Avellino Labs USA, Inc.

9月12日，由美国基因与细胞治疗学会（ASGCT）主办的Molecular Therapy Nucleic Acids学术期刊发表SiSaf公司明星产品SIS-101-ADO的临床前研究论文，证明了该药物在动物体内治疗染色体显性遗传石骨症2型（ADO2）的安全性和有效性。SIS-101-ADO是英国公司SiSaf开发的一款抑制ADO2特异性突变基因Clcn7表达的siRNA与其专有Bio-Courier硅稳定的杂化脂质纳米颗粒（sshLNP）结合的临床前阶段候选药物，用于治疗儿童常染色体显性遗传石骨症2型（ADO2）的严重或危及生命的表现，包括视神经压迫致盲，牙齿和颅面发育异常以及脊柱侧弯。ADO2是基因突变造成的骨密度增加，具有缺陷结构的骨积聚，于是骨变脆且容易骨折。大约70%的ADO2患者由Clcn7（电压门控氯离子通道7）基因杂合错义突变引起，该基因编码CLC-7蛋白，CLC-7蛋白存在于溶酶体和破骨细胞褶皱膜上，其突变对骨和脑的影响最大。在ADO2最严重的形式中，患者表现为广泛的骨硬化，尤其是颅底、骨盆和椎骨，并出现多处骨折、骨髓炎、血液衰竭和感觉症状，包括视力和听力受损。全球每10万人种就有5.5人受到ADO2影响，目前没有批准上市的ADO2的治疗方法，也没有正在临床试验中的其他药物。SiSaf的Bio-Courier技术将专有的可生物吸收硅基质与表面脂质和氨基酸相结合，来稳定脂质纳米颗粒（LNP），这种结构克服了LNP递送的局限性。5月15日，SIS-101-ADO被FDA授予孤儿药指定（ODD）和罕见儿科疾病指定（PRDD）。石骨症患者的骨骼▲ 图片来源：Molecular Therapy Nucleic Acids在携带杂合突变Clcn7基因（Clcn7G213R）的ADO2小鼠模型中通过单次腹膜内注射SIS-101-ADO后，SIS-101-ADO显著下调了Clcn7G213R48小时股骨中的相关mRNA水平。在治疗后2周和4周（3次腹膜内注射/周）观察到确认性结果和骨恢复的表型，安全性也得到证明。▲ 图片来源：Molecular Therapy Nucleic AcidssshLNP硅脂纳米颗粒技术RNA疗法成功的关键在于其递送系统，它需要稳定和保护RNA分子不被降解，并且需要确保足够数量的RNA输送到靶细胞的细胞质，以引起所需的细胞反应，而几乎没有或没有不良影响或积累。LNP，▲ 图片来源：Sisaf官网脂质纳米颗粒（LNPs）是RNA药物采用的目前主流载体，但比较脆弱，当LNP的静电电位发生不良变化时，RNA会分离，过早释放从而无法发挥作用，因此需要超冷储存和运输。此外，LNP使用的高比例阳离子脂质在氧化时会产生毒性，LNP制备过程中使用的聚乙二醇（PEG）会触发抗体（αPEG Abs）反应，机体会将PEG识别为外来物，发起免疫反应来清除。sshLNP，▲ 图片来源：Sisaf官网SiSaf的Bio-Courier 硅稳定的杂化脂质纳米颗粒（sshLNP）RNA递送技术使得RNA更加持久稳定，提高了转染效率，具备非免疫原性、高效转染、无积累的特点。与传统的脂质纳米颗粒不同，它们可以空载体的形式运输，并且可以在单独的步骤中引入RNA。具体来说，有以下优点：1.硅的正电荷和高ζ电位静电结合和凝聚RNA，防止RNA过早解离并减少对潜在有毒阳离子脂质的依赖，减少免疫原性；2.大量紧密缩合的RNA静电键合到硅基质上，保护它们免受水解，延长其全身存活时间；3.硅准金属的结构完整性可防止颗粒的物理塌陷，从而在保持RNA完整性的同时进行冻干，减少了对聚乙二醇化的需求，这是避免超冷供应链存储需求的关键步骤；4.定制的表面脂质与pH依赖性硅/RNA键溶解相结合，最大限度地提高了转染效率；5.粒径、表面电荷和表面配体的添加的多功能性可实现RNA药物被动或主动细胞靶向和多种给药途径。6.涂覆在硅核心上的表面脂质和氨基酸控制硅水解的速度，提供特定细胞和组织的受控释放和靶向性。SiSaf的创始人是领先的生物材料专家Suzanne Saffie-Siebert博士，风险投资者包括Vickers Venture Partners和UK Future Fund，总部位于英国吉尔福德。SiSaf正在开发针对常染色体显性遗传疾病的siRNA治疗方法，包括罕见的骨骼疾病，如石骨症和软骨发育不全。SiSaf最近将其RNA研发管线拓展到肿瘤和眼科适应症，公司与美国眼科遗传病诊断研究公司Avellino Labs合作，开发了用于II型角膜营养不良的滴眼液RNA干扰疗法SIS-201-CD和CRISPR Cas9基因编辑疗法SIS-201-CDC。SiSaf产品管线,▲ 图片来源：Sisaf官网参考资料1.https://sisaf.com2.https://doi.org/10.1016/j.omtn.2023.08.020抗体行业在过去几十年中经历了快速而显著的发展，成为生物医药领域中一个快速发展且备受关注的领域。自20世纪80年代第一种单克隆抗体药物（Muromonab-CD3）获得批准以来，许多抗体药物已经被批准用于各种疾病的治疗，包括肿瘤、免疫性疾病和感染性疾病等。市场上已经有数十种成功抗体药物，并且在临床开发和研究中有更多的候选药物，预计未来将有更多新药上市。如今，除了传统的单克隆抗体，越来越多的新型抗体技术被陆续开发出来，包括双特异性抗体、多克隆抗体、人源化抗体、重组抗体、抗体偶联药物等。这些新技术使得抗体药物具有更高的特异性、亲和力、稳定性和生产可行性，拓宽了抗体的应用范围。今年是COVID-19大流行趋于稳定后的第一年，人民生活逐渐走向正轨，经济也在缓慢但有力的复苏之中，而各抗体企业也依然在不断奋进、不断突破、不断超越，使抗体领域蓬勃发展，硕果累累。为促进抗体行业的交流与创新，2023年10月14-15日第六届金秋十月抗体产业发展大会如约而至。会议旨在为研究人员提供一个互动交流的平台，有助于推动抗体产业的进一步发展。会议内容时间：2023年10月14-15日地点：苏州（酒店定向通知）规模：600-800人主办单位：生物制品圈、抗体圈指导单位：康维讯生物、夏尔巴生物会议费用：免费FREE!（仅收取50元报名定金，含参会学习、茶歇、会议手册，定金概不退还），9月15日报名定金提高到100元，先到先得，报完即止，超过9月20日预登记将收取会议费！报名方式：扫描下方二维码或点击文章最底部“阅读原文”→ 填写表格 → 报名成功（报名志愿者，承担一定工作，请慎重考虑，免交定金）！组委会获得报名信息后，根据报名信息进行初筛，并进一步与报名者沟通确认，实现精准邀请。最终有机会进入大会微信群（严格审核通过）。日程安排更多嘉宾仍在邀请中。。。。。。识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

Enhanced user experience in NGS sample preparation workflow SANTA CLARA, Calif.--(BUSINESS WIRE)-- Agilent Technologies Inc. (NYSE: A) today announced the release of an on-deck thermal cycler (ODTC) that integrates with the Agilent Bravo NGS automated liquid handling platform. The optional ODTC accessory enables the Bravo platform to provide thermal cycling as part of an automated workflow for next-generation sequencing (NGS), end-point PCR, and cell-based applications. An on-deck thermal cycler is critical in many genomics and molecular biology workflows. This optional accessory enables the Bravo NGS automated liquid handling platform to provide thermal cycling as part of an automated protocol and increases "walk-away" time, providing operators with more freedom to focus on higher-level tasks. This setup is beneficial, particularly for applications such as library preparation and target enrichment steps in NGS workflows. "The on-deck thermal cycler enables more seamless workflows on Bravo NGS platforms by eliminating multiple touch points where users must remove a microplate from the Bravo deck and place it on an offline, third-party thermal cycling instrument," said Lars Kristiansen, general manager of Agilent’s Automation Solutions Business. "Adding an ODTC to the Bravo platform extends walk-away opportunities, improves performance, and enhances ease-of-use with preprogrammed protocols. This release supports our customers in high-throughput academic and service provider labs who want to improve productivity and reproducibility while maintaining workflow flexibility." Kevin Kim, head of automation at Avellino Labs, added, "The Bravo with ODTC is a cost-effective and powerful addition to any lab that needs a solution with more walk-away time, smaller footprint, and direct pipetting into the thermal cycler, which is critical for temperature-sensitive assay steps." Kevin Meldrum, general manager of Agilent’s Integrated Genomics Division, added, “With the ODTC on the Bravo, SureSelect users will have an enhanced experience while taking advantage of the flexibility and quality performance of the SureSelect XT HS2 chemistry.” Agilent’s modular workflow solutions are designed to integrate easily and work together seamlessly. The Agilent Bravo NGS workstation with ODTC integrates with SureSelect reagents to provide an automated NGS solution that saves time and minimizes the burden of optimizing new automated assays while providing the flexibility to serve a broad range of application needs. About Agilent Technologies Agilent Technologies Inc. (NYSE: A) is a global leader in the life sciences, diagnostics, and applied chemical markets, delivering insight and innovation that advance the quality of life. Agilent’s full range of solutions includes instruments, software, services, and expertise that provide trusted answers to our customers' most challenging questions. The company generated revenue of $6.85 billion in fiscal 2022 and employs 18,000 people worldwide. Information about Agilent is available at To receive the latest Agilent news, please subscribe to the Agilent Newsroom. Follow Agilent on LinkedIn and Facebook.

June 8, 2022 11:00 UTC Ms. Gambale’s experience in data technology and finance supports Avellino’s bioinformatics precision medicine mission MENLO PARK, Calif.--(BUSINESS WIRE)-- Avellino Labs USA, Inc. (Avellino), a global leader in molecular diagnostics, is pleased to announce the appointment of Virginia Gambale to its Board of Directors. This press release features multimedia. View the full release here: Virginia Gambale, new member of the Board of Directors for Avellino Lab USA. (Photo: Business Wire) Ms. Gambale is currently Managing Partner of Azimuth Partners LLC, which is focused on the use of disruptive technologies to assist transforming or facilitating growth in both middle market and large corporations. She has served on more than 20 public and private boards as Chair, Lead Director and Committee Chair roles, including at JetBlue, Motive, Piper Jaffray, Workbrain, Synchronoss Technologies, and IQ Financial. Ms. Gambale currently serves on several boards, including as Chair of the Board, Director, Chair of Nominating and Corporate Governance, and Audit Committee member at Nutanix; JAMF Audit Committee Member; FD Technologies as their Chair of Nominating and Governance and Audit Committee member; and Virtu Financial as a member of their Audit and Chair of Risk Committee. She is also an Executive Advisor for AttackIQ, a leading cybersecurity optimization platform, and private company director for Banking 10X. “We are thrilled to welcome Virginia to Avellino. Her expertise in both technology and finance will be invaluable as we continue to leverage the power and promise of genetic science and data technology to support our mission to improve human health,” said Avellino CEO Jon Robson. “We are in an exceptional time as the scientific community is making novel discoveries on a continual basis, not to mention advances in areas such as AI and machine learning that are accelerating the pace of this scientific knowledge,” said Ms. Gambale. “It is a privilege to join a leading biotechnology firm that is committed to applying the latest scientific and technological knowledge to help improve health and further unlock the mysteries of the human genome. I look forward to applying my skills and expertise toward their mission to advance how we both diagnose and treat disease.” Ms. Gambale has held significant senior management positions, including as CIO and Managing Director at firms including Merrill Lynch, Bankers Trust, Deutsche Bank and Marsh McLennan. Additionally, she was the Head of Deutsche Bank Strategic Ventures and subsequently a General Partner at Deutsche Bank Capital and ABS Ventures until founding Azimuth Partners in 2003. A former concert pianist, Ms. Gambale was the Board President of the Newport Music Festival from 2014 to 2019. She is also an adjunct faculty member at Columbia University and Mentor for Columbia University’s Master’s in Technology Leadership program. In addition, she is a frequent lecturer and keynote speaker on the strategic use of emerging technologies and cybersecurity and was one of the first National Association of Corporate Directors (NACD) Fellows. Ms. Gambale has a B.S. degree in Mathematics and Computer Science from the New York Institute of Technology. About Avellino Avellino Lab USA, Inc. is a global leader in molecular diagnostics at the forefront of precision medicine for eye care. With a long-term mission to develop personalized approaches to improve health and disease management through genomics, the company is developing a transformative genetic diagnostics product pipeline, as well as genetic therapeutics leveraging CRISPR gene editing, to better manage, and potentially cure, inherited diseases. The company also developed the Avellino SARS-CoV-2 RT-PCR diagnostic test to aid in COVID-19 pandemic testing efforts in the U.S. The first private company in the U.S. to receive EUA for its COVID-19 test, Avellino has performed more than three million COVID-19 related tests to date. Avellino is headquartered in Silicon Valley, California, with operations in Korea, Japan and the UK. To learn more about Avellino, visit .