treating RPE65 mutation-associated retinal dystrophy [14]. 9. **Lyfgenia®**: Projected sales of $11.605 million in 2024, developed by bluebird bio, for treating sickle cell disease [16]. 10. **Skysona®**: Projected sales of $9.917 million in 2024, developed by bluebird bio, for treating cerebral adrenoleukodystrophy [16].
康弘药业(002773) - 2025年5月15日投资者关系活动记录表
2025-05-15 09:54
Group 1: Clinical Trials and Research - KH631 and KH658 are currently in clinical trials, with safety and efficacy data not yet fully compiled [2] - KH631's clinical data is expected to be disclosed at the 2025 ARVO conference [2] - The company has a diverse R&D system covering traditional Chinese medicine, antibody drugs, gene therapy, and small molecule drugs [10] Group 2: Financial Performance and Projections - In 2024, the company achieved a revenue of CNY 4,452,657,799, a year-on-year increase of 12.51%, and a net profit of CNY 1,191,230,760.93, up 14.02% [14] - For 2025, the company projects a revenue growth of 5%-15% and a net profit growth of 5%-15% compared to 2024 [9] - R&D expenses in 2024 increased by 33.57% to CNY 604,378,287.33 [8] Group 3: Strategic Focus and Market Position - The core competitive advantage lies in strategic layout and continuous innovation across various product lines [3] - The company is focusing on enhancing internal control management and performance assessment to improve overall management efficiency [26] - The company maintains a strong market position in the anti-VEGF sector, with confidence in overcoming competition from global brands [6] Group 4: Product Development and Pipeline - Key products in clinical trials include KH631 for nAMD, KH658 for nAMD, and KH617 for advanced solid tumors [5] - The company is advancing the clinical trials of KH110 for Alzheimer's disease and KH109 for anxiety [15] - The company is actively responding to market competition and regulatory changes, particularly in the traditional Chinese medicine sector [8] Group 5: Future Outlook and Industry Trends - The pharmaceutical industry is viewed as a "perpetual sunrise industry" with significant growth potential [13] - The company is committed to increasing R&D investments to provide high-quality, cost-effective products [22] - The company is exploring international collaboration opportunities to enhance its global market presence [30]
健康元药业集团股份有限公司关于 2024年度暨2025年第一季度业绩暨现金分红说明会召开情况的公告
Zhong Guo Zheng Quan Bao - Zhong Zheng Wang· 2025-05-13 01:02
Summary of Key Points Core Viewpoint - The company held a performance and cash dividend briefing for the fiscal year 2024 and the first quarter of 2025, emphasizing its optimistic outlook for the pharmaceutical industry driven by multiple favorable factors [1][2]. Company Performance and Strategy - The company is focusing on innovative drugs as its core development direction, particularly in the respiratory disease treatment area, with over ten innovative drug projects in the pipeline [3][5]. - The company has successfully reserved over twenty innovative drugs covering various important therapeutic areas, with several entering critical clinical phases [3][5]. - The company aims to enhance its operational efficiency and resource utilization to achieve sustainable growth [6]. Industry Outlook - The pharmaceutical industry is expected to experience medium to high-speed growth, with significant improvements in innovation capabilities and modernization of the industrial chain by 2025 [5]. - The industry reported a total revenue of 25,298.5 billion yuan in 2024, with a slight decrease in total profit by 1.1% year-on-year due to temporary adjustments [5]. Financial Performance - The company reported a steady performance in the first quarter of 2025, despite facing some pressure from industry policy adjustments [9]. - The company's R&D expenses for 2024 are projected to be around 400 million yuan, reflecting a year-on-year increase of approximately 30% [8]. Market Development - The company is actively working on market expansion for its newly approved products, such as the Tobramycin inhalation solution and the Salmeterol/Fluticasone inhalation powder, aiming for significant growth contributions in the coming years [6][9].
红杉中国医疗成员企业再创多项“首个”记录|Healthcare View
红杉汇· 2025-04-29 11:30
金仕生物 全球首款预装干瓣Prostyle A® 获批上市 4月9日,金仕生物宣布由公司自主研发的 Prostyle A®经导管人工主动脉瓣膜置换系统-干法预装可回收输送 系统 获得国家药品监督管理局 (NMPA) 批准上市 ( 国械注准20253130729) 。 作为全球首款预装干瓣,金仕生物推出的新一代介入主动脉瓣Prostyle A®集成了众多瓣膜领域的前沿技 术,拥有干瓣技术、巧妙便捷的预装系统、卓越的瓣膜血流动力学性能和耐久性,优秀的输送性和通过 性,平稳的释放和可靠的锚定等特点。金仕生物自主创新的干瓣技术,在国内、欧洲、美国、日本等国家 享有专利保护。这一产品的出现,迎来中国TAVR的新时代 ——干瓣时代。 艾柯医疗 全新血流导向密网支架系统"Lattice COUPLE"成功获批 近日,艾柯医疗全新血流导向密网支架系统"Lattice COUPLE",成功获得国家药监局批准注册 ( 注册证编 号:国械注准20253130668) 。至此, 艾柯医疗成为神经介入行业首家拥有三张密网注册证的企业 。 微光医疗 FDA 首次批准中国高端腔内介入影像设备 4月11日,微光医疗宣布其两款冠脉OCT设备及 ...
【私募调研记录】彤源投资调研上海家化、卓易信息等4只个股(附名单)
Zheng Quan Zhi Xing· 2025-04-29 00:10
Group 1: Shanghai Jahwa - Shanghai Jahwa is confident about achieving high double-digit GMV growth for its core brands during the 618 shopping festival [1] - The employee stock ownership plan aims to enhance competitiveness, reduce turnover, and foster a sense of ownership among employees [1] - The company has made strategic adjustments in the past six months, including organizational design, talent development, and cultural initiatives [1] - E-commerce strategies include inventory optimization, pricing adjustments, organizational restructuring, and product iteration [1] - The company targets double-digit revenue growth this year and aims to turn profits around [1] - Long-term profit recovery is expected to follow a 1-2 year revenue growth, 2-3 year profit improvement, and 3-5 year profit margin targets [1] Group 2: Zhuoyi Information - Zhuoyi Information has approximately 7,000 active users for its SnapDevelop platform and plans to release a paid commercial version [2] - EazyDevelop, which focuses on rapid development using AI technology, will have a free trial version available after development [2] - The company has made breakthroughs in RISC-V firmware and is collaborating with major firms like Huawei and Lenovo [2] Group 3: Chipbond Technology - Chipbond Technology has made progress in advanced packaging, with its WLP2000 equipment entering mass production preparation after client validation [3] - The company benefits from advantageous direct-write lithography technology and has seen a 22.31% year-on-year increase in revenue and a 30.45% increase in net profit in Q1 [3] - The company expects overseas orders to account for nearly 20% by 2024, indicating strong international market competitiveness [3] Group 4: Kanghong Pharmaceutical - Kanghong Pharmaceutical's KPB-101 has established a leading position in China's ophthalmic anti-VEGF market and aims for further achievements [4] - The company focuses on diverse research and development in ophthalmology, neurology, and oncology, with a strong emphasis on innovative drug sales [4] - The company anticipates a revenue and net profit growth of 5%-15% year-on-year by 2025, with specific products expected to see significant sales growth [4]
康弘药业(002773) - 2025年4月27日投资者关系活动记录表
2025-04-28 09:38
Market Position and Growth Potential - Kanghong Pharmaceutical has established a leading position in the Chinese ophthalmic anti-VEGF market, competing against three major global brands [2] - The company has successfully navigated market challenges over the past decade, demonstrating confidence in continuing to outperform international competitors [2] - The incidence rates of the five major indications for anti-VEGF products are stable, suggesting that Kanghong's growth will align with the overall market trend [2] Research and Development Strategy - The company focuses on key therapeutic areas: ophthalmology, psychiatry/neuroscience, and oncology, with a diversified R&D system covering traditional Chinese medicine, antibody drugs, gene therapy (AAV), and small molecule drugs [3] - Kanghong is advancing multiple innovative projects, including the "Shu Gan Jie Yu Capsule" for anxiety disorders, which is currently in phase III clinical trials [3][7] - The company plans to disclose clinical data for its gene therapy products through international academic journals and conferences [4] Financial Outlook - For 2025, Kanghong anticipates a revenue growth of 5%-15% compared to 2024, with a similar net profit growth forecast [7] - The "Shu Gan Jie Yu Capsule" is expected to see double-digit growth in 2024, outpacing the overall antidepressant market [7] Competitive Landscape and Policy Impact - The competitive landscape for anti-VEGF products has intensified, with four innovative drugs and two biosimilars entering the market following successful negotiations with national health insurance [5] - Kanghong is adapting to national procurement policies, with a significant portion of its chemical drugs selected for national procurement [4] Talent and Collaboration - The company emphasizes talent acquisition and development to support its R&D needs, particularly in gene therapy and advanced drug delivery systems [6] - External collaborations are viewed as a core strategy for long-term value growth, focusing on complementary technologies and commercial synergies [6]
复星医药2024年净利润增长16% 核心产品表现亮眼
Xi Niu Cai Jing· 2025-04-28 08:59
Core Insights - Fosun Pharma reported a revenue of 41.067 billion yuan in 2024, a slight decrease of 0.80% year-on-year, while net profit attributable to shareholders increased by 16.08% to 2.770 billion yuan [1][2] - The company achieved a net cash flow from operating activities of 4.477 billion yuan, reflecting a significant increase of 31.13% compared to the previous year, indicating improved free cash flow to support strategic transformation and R&D investments [2][3] Financial Performance - Revenue: 41.067 billion yuan in 2024, down from 41.400 billion yuan in 2023, a decrease of 0.80% [2] - Net Profit: 2.770 billion yuan in 2024, up from 2.386 billion yuan in 2023, an increase of 16.08% [1][2] - Net Profit (excluding non-recurring items): 2.314 billion yuan, a rise of 15.10% from 2.011 billion yuan in 2023 [2] - Basic Earnings per Share: 1.04 yuan [1] Business Segmentation - Pharmaceutical Revenue: 28.924 billion yuan, a decline of 4.29% year-on-year; key products like Hanquyou® (trastuzumab) saw global sales of 2.810 billion yuan, an increase of 11% [3] - Medical Devices and Diagnostics Revenue: 4.323 billion yuan, down 1.53% due to decreased demand for COVID-19 products [3] - Healthcare Services Revenue: 7.647 billion yuan, an increase of 14.61% driven by specialized construction and smart healthcare initiatives [3] R&D and Innovation - R&D Investment: 5.554 billion yuan, accounting for 13.52% of revenue; seven innovative drugs/biosimilars were approved for market [3] - Notable Approvals: Slulizumab received EU approval, and HLX14 (biosimilar to dezuizumab) is under review in the US and EU [3] - Collaboration: The company is exploring new directions in nuclear medicine and gene therapy through partnerships with industry funds [3] Shareholder Returns - Proposed Dividend: The company plans to distribute a cash dividend of 3.20 yuan per 10 shares (tax included) [3]