证券代码：301080 证券简称：百普赛斯 北京百普赛斯生物科技股份有限公司 投资者关系活动记录表 编号：2025-003 值得特别关注的是，公司在拥有 GMP 级别质量管理体系平台基础上， 结合细胞治疗药物生产规范，以严格的质量管理和药品级放行检测标准，已 成功开发超 50 款高质量的 GMP 级别产品，包括细胞因子、细胞激活用抗体 和磁珠、全能核酸酶以及 Cas 酶等，适用于 CGT 药物 CMC、商业化生产和 临床研究。公司还可提供用于临床前研究的 Premium (Pre-GMP) 级别原料， 助力客户实现从临床前开发到临床阶段的无缝过渡。另外，公司针对细胞治 疗应用进行针对性的蛋白结构设计，能够提供从基因合成、载体构建到蛋白 质表达、纯化的 GMP 级别蛋白的一站式开发服务。 问：公司在 ADC 领域的布局？ 抗体偶联药物（ADC）作为新一代抗癌免疫治疗药物的代表，其独特之 处在于将高靶向性的抗体与强大的化疗药物相结合。这种结合方式不仅能在 肿瘤细胞内部实现精准投药，有效避免化疗药物对正常细胞的伤害，还显著 减少治疗过程中的不良反应。鉴于 ADC 在肿瘤等疾病治疗中展现出的巨大 潜力，现已成为抗体药 ...

2025.06. 20 本文字数：1762，阅读时长大约3分钟 作者 | 第一财经 吴斯旻 去年，我国新药注册临床试验数量再创历史新高。其中，细胞与基因治疗药物、"跨界药王"司美格鲁肽以 及儿童新药研发的市场热度高涨。 19日，国家药监局药审中心（CDE）发布《中国新药注册临床试验现状年度报告（2024年）》（下 称"报告"）。CDE表示，该报告旨在展示中国新药注册临床试验现状，进一步提升临床试验的透明度，为 新药研发与审评审批提供科学参考。报告分析基于药物临床试验登记与信息公示平台的临床试验数据。 第一财经梳理近五年CDE年度报告发现，2024年，我国新药注册临床试验数量为近五年新高。继2023 年"药物临床试验登记与信息公示平台登记临床试验总量首次突破4000项"后，2024年这一数量逼近 5000项（4900项），较上一年增长了13.9%。 ▲来源：《中国新药注册临床试验现状年度报告（2024年）》 生物制品开展临床试验数量前10位品种中，司美格鲁肽注射液同样开展临床试验数量最多，有15项。也 就是说，去年我国司美格鲁肽临床试验注册数量已达29项。 ▲来源：《中国新药注册临床试验现状年度报告（2024年 ...

在今天(12日)的发布会上，国家发展改革委相关负责人表示，深圳综合改革试点实施近五年来，各项改 革任务扎实有序推进、取得了重要进展。在推动创新发展方面，深圳推出了一大批创新举措，率先开展 本外币合一跨境资金池业务试点，允许跨国集团在境内办理境外成员企业本外币集中收付业务，实现币 种自选、划转自由、资金混用，截至今年5月底，55家跨国公司参与了试点，业务额达到了3400亿美 元。同时，金融支持科技创新的体制机制不断完善，已累计发行知识产权证券化产品104单，发行规模 225亿元，惠及企业1535家，有效支持了科技创新型企业的发展。国家发展改革委副主任李春临表示， 在推进试点过程中，深圳相继推出了人工智能产业促进条例、智能网联汽车管理条例、数据条例、细胞 和基因产业促进条例、个人破产条例、低空经济产业促进条例等一批具有全国首创性、引领性的经济特 区法规，多项法规填补了立法空白，为国家相关领域立法提供了第一手的经验。(央视新闻) ...

Abeona Therapeutics (ABEO) Q1 2025 Earnings Call May 15, 2025 07:00 AM ET Company Participants Greg Gin - Vice President, Investor Relations and Corporate CommunicationsVishwas Seshadri - CEO & DirectorMadhav Vasanthavada - Senior VP, Chief Commercial Officer & Head of Business DevelopmentJoseph Vazzano - Chief Financial OfficerAmin Makarem - Vice President, Biotech Equity ResearchRichard Miller - VP - Biotech Equity ResearchStephen Willey - Managing DirectorRaghuram Selvaraju - Managing Director, Healthcar ...

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024, before accounting for the proceeds from the Priority Review Voucher (PRV) sale [17] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [18] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [18] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [19] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [4][5] - The first Qualified Treatment Center (QTC) activated was Lurie Children's Hospital of Chicago, which is expected to treat the first patient in Q3 2025 [6][8] Market Data and Key Metrics Changes - The company has received inquiries from approximately 30 patients and families across the country since ZivaSkin's approval, indicating strong demand [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [16] Company Strategy and Development Direction - The company aims to treat 10 to 14 patients with ZivaSkin in 2025 and anticipates a robust start to 2026 [13] - The company has entered into an agreement to sell the PRV for $155 million, which will strengthen its balance sheet and fund operations for over two years [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [10][13] - The company is focused on ensuring that the value of ZivaSkin is recognized by payers and that patients have timely access to the therapy [16] Other Important Information - The company expects to achieve profitability driven by ZivaSkin revenues in early 2026 [17] - The company is on track to activate additional QTCs by the end of 2025, with ongoing discussions with recognized institutions [12] Q&A Session Summary Question: What does site activation mean? - Site activation means that staff and surgeons are trained and ready to identify patients for ZivaSkin treatment [22][25] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen patients treated at Lurie, with potential for more referrals from the local community [26] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [29] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be two patients per month per site, with potential to ramp up to four patients per month at some centers [39] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be about $152 million [54] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [72][75]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was $8.5 million, an 11% increase from $7.6 million in Q1 2024 [7] - Gross margin improved to 60%, up from 59% in Q1 2024 [10] - Cash and cash equivalents decreased to $12.4 million from $20.1 million at the end of 2024 [11] Business Line Data and Key Metrics Changes - Biologics and Drug Delivery revenue increased by 9% to $4.7 million, driven by a $1.2 million increase in product revenue [8] - Neurosurgery Navigation revenue surged 70% to $3.3 million, attributed to higher sales of new product offerings [9] - Capital Equipment and Software revenue fell 63% to $500,000 due to fewer new placements of navigation and laser units [10] Market Data and Key Metrics Changes - The company activated two new sites in Q1 2025, which were both capital placements [51] - The introduction of the three point zero navigation software has allowed expansion into operating rooms, enhancing market reach [14] Company Strategy and Development Direction - The company aims to extend its lead in cell and gene therapy through a comprehensive drug delivery ecosystem [4] - Plans to evolve the product portfolio to focus on fast, simple, and predictable workflows to increase hospital efficiency [5] - The company is pursuing global scale by expanding its installed base and generating more access to its ecosystem [5] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential for additional cell and gene therapy platforms to gain approvals in the next two years [16] - The recent partnership with Overland Capital provides the company with the necessary funding to execute its strategy [6] - Management anticipates that operational cash burn will continue to increase but expects to generate meaningful cash flow as new drugs come online [31] Other Important Information - The company has implemented a Pathfinder subscription program to smooth capital revenue recognition over time [22] - The three point zero navigation software has been well received, with positive feedback from surgeons regarding efficiency and accuracy [18] Q&A Session Questions and Answers Question: Are there initiatives that can be prioritized now with access to incremental capital? - Management confirmed that there are opportunities to accelerate hiring and expand preclinical resources in response to partner requests [29] Question: How does the new capital runway relate to achieving sustained breakeven? - Management believes the new debt facility can support the company towards cash breakeven, especially with the flexibility of an interest-only period [31] Question: How is the neuro franchise tracking relative to expectations? - Management stated that the neuro franchise is on plan and expects significant growth throughout the year, with potential upside from new product launches [34] Question: What is the expected mix of MRI versus OR procedures in the future? - Management indicated that while MRI procedures currently dominate, there is potential for OR procedures to grow faster as new therapies are introduced [40] Question: What is the status of GLP certification and customer interest? - Management confirmed that they are on track for GLP certification by the end of the year and noted strong interest from pharma partners for studies once certified [42][46]

世界正迅速进入一个激动人心的医学新时代。曾经设计用于治疗数百万人的大型药物时代，正快速过渡到 个性化治疗的新纪元。依靠普适法则确保治疗方案适用于大多数受疗者，虽然惠及了无数人，但也使得许 多"非典型"患者无法获得有效的治疗解决方案——同时，支付方为无效治疗支付了大量资金。 在2021年，细胞和基因疗法（Cell and Gene Therapies, CGT）仅占全球药物市场总收入的0.8%。但随着超 过500种CGT进入研发状态，它们正准备在与传统技术的竞争中占据越来越大的市场份额。我们估计，全 球CGT市场将在2021年至2026年间以每年超过50%的年化增速增长，届时将有近200种疗法产生约570亿美 元的销售额，占全球药物市场的4%以上（见图1）。确实，CGT正逐步发展成为标准治疗的下一个浪潮， 瞄准一些现代社会中最常见的疾病，包括镰状细胞贫血、2型糖尿病和阿尔茨海默症。 美国FDA和其他监管机构已经批准了CGT（细胞与基因治疗）用于治疗冠状动脉疾病、遗传性视网膜病、 大B细胞淋巴瘤和脊髓性肌萎缩症等多种病理现象。 然而，最近的上市情况喜忧参半，公司在摸索前进时面临着未知的挑战，包括与往往百万美元的定 ...

Start Time: 17:00 January 1, 0000 5:41 PM ET Cryoport, Inc. (NASDAQ:CYRX) Q4 2024 Earnings Conference Call March 04, 2025, 17:00 PM ET Company Participants Jerrell Shelton - CEO Robert Stefanovich - CFO Mark Sawicki - Chief Scientific Officer Thomas Heinzen - VP IR and Corporate Development Todd Fromer - IR, KCSA Strategic Communications Conference Call Participants Anna Snopkowski - KeyBanc Capital Markets Puneet Souda - Leerink Partners David Saxon - Needham Subbu Nambi - Guggenheim David Larson - BTIG Ri ...