撰文丨王聪 编辑丨王多鱼 排版丨水成文 核酸适配体 （Aptamer） 是一类短的单链 DNA 或 RNA 寡核苷酸，通过形成三维空间结构，靶向结合特定蛋白或者细胞发挥作用， 其具有广泛优点，包括高度 特异性、易于合成、可定制、热稳定性好、分子小、组织穿透能力强等。成为选择性靶向特定蛋白质或细胞以治疗多种疾病类型的新选择。 近日， 中国科学院杭州医学研究所 谭蔚泓 院士团队连续发表了 3 篇论文，开发了多款基于 核酸适配体 （Aptamer） 的候选药物，在 阿尔茨海默病 以及包括 三阴性乳腺癌 在内的多种癌症类型中显示出了有潜力的治疗效果。 Apt-M，治疗阿尔茨海默病 2025 年 10 月 1 日，中国科学院杭州医学研究所 谭蔚泓 院士、 邱丽萍 研究员团队在 Nature 子刊 Nature Biomedical Engineering 上发表了题为 ： Targeted clearance of extracellular Tau using aptamer-armed monocytes alleviates neuroinflammation in mice with Alzheimer's d ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 阿尔茨海默病 （AD） 是最常见的痴呆症类型，其特征是记忆和认知功能逐渐衰退。临床发现表明，细胞外的 β-淀粉样蛋白 （Aβ） 沉积和神经元内的 神经原纤 维缠结 的 形成与阿尔茨海默病 （AD） 病理生理学有关，然而，靶向这些病理特征的治疗方法，却未能阻止或逆转神经退行性病变的过程。 现在，研究人员不再仅仅关注特定的以神经元为中心的特征，而是出现了一种整体观——多种紊乱事件的复杂相互作用导致了阿尔茨海默病中神经级联反应的逐 渐失调。从这个角度来看，在易受损的神经网络中识别并靶向易感节点进行干预，有望为阿尔茨海默病 （AD） 的防治提供新机会。 2025 年 10 月 1 日，中国科学院杭州医学研究所 谭蔚泓 院士/ 邱丽萍 研究员团队在 Nature 子刊 Nature Biomedical Engineering 上发表了题为 ： Targeted clearance of extracellular Tau using aptamer-armed monocytes alleviates neuroinflammation in mice with Alzheim ...

Core Points - Mesoblast Limited announced that the Healthcare Common Procedure Coding System (HCPCS) J-Code J3402 for Ryoncil became active for billing and reimbursement on October 1, 2025, marking a significant milestone for the product [1][2] - The permanent J-Code provides a standardized billing pathway for Ryoncil, facilitating reimbursement and improving patient access [2] - Ryoncil is the first FDA-approved mesenchymal stromal cell (MSC) product for any indication and is specifically approved for children under 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD) [3][6] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing a proprietary mesenchymal lineage cell therapy technology platform [5] - The company is committed to developing additional cell therapies for various indications, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease, as well as heart failure and chronic low back pain [7] - Mesoblast has a strong intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection expected to extend through at least 2041 in major markets [8]

Core Viewpoint - China is creating a rich ecosystem for life sciences innovation, presenting more opportunities than challenges for companies like AstraZeneca [2][4] Group 1: China's Innovation Landscape - China has made significant progress in new molecular drugs, AI applications, and cell and gene therapies, attracting multinational companies to expand their R&D presence [3] - The country has established high-level research platforms and nurtured innovative talent, forming unique research advantages [3] - China is actively promoting the integration of basic research with clinical needs and participating in global scientific collaboration [3] Group 2: AstraZeneca's Commitment to China - AstraZeneca has established its sixth global strategic R&D center in Beijing, emphasizing its commitment to China's scientific innovation capabilities [4] - The global R&D team in China is responsible for 20% of AstraZeneca's overall clinical R&D tasks, with nearly 20 global clinical trial projects led by this team [4] - AstraZeneca plans to support the establishment of approximately 800 diagnostic centers across China by the end of 2025 to enhance standardized treatment levels [6] Group 3: Collaboration and Future Prospects - AstraZeneca has engaged in 15 collaborations with 14 Chinese innovative drug companies over the past two years, indicating a vibrant life sciences ecosystem in China [5] - The launch of the "AstraZeneca Global Chronic and Rare Disease R&D Postdoctoral Program" aims to foster future leaders in drug discovery [6] - AstraZeneca aims to leverage China's research capabilities to accelerate the market introduction of early-stage drugs developed by Chinese scientists [6]

Core Insights - CAR-T therapy has emerged as a promising treatment for certain types of cancers, particularly blood cancers, showing significant efficacy in patient outcomes [1][2][3] - The high cost of CAR-T therapy, typically ranging from 1 million to 1.3 million RMB, poses a significant barrier for many patients, as it is not covered by insurance [2][10] - There are ongoing discussions and research aimed at making CAR-T therapy more accessible through cost reduction and expanding its applications beyond blood cancers [12][14][15] Group 1: CAR-T Therapy Overview - CAR-T therapy is an advanced immunotherapy that enhances the patient's own T cells to target and eliminate cancer cells, demonstrating remarkable success in treating blood cancers [1][4][8] - Currently, there are six CAR-T products available in China, primarily for leukemia, lymphoma, and multiple myeloma, with some achieving over 50% ten-year survival rates [2] Group 2: Economic Barriers - The cost of CAR-T therapy is a major concern, with prices in China around 1.29 million RMB per patient, making it unaffordable for many [10][12] - Patients often resort to "compassionate CAR-T" treatments, which are experimental and funded by research institutions, but access is limited due to strict eligibility criteria [10][11] Group 3: Research and Development Challenges - The high cost of CAR-T therapy is attributed to the complex manufacturing process, reliance on imported materials, and the small patient population, which makes cost distribution challenging [11][12] - Experts suggest that developing universal CAR-T products could significantly reduce costs and improve accessibility, but this requires overcoming immune rejection issues [14][15] Group 4: Future Directions - Expanding the indications for CAR-T therapy to include solid tumors and other diseases could increase its patient base and help lower costs through economies of scale [15] - The introduction of a dual-directory system for insurance coverage may provide more options for patients while supporting pharmaceutical innovation [13][14]

Core Insights - BioCardia, Inc. announced positive primary endpoint results from the CardiAMP Cell Therapy in Chronic Myocardial Ischemia Trial, indicating the therapy's potential in treating refractory angina [1][2][4] Company Overview - BioCardia, Inc. is a leader in cellular and cell-derived therapeutics for cardiovascular and pulmonary diseases, with a focus on developing innovative therapies like CardiAMP and CardiALLO [7] Clinical Trial Results - The open-label roll-in cohort showed that the CardiAMP Cell Therapy was well tolerated, with no major adverse cardiac events reported [2] - Patients experienced an average increase in exercise tolerance of 80 seconds and an 82% reduction in angina episodes at the six-month primary endpoint compared to pre-treatment levels [2] - 60% of patients demonstrated substantial improvements in both exercise tolerance and angina episodes [2] Comparison with Existing Therapies - Early results from the CardiAMP therapy compare favorably with current FDA-approved therapies such as Ranolazine and Enhanced External Counter Pulsation (EECP), which also showed significant reductions in angina episodes [3] Unmet Medical Need - Chronic myocardial ischemia with refractory angina affects an estimated 600,000 to 1.8 million patients in the U.S., highlighting a significant unmet need for effective therapies [5] - Up to 15% of patients with ischemia or angina are suboptimal candidates for conventional revascularization, indicating a demand for new treatment options [5] Therapy Mechanism - CardiAMP Cell Therapy utilizes a patient's own bone marrow cells delivered via a minimally invasive procedure, aiming to stimulate the body's natural healing response [6] - The therapy incorporates unique elements such as pre-procedural cell analysis, high target dosage, and a proprietary delivery system, enhancing safety and cell retention [6]

Company Overview - Arcellx was founded approximately 10 years ago with the aim of addressing three primary needs in cell therapy: safety, efficacy, and scale, along with reliable manufacturability to enhance accessibility [2] - The company has developed a core technology known as the D-Domain, which is utilized in its lead asset, anito-cel, considered a best-in-class therapeutic option for patients with multiple myeloma [2] Pipeline and Development - Arcellx is actively leveraging its technology in ongoing clinical trials for acute myeloid leukemia (AML) and myasthenia gravis, indicating a commitment to expanding its therapeutic applications [3]

Bio-Techne FY Conference Summary Company Overview - **Company**: Bio-Techne (NasdaqGS: TECH) - **Industry**: Life Science Tools Key Points Company Positioning and Growth Strategy - Bio-Techne is a leader in reagents for life science research, focusing on proteins, antibodies, and assays for diagnostics and therapeutics [4][6] - The company has invested heavily in automation of protein analytics, spatial biology, and cell therapy, which now account for approximately 45% of revenue, up from 30% pre-COVID [6][7] - 90% of Bio-Techne's business is recurring, primarily from consumables, which supports sustainable growth [7] Recent Financial Performance - The company reported a 3% growth in the most recent quarter, with notable performance in China (10% growth) and continued double-digit growth in large pharma [9][10] - Challenges included delays in shipping high-priced instruments due to geopolitical issues [9] Academic Market Exposure - Academic sales account for about 20% of total revenue, with U.S. academia contributing approximately 11-12% [13][14] - Despite funding constraints, consumables have remained flat, indicating resilience in the product portfolio [14][15] Market Dynamics in China - Positive sentiment in China suggests a gradual ramp-up in growth, with expectations of returning to double-digit growth in the long term [18][19] Biopharma Market Insights - Biopharma constitutes roughly 50% of revenue, with large pharma showing strong recovery and growth, while biotech funding has decreased significantly [20][21] - The uncertainty in biotech funding may impact future spending, but recent improvements in funding could signal a recovery [22] Cell Therapy Business - Bio-Techne is well-positioned in the cell therapy market, focusing on GMP proteins, with a significant increase in revenue from this segment [23][24] - The company has built a strong reputation for quality and has a competitive edge in the regen med space, which has greater market potential than CAR-T therapies [25][26] Core Reagents and Instrumentation - The core reagents business remains stable, with healthy competition in the antibody market [30][31] - The ProteinSimple platform has shown resilience, with growth driven by increased usage of consumables despite a downturn in new instrument placements [34][36] Spatial Biology and Competitive Position - Bio-Techne leads in spatial biology with RNAscope reagents and the new COMET instrument, which automates RNA and protein detection [39][41] - The company is positioned to capitalize on the growing demand for translational research tools [40] Strategic Divestiture - The recent divestiture of the Exosome Diagnostics business was driven by a misalignment with Bio-Techne's core capabilities and a lack of a clear path to profitability [43][45] Revenue Outlook and Market Conditions - The company anticipates low single-digit growth, managing costs and investments amid market uncertainties [47][50] - Optimism remains regarding recovery in key markets, with expectations that administrative-induced noise will settle down [51][53] Conclusion Bio-Techne is strategically positioned for growth in the life science tools industry, with a strong focus on automation, spatial biology, and cell therapy. Despite current market challenges, the company maintains a positive outlook for future performance driven by its recurring revenue model and innovative product offerings.

Financial Data and Key Metrics Changes - The company has revised its revenue guidance for FY 2025 to a range of $250 to $300 million, reflecting a reset based on learnings from the market [4][6] - There was a quarter-over-quarter growth of 24% in Q2, indicating a ramp-up in performance [6] Business Line Data and Key Metrics Changes - The company is focusing on improving patient access to its product, Amtagvi, by adding authorized treatment centers and large community centers [6][7] - The average time for financial clearance has improved significantly to around three weeks, with efforts to streamline the patient journey from ordering to infusion [9][10] Market Data and Key Metrics Changes - The company is targeting a global addressable patient population of approximately 30,000 patients per year for Amtagvi [23] - The company has identified a significant opportunity in large community practices that have grown in recent years, which were previously not targeted [16][18] Company Strategy and Development Direction - The company is committed to financial discipline and has undertaken a restructuring to right-size the organization, aiming for profitability as soon as possible [5][32] - The company is exploring a virtual control arm for its European Medicines Agency application to expedite the approval process [25][27] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, citing the effectiveness of the product and ongoing improvements in manufacturing and reimbursement processes [4][5] - The company is focused on capturing earlier-stage patients to improve treatment outcomes, supported by real-world evidence showing better results than clinical trials [12][15] Other Important Information - The company has a pipeline that includes trials for non-small cell lung cancer and endometrial cancer, with expectations for data releases later this year [38][51] - The company is also working on improving gross margins through operational efficiencies and cost control measures [32][35] Q&A Session Summary Question: What are the key initiatives to achieve the revised revenue guidance? - The company is ramping up external-facing initiatives and adding treatment centers to improve patient access [6][7] Question: What improvements have been made in the patient journey? - Financial clearance times have improved, and the company is working to streamline scheduling and infusion processes [9][10] Question: What is the potential for Amtagvi in the U.S. market? - The company believes Amtagvi has the potential to reach $1 billion in sales, contingent on effective patient access and education initiatives [23] Question: What is the status of the European Medicines Agency application? - The company withdrew its application to gather necessary data for a virtual control arm and plans to resubmit [25][27] Question: What are the expectations for gross margin improvements? - The company anticipates significant savings from restructuring and operational efficiencies, aiming for improved gross margins [32][35] Question: What is the competitive landscape for non-small cell lung cancer? - The company sees limited competition in the second-line treatment space, with current options primarily being chemotherapy [43][44] Question: What are the expectations for the endometrial cancer trial? - The company is optimistic about the trial's potential, with plans to release first-in-human data by the end of the year [51][54]

Financial Data and Key Metrics Changes - The company reported a strong second quarter with increased guidance for both top-line and bottom-line growth, driven by strong performance across all business units [3] - The HIV prevention business saw significant growth, with Descovy sales up 37% quarter over quarter [7] Business Line Data and Key Metrics Changes - The launch of YES2Go for HIV prevention has been described as the strongest execution seen in a launch, with 73% unaided awareness and 95% aided awareness [6][7] - The company is on track to achieve 75% coverage in the U.S. by the end of the year, with significant early wins with commercial and Medicaid payers [11][12] Market Data and Key Metrics Changes - The HIV prevention market is currently underpenetrated, with only 50,000 to 400,000 patients on the drug, compared to a potential 1.2 to 3 million [25] - The company anticipates significant market growth as awareness and access to HIV prevention therapies increase [29] Company Strategy and Development Direction - The company is focused on expanding its HIV prevention market, targeting individuals already on HIV prevention therapies and those at risk of HIV [27][30] - The company is also investing in its oncology pipeline, with multiple launches expected, including a CAR T therapy for multiple myeloma [56][65] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing growth of the HIV treatment business despite headwinds from Part D reforms, expecting a 3% growth rate [34][38] - The company is preparing for potential macroeconomic headwinds but believes its strong clinical profile will help mitigate risks [51][52] Other Important Information - The company has made substantial investments in internal research and development, enhancing the quality and breadth of its portfolio [72] - The approval of a reimbursement code (J code) for YES2Go was granted earlier than expected, which is crucial for the launch [13] Q&A Session Summary Question: How is the launch of YES2Go performing? - The launch has been strong, with high awareness and a solid execution plan in place [5][6] Question: What challenges are anticipated with the new injectable model? - The company acknowledges logistical challenges but is confident in its execution strategy and coverage goals [9][10] Question: How does the company view the competitive landscape for HIV treatments? - Management believes Biktarvy remains the gold standard and expects to maintain market share despite upcoming generic competition [40][41] Question: What is the outlook for the oncology pipeline? - The company is optimistic about the potential of its CAR T therapy and other oncology products, viewing them as significant growth opportunities [56][65] Question: How is the company preparing for potential Medicaid funding cuts? - The company is actively engaging with policymakers and believes it can manage any changes due to its strong clinical data [51][52]