AAVantgarde Bio Srl

MILAN, Oct. 28, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, is pleased to congratulate Professor Alberto Auricchio, the Company’s Founder and Chief Scientific Officer (CSO), on his appointment as President and Board member of the European Society of Gene and Cell Therapy (ESGCT). The ESGCT Board is composed of researchers and clinicians representing a broad range of European Countries. Members are elected by nomination and vote at the Annual General Meeting. Election to the Board of ESGCT is a great honor and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. As well as being CSO of AAVantgarde, Professor Auricchio is Scientific Director of TIGEM and Professor of Medical Genetics at University “Federico II” in Naples, Italy. He is co-author of over 140 peer-reviewed publications and inventor of several patents related to the use of viral vectors for gene therapy and has received the Outstanding New Investigator Award of the American Society of Gene Therapy and the International Prize for Scientific Research “Arrigo Recordati”. Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde said, “Congratulations to Alberto on this important and well-deserved recognition of his outstanding scientific contributions to the field of gene therapy. We are privileged and honoured to work with him as Founder and Chief Scientific Officer of AAVantgarde.” About AAVantgarde BioAAVantgarde Bio is a clinical stage, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. AAVantgarde is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Dr. Magda Blanco – Head of Corporate Development, AAVantgardeEmail: info@aavantgarde.com

MILAN, Oct. 08, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces three oral presentations at the European Society of Gene & Cell Therapy 31st Annual Meeting (ESGCT), to be held October 22-25 in Rome. AAVantgarde is validating its platform technology in two inherited retinal diseases with clear unmet need, with Usher syndrome type 1B already in the clinic and Stargardt advancing towards clinical development. At this conference, we will be presenting NHP preclinical data from our Stargardt’s program and the process development, analytics and manufacture of the dual vectors to enable the clinical supply for our programs. In addition, our Scientific Founder, Professor Alberto Auricchio, TIGEM Scientific Director, will give a presentation on expediting AAV-based gene therapies. Oral Presentations details: Presentation Date/Time: Tuesday 22 October 2024, 14.00-16.30 h CESTSession title: ExpEditing AAV gene TherapySession Room: Plenary HallFinal abstract number: INV01 Presentation Date/Time: Tuesday 22 October 2024, 17.00-19.30 h CESTSession title: Safety and expression of intein-based Dual AAV8.ABCA4 in the non-human-primate retinaSession Room: Meeting room 2Final abstract number: OR017 Presentation Date/Time: Friday 25 October 2024, 11.00 – 13.00 h CESTSession title: Production of dual AAV vectors for the delivery of large genesSession Room: Plenary HallFinal abstract number: INV73 About AAVantgardeAAVantgarde Bio is a clinical stage, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. AAVantgarde is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Magda Blanco – Head of Corporate Development AAVantgardeEmail: info@avvantgarde.com

▎药明康德内容团队编辑 近期，全球细胞和基因疗法（CGT）领域迎来系列进展。Vertex Pharmaceuticals公司公布了其在研细胞疗法VX-880的积极临床试验数据，75%的1型糖尿病患者在治疗180天及之后不再使用外源性胰岛素，实现糖尿病“功能性治愈”。用于治疗实体肿瘤的嵌合抗原受体-T（CAR-T）细胞疗法领域迎来重要进展，GCC19CART疗法在经过大量先前治疗的转移性结直肠癌（mCRC）患者中实现了57%的客观缓解率（ORR），患者的中位总生存期（OS）达22.8个月。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 图片来源：123RF ———✦研发进展✦——— ◇ Vertex Pharmaceuticals公司公布了其在研细胞疗法VX-880治疗1型糖尿病的最新临床试验结果。截至2024年5月，共有14例患者接受了VX-880治疗，其中12例患者接受了完整剂量的VX-880单次输注，2例患者接受了一半剂量的VX-880。研究结果显示，单次输注VX-880可显著改善患者的血糖水平且不出现严重低血糖事件，患者有望摆脱对外源胰岛素的依赖，实现糖尿病“功能性治愈”。92%（11/12）患者使用外源性胰岛素的频率大幅减少，75%（9/12）患者在治疗180天及之后不再使用外源性胰岛素。4例随访超过1年的患者达到了研究主要终点，即未发生严重低血糖事件、糖化血红蛋白（HbA1c）水平＜7个百分点，以及不再依赖外源性胰岛素治疗。 VX-880是一种在研干细胞来源、完全分化、产生胰岛素的同种异体胰岛细胞疗法。VX-880有可能通过恢复胰岛细胞功能，包括葡萄糖反应性胰岛素产生，恢复机体调节葡萄糖水平的能力。VX-880通过肝门静脉输注给药，需要长期免疫抑制治疗以保护输入的胰岛细胞免受免疫排斥。 图片来源：123RF ◇ Kyverna Therapeutics公司分享了其CAR-T细胞疗法KYV-101正在进行的僵人综合征、重症肌无力和多发性硬化临床试验的病例报告。其中，两名接受KYV-101治疗的僵人综合征患者表现出行动能力和/或行走速度的改善，并且自身抗体滴度降低，其中一名患者在接受治疗一年后仍表现出改善。3名接受KYV-101治疗的重症肌无力患者显示出致病性抗乙酰胆碱受体（anti-AChR）抗体滴度的降低，且病情得到持续的控制，3名患者维持疾病控制超8个月，两名患者维持疾病控制超1年。5名此前接受抗CD20药物治疗失败的多发性硬化患者接受KYV-101治疗后，中枢神经系统中的寡克隆区带数量显著减少，这是多发性硬化疾病进展减缓的潜在替代生物标志物。安全性方面，迄今已有41名自身免疫性疾病患者接受了KYV-101治疗，未观察到严重的细胞因子释放综合征（CRS）或免疫效应细胞相关神经毒性综合征（ICANS）。 KYV-101是一款自体CD19靶向CAR-T细胞疗法。KYV-101中的CAR由美国国立卫生研究院（NIH）设计，旨在提高耐受性。迄今为止，KYV-101在肿瘤和自身免疫性疾病中的临床经验突显了其独特的特性及其为自身免疫性疾病患者提供治疗的潜力。 ◇ BOOST Pharma公司公布了其突破性细胞疗法治疗罕见骨病成骨不全症（OI）的1/2期临床研究的积极顶线结果。该研究中的17名患者确诊为严重的III型和IV型OI，并在卡罗林斯卡大学医院接受了为期12个月的治疗。结果显示，该治疗方案在患儿出生前和出生后给药时均安全且耐受性良好。最后一次给药后的12个月内，患儿的骨折率减少了超过75%。 该疗法基于一种使用人类干细胞的全新细胞疗法，具有很高的成骨能力。这种治疗方法旨在确诊后立即进行干预，无论是在出生前还是出生后，这为生命早期（儿童活动增多的阶段）提供了治疗优势。对于OI患儿来说，预防或减少骨折次数是最重要的治疗目标，但目前尚无获批的治疗方法可以实现这一目标。 图片来源：123RF ◇ Beacon Therapeutics公司公布了其主打项目基因疗法AGTC-501用以治疗X连锁视网膜色素变性（XLRP）患者的1/2期临床试验的中期结果。结果显示，在纳入研究的29名患者中，AGTC-501总体上安全且耐受性良好，未观察到与该疗法相关的临床显著安全事件。数据还显示，治疗眼与未治疗眼之间的视觉功能差异在36个月时仍然存在。AGTC-501的效益-风险比支持其在由RPGR基因突变引起的XLRP患者中的持续临床开发。 ◇ AAVantgarde Bio公司宣布已在LUCE-1试验中为首位受试者进行了给药。LUCE-1是一项1/2期开放标签临床试验，旨在评估以腺相关病毒8（AAV8）为载体的视网膜内基因疗法AAVB-081用于治疗Usher综合征1B型（USH1B）相关的视网膜色素变性患者的安全性和耐受性。AAVB-081使用该公司的专有的双杂交基因治疗平台，向USH1B相关的视网膜色素变性患者递送MYO7A蛋白。AAVantgarde公司的双杂交平台使用两个AAV8载体，每个载体都携带目标基因的一半信息，两个基因片段在细胞核内重新组合为一个完整的基因，从而能够生成治疗水平的蛋白质。 ◇ 斯丹赛生物（Innovative Cellular Therapeutics）宣布其自主研发的CAR-T细胞疗法GCC19CART在转移性结直肠癌患者中的临床研究成果已发表在JAMA Oncology杂志。结果显示，在2X106 CAR-T细胞/kg的剂量下，GCC19CAR治疗经过大量先前治疗的mCRC患者的ORR为57%，中位总生存期（OS）为22.8个月。新闻稿指出，这项研究在CAR-T细胞疗法用于实体肿瘤（特别是mCRC）的治疗方面具有重要的里程碑意义。 斯丹赛还公布了其在美国正在进行的1期临床试验的数据。接受1X106 CAR-T细胞/kg剂量的4例患者中有2例达到部分缓解（PR），ORR为50%。接受2X106 CAR-T细胞/kg剂量的5例患者中有4例达到PR，ORR为80%。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ GC Therapeutics公司宣布正式成立，将致力于推进并解锁下一代细胞疗法。该公司的诱导多能干细胞（iPSC）编程平台TFome（转录因子组）由哈佛医学院和Wyss研究所的教授George Church博士的实验室开发，汇集了合成生物学、基因编辑、细胞工程和机器学习的最新进展，以克服与细胞疗法相关的开发和规模化方面的复杂性，并提高患者在多种疾病领域的治疗可及性。TFome平台旨在通过一步式工艺以比传统方法快100倍的速度提供基于iPSC的现货型细胞疗法，以提高效力、效率、质量并降低成本，从而彻底改变细胞疗法。该公司最初的管线将专注于胃肠道、神经和免疫疾病。 GC Therapeutics已完成6500万美元的A轮融资，由Cormorant Asset Management领投，Mubadala Capital、Andreessen Horowitz （a16z） Bio + Health、Medical Excellence Capital、Cercano Management和Pear VC参投，使该公司的总筹资金额达到7500万美元。 ◇ 贝斯生物宣布完成数千万人民币的A2轮融资，由戈壁大湾区（Gobi Partners GBA）管理的AEF大湾区创业基金领投。本轮融资将用于加强贝斯生物的研发团队、优化产品管线、加速临床试验，并为未来的商业化奠定坚实的基础。 该公司致力于开发创新的细胞疗法和基因编辑产品，用于治疗癌症和遗传疾病。今年7月，贝斯生物首款产品NK510——一款基于碱基编辑技术的自然杀伤细胞（NK）产品已经向中国国家药品监督管理局（NMPA）申报IND并获得受理。除了NK510之外，贝斯生物的产品管线还包括多个针对血液恶性肿瘤和自身免疫性疾病的碱基编辑NK细胞产品，以及通用CAR免疫疗法和体内基因编辑产品。 ◇ LifeSpan Vision Ventures宣布完成对Remedium Bio的投资。Remedium Bio是一家新型基因疗法开发公司。该公司正在开发一种可调剂量基因治疗平台，可以用单次注射、剂量可调的基因治疗取代广泛的多蛋白注射治疗。这种通过皮下注射进行的治疗更具安全、局部、持久优势，且成本低于许多蛋白质疗法。该轮融资将用于推动该公司主要候选产品RMD-1202进入1期研究。RMD-1202是GLP-1受体激动剂，其减肥、血糖控制和胰岛素反应优于每日GLP-1蛋白注射。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] T Reichman, P Witkowski, J Markmann, et al. LBA 31Improvement in glycaemic control and elimination of exogenous insulin use in patients with type 1 diabetes infused with fully differentiated islet cells (VX-880). https://cattendee.abstractsonline.com/meeting/20620/session/660 [2] Kyverna Therapeutics Presents Patient Data Reinforcing Potential of KYV-101 for Treatment of Neuroinflammatory Diseases in Symposium at ECTRIMS 2024. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/kyverna-therapeutics-presents-patient-data-reinforcing-potential-of-kyv-101-for-treatment-of-neuroinflammatory-diseases-in-symposium-at-ectrims-2024-302251529.html [3] Karolinska Developments portfolio company BOOST Pharma announces positive top-line results from a Phase 1/2 study with over 75% reduction of fracture rates in children with rare bone disease. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/17/2947159/0/en/Karolinska-Developments-portfolio-company-BOOST-Pharma-announces-positive-top-line-results-from-a-Phase-1-2-study-with-over-75-reduction-of-fracture-rates-in-children-with-rare-bon.html [4] Beacon Therapeutics Presents 36-Month Interim Results from Phase I/2 HORIZON Trial of AGTC-501 in Patients with XLRP. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-presents-36-month-interim-results-from-phase-i2-horizon-trial-of-agtc-501-in-patients-with-xlrp-302252375.html [5] AAVantgarde announces first patient dosed in First-In-Human Phase 1/2 LUCE-1 study, evaluating AAVB-081 (Dual-AAV) in retinitis pigmentosa related to Usher Syndrome type 1B. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/16/2946331/0/en/AAVantgarde-announces-first-patient-dosed-in-First-In-Human-Phase-1-2-LUCE-1-study-evaluating-AAVB-081-Dual-AAV-in-retinitis-pigmentosa-related-to-Usher-Syndrome-type-1B.html [6] Innovative Cellular Therapeutics Announces the Publication of Groundbreaking GCC19CART™ Study to Treat mCRC in JAMA Oncology. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/19/2949259/0/en/Innovative-Cellular-Therapeutics-Announces-the-Publication-of-Groundbreaking-GCC19CART-Study-to-Treat-mCRC-in-JAMA-Oncology.html [7] GC Therapeutics Launches to Scale and Unlock the Next Generation of Cell Therapy. Retrieved September 20, 2024, from https://www.businesswire.com/news/home/20240918923504/en/GC-Therapeutics-Launches-to-Scale-and-Unlock-the-Next-Generation-of-Cell-Therapy [8] LifeSpan Vision Ventures Invests in Remedium Bio. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/lifespan-vision-ventures-invests-in-remedium-bio-302243756.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新