更新于：2024-06-20
RBI-4000
更新于：2024-06-20
概要
基本信息
原研机构 |
在研机构 |
非在研机构- |
最高研发阶段临床1期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评- |
关联
1
项与 RBI-4000 相关的临床试验A Study to Evaluate the Safety, Reactogenicity and Immunogenicity of RBI-4000 in Healthy Volunteers
The primary purpose of the study is to evaluate the safety, reactogenicity, and immunogenicity of RBI-4000 administered at various dose levels via intramuscular injection and to determine the lowest dose of RBI-4000 necessary to elicit the rabies virus neutralizing antibody titer of equal or greater than (>=) 0.5 international unit per milliliter (IU/mL).
开始日期2023-09-01 |
申办/合作机构 |
100 项与 RBI-4000 相关的临床结果
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100 项与 RBI-4000 相关的转化医学
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100 项与 RBI-4000 相关的专利（医药）
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12
项与 RBI-4000 相关的新闻（医药）2024-05-19
·药明康德
▎药明康德内容团队编辑本期看点1. 罗氏（Roche）公布了其潜在“best-in-class”减重疗法CT-388在1/2期临床试验中的亮眼数据，经过24周每周一次的CT-388皮下注射治疗，100%患者的体重降低幅度超过5%，平均体重降低幅度达18.8%，具有临床与统计学意义。2. Rocket Pharmaceuticals公司公布了3项在研基因疗法的积极数据，其中两款疗法已分别在美国和欧洲递交上市许可申请。3. 在自体CAR-T细胞疗法NXC-201用于治疗复发/难治性轻链（AL）淀粉样变性的1b/2a期临床试验中，患者的总缓解率（ORR）为92%，完全缓解（CR）率接近70%。药明康德内容团队整理CT-388：公布1/2期临床试验数据 罗氏公布了其在研、潜在“best-in-class”葡萄糖依赖性促胰岛素多肽（GIP）和胰高血糖素样肽-1（GLP-1）受体双重激动剂CT-388用于治疗肥胖和2型糖尿病患者的1期临床试验的积极数据。CT-388是一种每周一次皮下注射的GLP-1/GIP受体双重激动剂，旨在对GLP-1和GIP受体均具有强效活性，但最小化β-arrestin募集至任一受体。这种偏向信号可显著减少受体内化和随之而来的脱敏现象，预计可延长药物的药理活性。该疗法有望成为单药或与其他疗法结合使用，以改善体重减轻效果，并可能扩展至其他适应症。罗氏在2023年12月斥资约31亿美元收购Carmot Therapeutics，囊获多款包含CT-388在内处于临床开发阶段的皮下和口服肠促胰岛素疗法。此次公布的结果显示，与安慰剂相比，每周皮下注射一次CT-388并持续24周可使肥胖健康成年人体重显著减轻。CT-388所取得的减重效果具有临床意义，经安慰剂调整后的平均减重效果为18.8%（p<0.001）。在第24周，100%接受CT-388治疗的受试者实现了超过5%的体重减轻，85%实现了超过10%，70%实现了超过15%，45%实现了超过20%的体重减轻。此外，所有基线时处于糖尿病前期状态的受试者在接受CT-388治疗24周后血糖均恢复正常，而接受安慰剂治疗的受试者的血糖状态在此期间基本保持不变。安全性方面，CT-388展现良好的耐受性，最常见的是轻度至中度胃肠道相关不良事件，这与CT-388所属的肠促胰岛素（incretins）类药物中所观察到的一致。RP-L201、RP-L102、RP-L301：公布积极早期临床数据 Rocket Pharmaceuticals在美国基因和细胞治疗学会（ASGCT）年会中公布了数款慢病毒（LV）载体基因疗法的最新数据。分析显示，其用以治疗严重白细胞粘附缺陷-I（LAD-I）的基因疗法Kresladi（marnetegragene autotemcel），与用以治疗范可尼贫血（FA）的RP-L102皆在1/2期关键研究中展现良好的有效性与安全性。其另一款用以治疗丙酮酸激酶缺乏症（PKD）的在研基因疗法RP-L301也在1期试验中展现积极结果。▲接受Kresladi治疗的LAD-I患者发生严重感染而需要住院的发生率大幅下降（图片来源：参考资料[12]）▲接受RP-L102治疗的FA患者的疾病表型获得校正（图片来源：参考资料[12]）▲接受RP-L301治疗的PKD患者的血红蛋白水平获得改善（图片来源：参考资料[12]）目前，美国FDA正在对Kresladi用以治疗LAD-I的生物制品许可申请（BLA）进行审评，PDUFA目标日期为2024年6月30日。同时，欧洲药品管理局（EMA）也已接收RP-L102的上市许可申请（MAA），Rocket预计将在2024年上半年向美国FDA提交相关BLA。NXC-201（HBI0101）：公布1b/2a期临床试验的新数据Immix Biopharma公司公布了其子公司Nexcella公司开发的靶向BCMA的自体CAR-T细胞疗法NXC-201的1b/2a期临床试验的新数据。该研究旨在评估NXC-201用于治疗复发/难治性AL淀粉样变性的安全性和有效性。此前，NXC-201已被FDA授予孤儿药资格，用于治疗AL淀粉样变性和多发性骨髓瘤（MM）。此次公布的数据显示，复发/难治性AL淀粉样变性患者的ORR为92%（12/13），12例既往未接受过BCMA靶向双特异性治疗的患者均对NXC-201有应答，近69.2%（9/13）的患者获得了CR。1例既往接受过BCMA靶向双特异性治疗的患者对NXC-201没有应答。截至2024年5月10日，最佳缓解者的缓解持续时间已达28.0个月，并仍在持续获得缓解。安全性方面，未发生免疫效应细胞相关神经毒性综合征（ICANS）事件，细胞因子释放综合征（CRS）的中位持续时间为2天（范围：1-5）。ION582：布1/2a期临床试验数据Ionis Pharmaceuticals公司宣布，其反义寡核苷酸（ASO）疗法ION582用于治疗天使综合征（Angelman syndrome）的1/2a期开放标签研究HALOS获得了积极的顶线数据。天使综合征由来自母体的UBE3A等位基因功能丧失引起，表现为严重的运动、语言和认知功能发育迟缓、癫痫发作和共济失调，导致患者完全依赖护理人员。ION582是一款通过脊椎穿刺鞘内给药的ASO疗法，旨在激活来自父体的UBE3A等位基因，从而增加大脑中UBE3A蛋白的产生。此次公布的结果究表明，ION582在所有剂量水平下均显示良好的安全性和耐受性，并在认知、沟通和运动技能等关键功能领域显示出令人鼓舞和一致的益处。接受治疗6个月后，大约65%的患者在Bayley-4认知评估（一种由医生进行的临床功能客观评估）中显示出改善，超过了同一时期疾病自然史研究中观察到的改善。大约70%的患者在Bayley-4的接受性和/或表达性沟通评估中显示出改善。大约65%的患者在Bayley-4的运动技能评估中显示出改善。RBI-4000：公布1期临床试验数据Replicate Bioscience公司公布了其在研RNA疫苗RBI-4000的1期临床试验的积极结果。RBI-4000是一款基于自我复制RNA（srRNA）的狂犬病疫苗，旨在刺激针对狂犬病病毒的中和免疫反应，用于预防狂犬病感染。在临床前研究中，肌肉注射RBI-4000可提供针对狂犬病病毒的持久保护，诱导抗体生成和产生强大的病毒特异性T细胞。这次公布的结果显示，接受一剂疫苗后，92%的受试者达到了世界卫生组织（WHO）定义的替代保护指标，接受两剂疫苗后，100%的受试者都达到了WHO定义的替代保护指标。安全性方面，RBI-4000在所有队列中都具有良好的耐受性，没有严重的不良事件。此外，未观察到剂量限制性毒性。新闻稿指出，Replicate公司的新型srRNA载体可扩大RNA技术在疫苗以外的生物治疗蛋白模板化表达方面的应用，如用于治疗免疫疾病、代谢疾病和癌症等。IMA203：公布1期临床试验数据 Immatics公司在2024年第一季度的财务报告上公布了其在研T细胞受体T细胞（TCR-T）疗法IMA203用以治疗经治转移性黑色素瘤患者的最新数据。IMA203是由Immatics专有ACTengine平台所开发的TCR-T细胞，靶向由HLA-A*02呈递的黑素瘤抗原PRAME。PRAME是一种在各种实体瘤中经常表达的蛋白质，因此IMA203具潜力治疗广泛的癌症患者群体。此次公布的分析结果显示，87%的患者实现肿瘤缩小，患者的确认ORR达55%。Immatics计划在2024年下半年的医学会议上，提供IMA203的下一次临床数据更新。同时，该公司亦计划在与美国FDA进一步讨论后，于今年启动IMA203作为二线及以上疗法，治疗黑色素瘤患者的注册性2/3期临床试验。 ▲IMA203的疗效结果（图片来源：参考资料[5]）ProtheraCytes：公布1/2b期临床试验数据 CellProthera日前宣布，其主打在研产品ProtheraCytes在一项针对严重心肌梗死（AMI）患者的1/2b期研究中获得积极结果。ProtheraCytes来源于从患者外周血中采集的CD34阳性干细胞，这些细胞能促进血管再生，调节炎症、细胞凋亡和心肌细胞再生，并有望诱导受损心肌组织的修复过程。此次公布的数据显示，接受治疗6个月后，包括梗死面积、左心室尺寸和NT-proBNP水平在内的预后生物标志物的积极变化支持ProtheraCytes在心肌梗死后有心力衰竭风险患者中的疗效。研究人员表示，这一结果支持在更大规模的3期临床试验中评估ProtheraCytes的效果。Vepdegestrant（ARV-471）：公布1b期临床试验的新数据 Arvinas公司和辉瑞（Pfizer）公布了在研雌激素受体（ER）PROTAC蛋白降解剂vepdegestrant（ARV-471）联合CDK4/6抑制剂Ibrance（palbociclib）的1b期临床试验的积极数据。作为一款口服、靶向降解ER的蛋白降解剂，vepdegestrant可在肿瘤细胞内降解几乎所有的ER分子，并在许多ER驱动的动物肿瘤模型中，引起肿瘤大量的缩小。该试验入组患者为局部晚期或转移性雌激素受体阳性/人表皮生长因子2阴性（ER+/HER2-）乳腺癌患者，既往接受过中位4线治疗。这次所公布的数据是去年12月所公布数据的额外6个月随访数据更新。分析显示，接受联合疗法乳腺癌患者的ORR为41.9%，中位无进展生存期（PFS）为11.2个月，显示vepdegestrant联合palbociclib治疗乳腺癌的潜力。INI-2004：公布1b期临床试验数据 Inimmune公司公布了INI-2004用于治疗过敏性鼻炎的1b期多剂量递增研究的最新结果。INI-2004是一种不针对特定过敏原的免疫疗法，能结合并激活TLR4。经鼻内给药后，INI-2004可显著降低临床前动物模型的过敏性鼻炎指标，包括气道阻力、嗜酸性粒细胞流入以及与过敏性鼻炎发病机制有关的细胞因子的产生。该研究纳入了35例对豚草过敏的受试者。此次公布的结果显示，与接受安慰剂相比，接受INI-2004治疗的受试者在接触到豚草后，鼻塞情况显示出剂量依赖性的改善。与接受安慰剂相比，接受INI-2004治疗的受试者的鼻拭子样本中细胞因子生物标志物具有呈剂量依赖性的升高趋势。此外，在多次给药过程中，所有剂量的INI-2004均具有良好的耐受性，没有达到最大耐受量。SRP-001：公布1期临床试验数据 South Rampart Pharma公司公布了其潜在“best-in-class”的非阿片类镇痛药SRP-001的1期随机对照试验的积极结果。SRP-001能够触发中脑PAG区域形成AM404，AM404是辣椒素受体（TRPV1）的一种强效激活剂，而TRPV1在大脑对疼痛的反应中起着至关重要的作用。SRP-001提供了一种不会像阿片类药物那样被滥用的潜在有效的镇痛解决方案，该疗法也不会出现肝脏毒性和肾脏毒性问题。此前，SRP-001获得了美国FDA授予的快速通道资格，用于治疗急性疼痛。此次公布的结果显示，SRP-001的安全性和耐受性良好，具有良好的药代动力学，使其可用于治疗神经性疼痛、急性/慢性疼痛和急性偏头痛患者。该疗法用于治疗急性疼痛的2期试验计划于2024年下半年启动。▲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] Immix Biopharma Announces Positive NXC-201 Relapsed/Refractory AL Amyloidosis Clinical Data in ASGCT 2024 Late Breaking Oral Presentation. Retrieved May 17, 2024, from https://www.globenewswire.com/news-release/2024/05/10/2879674/0/en/Immix-Biopharma-Announces-Positive-NXC-201-Relapsed-Refractory-AL-Amyloidosis-Clinical-Data-in-ASGCT-2024-Late-Breaking-Oral-Presentation.html[2] Verismo Therapeutics Receives IND Clearance from the FDA for SynKIR™-310 in Relapsed/Refractory B-cell NHL. Retrieved May 17, 2024, from https://www.prnewswire.com/news-releases/verismo-therapeutics-receives-ind-clearance-from-the-fda-for-synkir-310-in-relapsedrefractory-b-cell-nhl-302144410.html[3] Senti Bio Announces First Patient Dosed in Phase 1 Clinical Trial of SENTI-202 for the Treatment of Relapsed or Refractory Hematologic Malignancies Including Acute Myeloid Leukemia. Retrieved May 17, 2024, from https://investors.sentibio.com/news-releases/news-release-details/senti-bio-announces-first-patient-dosed-phase-1-clinical-trial[4] Immatics Announces First Quarter 2024 Financial Results and Business Update. Retrieved May 14, 2024 from https://investors.immatics.com/news-releases/news-release-details/immatics-announces-first-quarter-2024-financial-results-and-0[5] Immatics Corporate Presentation. Retrieved May 14, 2024 from https://investors.immatics.com/static-files/4eb058de-2c8c-4384-8b6e-2569e1379dab[6] Immune-Onc Therapeutics Announces Presentation of IO-202 Phase 1b Interim Data of Patients with Chronic Myelomonocytic Leukemia (CMML) at 2024 European Hematology Association (EHA) Annual Congress. Retrieved May 17, 2024 from https://www.globenewswire.com/news-release/2024/05/10/2879674/0/en/Immix-Biopharma-Announces-Positive-NXC-201-Relapsed-Refractory-AL-Amyloidosis-Clinical-Data-in-ASGCT-2024-Late-Breaking-Oral-Presentation.html[7] Ajax Therapeutics Announces FDA Clearance of IND Application for AJ1-11095, a First-in-Class Type II JAK2 inhibitor, for the Treatment of Myelofibrosis. Retrieved May 17, 2024 from https://www.businesswire.com/news/home/20240513313466/en[8] Voyager Therapeutics Announces First Participants Dosed in Single Ascending Dose Trial of VY-TAU01 for the Treatment of Alzheimer’s Disease. Retrieved May 17, 2024 from https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-announces-first-participants-dosed-single[9] A2 Bio Announces First Patient Dosed in EVEREST-2 Phase 1 Clinical Trial of Novel Mesothelin Logic-Gated CAR T. Retrieved May 17, 2024 from https://www.businesswire.com/news/home/20240516145046/en/[10] Carisma Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of CT-0525, a Novel HER2-Targeting CAR-Monocyte. Retrieved May 17, 2024 from https://www.prnewswire.com/news-releases/carisma-therapeutics-announces-first-patient-dosed-in-phase-1-clinical-trial-of-ct-0525-a-novel-her2-targeting-car-monocyte-302146805.html[11] Flamingo Therapeutics Announces First Patient Dosed in Investigator-Initiated Trial (IIT) Evaluating Danvatirsen in Phase 1 for AML/MDS Cancers. Retrieved May 17, 2024 from https://www.prnewswire.com/news-releases/flamingo-therapeutics-announces-first-patient-dosed-in-investigator-initiated-trial-iit-evaluating-danvatirsen-in-phase-1-for-amlmds-cancers-302147639.html[12] Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Retrieved May 10, 2024 from https://www.businesswire.com/news/home/20240510805823/en[13] CellProthera announces positive Phase I/IIb data for ProtheraCytes® cell therapy in acute myocardial infarction. Retrieved May 15, 2024, from https://www.cellprothera.com/en/cellprothera-announces-positive-phase-i-iib-data-for-protheracytes-cell-therapy-in-acute-myocardial-infarction/[14] Genprex Doses First Patient in Acclaim-3 Clinical Study of Reqorsa® Immunogene Therapy in Combination with Tecentriq® to Treat Small Cell Lung Cancer. Retrieved May 15, 2024, from https://www.prnewswire.com/news-releases/genprex-doses-first-patient-in-acclaim-3-clinical-study-of-reqorsa-immunogene-therapy-in-combination-with-tecentriq-to-treat-small-cell-lung-cancer-302144415.html[15] Delix Announces DLX-001 Demonstrates Evidence of CNS Penetration and Brain Activity Without Psychotomimetic, Dissociative, or Hallucinogenic Effects in Ongoing Phase 1 Trial. Retrieved May 15, 2024, from https://www.businesswire.com/news/home/20240513298581/en[16] Ryvu Therapeutics to present clinical and preclinical data on RVU120 at the 2024 European Hematology Association Congress. Retrieved May 15, 2024, from https://www.prnewswire.com/news-releases/ryvu-therapeutics-to-present-clinical-and-preclinical-data-on-rvu120-at-the-2024-european-hematology-association-congress-302145195.html[17] CASI Pharmaceuticals Receives FDA Clearance On The Investigational New Drug (IND) Application For CID-103 In Immune Thrombocytopenia (ITP). Retrieved May 15, 2024, from https://www.prnewswire.com/news-releases/casi-pharmaceuticals-receives-fda-clearance-on-the-investigational-new-drug-ind-application-for-cid-103-in-immune-thrombocytopenia-itp-302145848.html[18] Replicate Bioscience Presents Positive Data from Phase 1 Trial and Preclinical Data with Novel Self-Replicating RNA at the 2024 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. Retrieved May 17, 2024, from https://www.prnewswire.com/news-releases/replicate-bioscience-presents-positive-data-from-phase-1-trial-and-preclinical-data-with-novel-self-replicating-rna-at-the-2024-american-society-of-gene--cell-therapy-asgct-annual-meeting-302142558.html[19] Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome. Retrieved May 16, 2024, from https://ir.ionispharma.com/news-releases/news-release-details/ionis-announces-positive-topline-results-phase-12a-trial-ion582[20] Arvinas and Pfizer Announce Updated Clinical Data from Phase 1b Trial of Vepdegestrant in Combination with Palbociclib (IBRANCE®). Retrieved May 16, 2024 from https://ir.arvinas.com/news-releases/news-release-details/arvinas-and-pfizer-announce-updated-clinical-data-phase-1b-trial[21] [Ad hoc announcement pursuant to Art. 53 LR] Roche reports positive Phase I results for its dual GLP-1/GIP receptor agonist CT-388 in people with obesity. Retrieved May 16, 2024 from https://www.globenewswire.com/news-release/2024/05/16/2883079/0/en/Ad-hoc-announcement-pursuant-to-Art-53-LR-Roche-reports-positive-Phase-I-results-for-its-dual-GLP-1-GIP-receptor-agonist-CT-388-in-people-with-obesity.html[22] INIMMUNE ANNOUNCES SUCCESSFUL PHASE 1 CLINICAL RESULTS FOR DISEASE MODIFYING ALLERGY TREATMENT, INI-2004. Retrieved May 16, 2024 from https://www.prnewswire.com/news-releases/inimmune-announces-successful-phase-1-clinical-results-for-disease-modifying-allergy-treatment-ini-2004-302145985.html[23] South Rampart Pharma Publishes SRP-001's Unique Pain Relief Mechanism and Phase 1 Trial Results in Scientific Reports. Retrieved May 16, 2024 from https://www.globenewswire.com/news-release/2024/05/16/2883210/0/en/South-Rampart-Pharma-Publishes-SRP-001-s-Unique-Pain-Relief-Mechanism-and-Phase-1-Trial-Results-in-Scientific-Reports.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
细胞疗法基因疗法临床结果免疫疗法临床2期
2024-05-11
Positive data from Phase 1 trial of RBI-4000 vaccine achieve WHO-established surrogate of protection across all dose cohorts
Vector improvements demonstrate broad potential of Replicate's srRNA for expanded indications in complex infectious disease and in situ expression of therapeutic proteins
SAN DIEGO, May 11, 2024 /PRNewswire/ -- Replicate Bioscience, a clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for use across a range of infectious disease, oncology, autoimmune disease indications and beyond, presented new preclinical data and today will share interim clinical trial results from an ongoing Phase 1 study at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11 in Baltimore, Maryland.
"At ASGCT, Replicate is presenting two important breakthroughs in srRNA technology," said Nathaniel Wang, Ph.D., CEO of Replicate. "For vaccines, our clinical data demonstrate the induction of protective levels of immunity at doses significantly lower than any other reported mRNA or srRNA vaccine. This potency, combined with a best-in-class safety profile, demonstrates the capability of our technology to greatly expand the utility of RNA technology for vaccines. Beyond vaccines, further improvements to our library of vectors now enable us to control and prolong in vivo production of therapeutic proteins compared to circular RNA, linear mRNA, and current state-of-the art srRNA, opening up therapeutic areas such as immunology and metabolic disease."
RBI-4000's Phase 1 results will be presented on Saturday, May 11 at 10:15 a.m. ET by Replicate's Chief Medical Officer, Zelanna Goldberg, M.D., in an oral presentation titled "Single and Low Dose Self-Replicating RNA Vaccine Provides Effective Immune Protection Against Rabies in Healthy Volunteers." The results highlight the strong immunogenicity and favorable safety profile of RBI-4000 in the Phase 1 trial, which is the first clinical validation of Replicate's next-generation srRNA technology. Additional takeaways are as follows:
Day 85 datasets for all cohorts met the WHO-established surrogate of protection at doses significantly lower than any other reported RNA-based vaccines;
Substantive majority of participants achieved metric in all dose cohorts.
In the previously unreported 10 mcg dose cohorts:
100% of participants achieved surrogate of protection after two vaccine doses.
92% of participants achieved surrogate of protection after a single vaccine dose.
Safety data from the interim dataset demonstrate RBI-4000 was well tolerated with no severe adverse events across all cohorts; reactogenicity was transient and self-limiting.
No dose limiting toxicity (DLT) was observed; maximum tolerated dose (MTD) has not been reached, enabling further dose escalation.
On May 9, Parinaz (Paris) Aliahmad, Ph.D., Head of Research and Development at Replicate, delivered an oral presentation at ASGCT titled "Novel Self-Replicating RNA Vectors Broaden Therapeutic Window and Expand Use Outside of Vaccines." The results underscore the broad potential of srRNA as a new treatment modality across a wide range of disease areas. Additional takeaways are as follows:
Vaccines using Replicate's optimized vectors achieve protective immunity at ultra-low doses (1 picogram) with minimal reactogenicity.
Beyond vaccines, Replicate's novel srRNA vectors demonstrate >100-fold increased expression of encoded proteins and improved durability compared to circular RNA, linear mRNA vectors, and current state-of-the-art srRNA technologies.
Replicate's novel srRNA vectors can expand utility of RNA technology for templated expression of biotherapeutic proteins for applications outside of vaccines, in areas such as immune disorders, metabolic disease, and cancers.
About Replicate Bioscience
Replicate Bioscience, an Apple Tree Partners portfolio company, is a clinical-stage company amplifying the power of RNA therapeutics by pioneering its novel self-replicating RNA (srRNA) technology to overcome the shortcomings of existing mRNA approaches, with potential improvements in bioactivity at lower doses, induction of more robust and durable immune responses, and improved tolerability. Replicate's off-the-shelf srRNAs contain two components: virally derived genetic code to drive controlled and self-limiting amplification, and the RNA encoding therapeutic proteins. The company's library of viral vectors, selected for driving robust and sustained protein expression and orders-of-magnitude improved performance over linear mRNA, allow for the development of treatments in applications including oncology, infectious disease, and autoimmunity. Differentiated by a team of srRNA experts, a customizable library of synthetic srRNA vectors, and end-to-end development capabilities, Replicate is uniquely positioned to finally expand the reach of RNA treatments toward widespread use in infectious disease, immuno-oncology, autoimmune disease, and more. Visit us at .
SOURCE Replicate Bioscience
临床结果疫苗临床1期ASCO会议AACR会议
2024-04-22
SAN DIEGO, April 22, 2024 /PRNewswire/ -- Replicate Bioscience, a clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for use in infectious disease, oncology, autoimmune disease, and more, today announced acceptance of two oral presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11 in Baltimore, Maryland.
Presentation Details:
The full abstracts are now available through the ASGCT conference website.
Title: Novel Self-Replicating RNA Vectors Broaden Therapeutic Window and Expand Use Outside of Vaccines
Session Title: Emerging Viral Vectors
Location and Time: Ballroom 4, Thursday, May 9, 5:15 PM - 5:30 PM ET
Abstract: 204
Presenter: Parinaz (Paris) Aliahmad
Summary: Ultra-low doses of optimized srRNA vectors achieved immunization with protective antibody titers with minimal reactogenicity in a preclinical influenza model. This broadened therapeutic window presents the opportunity to use these vectors for many clinical applications.
Title: Single and Low Dose Self-Replicating RNA Vaccine Provides Effective Immune Protection Against Rabies in Healthy Volunteers
Session Title: Vector Product Engineering, Development, and Manufacturing (excluding AAV)
Location and Time: Room 314-317, Saturday, May 11, 10:15 AM - 10:30 AM ET
Abstract: 408
Presenter: Zelanna Goldberg
Summary: RBI-4000, an srRNA vector encoding the rabies glycoprotein encapsulated in a lipid nanoparticle, is the first clinical demonstration of next-generation srRNA technology. Interim analysis of a Phase 1 trial demonstrate that all doses were well tolerated and that RBI-4000 was immunogenic at all dose levels tested, conferring protection with a single, low dose.
About Replicate Bioscience
Replicate Bioscience, an Apple Tree Partners portfolio company, is a clinical-stage company amplifying the power of RNA therapeutics by pioneering its novel self-replicating RNA (srRNA) technology to overcome the shortcomings of existing mRNA approaches, with potential improvements in bioactivity at lower doses, induction of more robust and durable immune responses, and improved tolerability. Replicate's off-the-shelf srRNAs contain two components: virally derived genetic code to drive controlled and self-limiting amplification, and the RNA encoding therapeutic proteins. The company's library of viral vectors, selected for driving robust and sustained protein expression and orders-of-magnitude improved performance over linear mRNA, allow for the development of treatments in applications including oncology, infectious disease, and autoimmunity. Differentiated by a team of srRNA experts, a customizable library of synthetic srRNA vectors, and end-to-end development capabilities, Replicate is uniquely positioned to finally expand the reach of RNA treatments toward widespread use in infectious disease, immuno-oncology, autoimmune disease, and more. Visit us at .
SOURCE Replicate Bioscience
疫苗AACR会议临床1期免疫疗法
100 项与 RBI-4000 相关的药物交易
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 狂犬病 | 临床1期 | 美国 | 2023-09-13 |
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| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床1期 | 30 | 淵觸襯窪範壓淵構積齋(範繭夢簾鏇顧遞夢製醖) = 膚衊糧鹹淵齋遞夢餘簾 襯廠憲鬱膚壓憲壓觸餘 (淵遞鑰蓋鹹築鹹製觸窪 ) | 积极 | 2024-02-14 | |||
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临床1期 | - | 84 | 遞鹹積淵選齋廠網夢鑰(艱繭醖衊淵廠憲構範蓋) = none 膚醖築觸膚壓壓艱鏇衊 (襯鏇鑰蓋鹹繭襯遞淵構 ) | 积极 | 2024-02-14 |
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