A Single-Arm, Open-Label, Phase I Study to Determine the Safety, Tolerability and Preliminary Efficacy of Obecabtagene Autoleucel in Patients With Severe, Refractory Systemic Lupus Erythematosus

This is a Phase I study of obecabtagene autoleucel (obe-cel), autologous T cells engineered with a chimeric antigen receptor (CAR) targeting CD19, to establish the tolerability, safety, preliminary efficacy, and pharmacokinetics of obe-cel in patients with severe, refractory SLE.

开始日期2024-02-02

申办/合作机构

Autolus Ltd.

NCT06173518

/ Recruiting临床1期

A Single-Arm, Open-Label, Multi-Centre, Phase Ib Study Evaluating the Safety and Preliminary Efficacy of AUTO1 in Pediatric Patients With CD19-Positive Relapsed/ Refractory (r/r) B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL)

This is a Phase Ib study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL)

开始日期2023-11-16

申办/合作机构

Autolus Ltd.

NCT04404660

/ Active, not recruiting临床1/2期

An Open-Label, Multi-Centre, Phase Ib/II Study Evaluating the Safety and Efficacy of AUTO1, a CAR T Cell Treatment Targeting CD19, in Adult Patients with Relapsed or Refractory B Cell Acute Lymphoblastic Leukaemia

This is a Phase Ib/II study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting CD19 in adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).

开始日期2020-06-03

申办/合作机构

Autolus Ltd.

100 项与 Autolus Therapeutics Plc 相关的临床结果

登录后查看更多信息

0 项与 Autolus Therapeutics Plc 相关的专利（医药）

登录后查看更多信息

项与 Autolus Therapeutics Plc 相关的文献（医药）

2025-04-16·Science Translational Medicine

Preclinical development of anti-CD21 chimeric antigen receptor T cells to treat T cell acute lymphoblastic leukemia

Article

作者: Nannini, Francesco ; Khwaja, Asim ; Wade, Brandon ; Hoekx, Malika ; Pule, Martin ; Gritti, Giuseppe ; Onuoha, Shimobi ; Maciocia, Paul ; Karpanasamy, Thaneswari ; Marafioti, Teresa ; Ferrari, Mathieu ; Mansour, Marc ; Maciocia, Nicola ; Wawrzyniecka, Patrycja A. ; Schuldt, Maria ; Lee, Lydia ; Ng, Stephanie ; Ramanayake, Saumya ; O’Connor, David ; Burley, Amy ; Acuna, Ciaran

Patients with relapsed/refractory (r/r) T cell acute lymphoblastic leukemia (T-ALL) have a dismal prognosis, highlighting the urgent need for effective therapies. Chimeric antigen receptor (CAR)–T cell approaches targeting pan–T cell antigens may be limited by T cell aplasia and fratricide, necessitating “rescue” allogeneic hematopoietic stem cell transplantation. In this study, we identify CD21, a pan–B cell marker, as a promising target for T-ALL immunotherapy. CD21 is expressed in 50% of T-ALL cases at diagnosis but in fewer than 10% of mature T cells. We observed that CAR-T cells targeting membrane-distal CD21 epitopes were ineffective, likely because of the bulky, glycosylated nature of the antigen. However, when we engineered CAR-T cells to target membrane-proximal CD21 epitopes using an antigen-binding fragment (Fab)–CAR design, we demonstrated robust activity against T-ALL cell lines, primary tumors, and patient-derived xenografts in both in vitro and in vivo models. The enhanced efficacy of this Fab-CAR design was driven by its high stability and reduced surface expression, addressing limitations of traditional CAR constructs. In addition, pharmacological inhibition of the phosphatidylinositol 3-kinase axis up-regulated CD21 expression in T-ALL, further enhancing the potency of anti-CD21 CAR-T cells in vitro and in a patient-derived xenograft in vivo model. This study establishes CD21 as a viable CAR-T target and highlights advances in CAR design for bulky antigens, as well as the potential for pharmacological strategies to augment target expression. Anti-CD21 CAR-T cells represent a promising therapeutic option for improving outcomes for patients with T-ALL.

2024-12-01·Transplantation and Cellular Therapy

Risk-Based Qualification of Cellular Starting Material Collection Sites: Tools to Determine Scope of Manufacturer Audits and Reduce Duplicative Efforts

Review

作者: Wacker, Kara ; Anderson, Christina ; Hlucky, Tracey ; Contractor, Karishma ; Walters, Victoria ; Barnes, Linda ; Myers, Joan ; Holman, Peter ; Shingler, William

Cellular and Gene Therapy product (CGT product) manufacturers are required by regulations to qualify sites performing cell collection and processing as part of their manufacturing processes. The use of audits to qualify a site is part of the traditional supplier quality models for drug products. Due to the rapid growth of the CGT industry in recent years, healthcare institutions and manufacturers are finding it difficult to manage the increasing workload and resources needed to support audits when they are applied to the provision of Cellular Starting Material (CSM) collection from patients/subjects for the purpose of manufacturing. To alleviate this audit burden, several manufacturers have applied risk-based approaches to determine the needs and scope of audits. The authors of this commentary recommend that all manufacturers utilize a risk-based assessment program when appropriate and explain the use of tools created to facilitate a risk-based approach to streamline and reduce duplicative audits. This approach and tools, created by The NextGen Industry Working Group (IWG) site certification workstream with representation from pharmaceutical and biotech companies, health care institutions, accrediting organizations, and other stakeholders, is aligned with proposals from other multistakeholder groups, including the ASTCT 80/20 Task Force. The tools aim to streamline the site qualification processes performed by each manufacturer, offering a standardized approach to audits or gap analysis assessments. Offering an abbreviated audit model for sites that have already attained accreditation through agencies such as the AABB (Association for the Advancement of Blood & Biotherapies) and FACT (Foundation for the Accreditation of Cellular Therapy), helping the manufacturer to focus on product-specific requirements rather than re-evaluating systems that have already been audited in great detail by other parties.

2024-01-11·Blood

CD19/CD22 targeting with cotransduced CAR T cells to prevent antigen-negative relapse after CAR T-cell therapy for B-cell ALL

Article

作者: Bartram, Jack ; Mullanfiroze, Khushnuma ; Ko, Ka-Yuk ; Bonney, Denise ; Gilmour, Kimberly ; Gravett, Emma ; Hough, Rachael ; Chu, Jan ; Guvenel, Aleks ; Lopes, Andre ; Kunaseelan, Sangeetha ; Williams, Lindsey ; Adams, Stuart ; Lazareva, Arina ; Vora, Ajay ; Ngai, Yenting ; Lucchini, Giovanna ; Popova, Bilyana ; Wynn, Robert ; Hackshaw, Allan ; Watts, Kelly ; Thomas, Rebecca ; Lal, Alka ; Ghorashian, Sara ; Kokalaki, Evangelia ; Walding, Chloe ; Rao, Kanchan ; Pinner, Danielle ; Connor, Christopher ; Pule, Martin ; Chiesa, Robert ; Rao, Anupama ; Terris, Craig ; Richardson, Rachel ; Inglott, Sarah ; Oporto-Espuelas, Macarena ; Nguyen, Kyvi ; Yeung, Jenny ; Pavasovic, Vesna ; Carulla, Milena Balasch ; Amrolia, Persis J. ; Silva, Juliana

AbstractCD19-negative relapse is a leading cause of treatment failure after chimeric antigen receptor (CAR) T-cell therapy for acute lymphoblastic leukemia. We investigated a CAR T-cell product targeting CD19 and CD22 generated by lentiviral cotransduction with vectors encoding our previously described fast-off rate CD19 CAR (AUTO1) combined with a novel CD22 CAR capable of effective signaling at low antigen density. Twelve patients with advanced B-cell acute lymphoblastic leukemia were treated (CARPALL [Immunotherapy with CD19/22 CAR Redirected T Cells for High Risk/Relapsed Paediatric CD19+ and/or CD22+ Acute Lymphoblastic Leukaemia] study, NCT02443831), a third of whom had failed prior licensed CAR therapy. Toxicity was similar to that of AUTO1 alone, with no cases of severe cytokine release syndrome. Of 12 patients, 10 (83%) achieved a measurable residual disease (MRD)–negative complete remission at 2 months after infusion. Of 10 responding patients, 5 had emergence of MRD (n = 2) or relapse (n = 3) with CD19- and CD22-expressing disease associated with loss of CAR T-cell persistence. With a median follow-up of 8.7 months, there were no cases of relapse due to antigen-negative escape. Overall survival was 75% (95% confidence interval [CI], 41%-91%) at 6 and 12 months. The 6- and 12-month event-free survival rates were 75% (95% CI, 41%-91%) and 60% (95% CI, 23%-84%), respectively. These data suggest dual targeting with cotransduction may prevent antigen-negative relapse after CAR T-cell therapy.

206

项与 Autolus Therapeutics Plc 相关的新闻（医药）

2025-04-29

·蒲公英Ouryao

一周 | 国际法规药事(04.21-04.27)

◆ ◆ ◆ ◆Paul's Insight2025年4月21日-4月27日◆ ◆ ◆ ◆过去一周（4月21日至4月27日），全球药事监管和行业机构齐齐发声，犹如一场精彩的药品政策与科技盛宴。从FDA的合成色素退市行动，到EMA的罕见病新疗法推荐；从EDQM对生物制品检测标准的升级，到MHRA多款创新疗法的批准；再到行业协会PDA和ISPE在指南与实践层面的多重发力，这一连串新闻不仅彰显了各大机构的监管力度，更映射出制药行业在创新、合规与可持续性间的不断平衡。美国FDA从食药安全到创新药物的“双轨”发力1. 合成食用色素“退场”行动4月22日，美国FDA宣布，计划逐步淘汰美国食品供应中的合成色素（Petroleum-Based Synthetic Dyes），为儿童健康撑起防护伞。FDA表示，这一举措，是“让美国再次健康”（Make America Healthy Again）计划的重要里程碑。此前，1月15日赤藓红（FD&C Red No. 3）禁用已拉开了帷幕，后续这一更广泛的色素退市，将持续推动产业寻求天然、低风险的替代方案。图：赤藓红禁用后，FDA再推新规，八大合成食用色素将退场https://www.fda.gov/news-events/press-announcements/hhs-fda-phase-out-petroleum-based-synthetic-dyes-nations-food-supply2. 药品安全敲响警钟：外用非那雄胺安全红线4月22日，FDA对市场上未经批准的外用非那雄胺配药制剂发出警示，提醒医疗机构、药房及消费者：目前无任何局部非那雄胺产品拥有FDA批准的标签和安全、有效性验证，配制药剂可能隐藏严重风险，包括与米诺地尔等混合制剂的混用场景。FDA直指部分处方药店和远程医疗平台的药品安全风险行为，呼吁行业加强自律与审查。图：就非那雄胺外用安全问题，FDA发布脱发常用药警示https://www.fda.gov/drugs/human-drug-compounding/fda-alerts-health-care-providers-compounders-and-consumers-potential-risks-associated-compounded3. 中国创新药的海外“登台”——派安普利单抗获批4月24日，FDA官网放出重磅消息：康方生物自主研发的PD1单抗派安普利单抗（Penpulimabkcqx）获批，用于复发或转移性非角化型鼻咽癌（NPC）治疗。该产品采用IgG1亚型Fc段改造设计，依托突破性疗法认定、孤儿药资格和快速通道三重加速，联袂正大天晴药业集团布局后续商业化。此举不仅为全球鼻咽癌患者带来曙光，也标志着中国创新药在国际舞台上稳步迈进。（点击此处查看相关阅读）图：中国制药行业又一里程碑，派安普利单抗获FDA批准进入美国市场https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma美国PDA发布新闻季刊4月24日，美国注射剂协会（PDA）发布新闻季刊，总结其在过去一个季度的工作，要点如下。图：PDA发布新闻季刊https://www.pda.org/pda-letter-portal/home/full-article/community-news-quarterly-april-20251）2024年PDA出版奖揭晓PDA颁发奖项给Novartis&Ogilvy团队，表彰其用于无菌性检测的创新方法，该论文名为《一种利用固相流式细胞术进行细胞制备、培养基和缓冲液的快速无菌方法》。年度PDA Letter文章奖项授予武田制药的《理解日本品质》。2）区域分会动态：德国、奥地利和瑞士分会（DACH）亮点纷呈DACH分会“目视检测与颗粒挑战”网络研讨会吸引400余名专家，聚焦AI时代的风险基础方法与质量设计。PDA表示，此次网络研讨会超出预期，如此高的参与度凸显了业界对目视检测和颗粒物相关挑战的关注，同时也强化了DACH分会促进制药行业知识交流和最佳实践的使命。欧盟EMA欧盟罕见病与适应症扩展双管齐下1. DMD新疗法——组蛋白去乙酰化酶（HDAC）抑制剂获“有条件上市许可”4月25日，欧洲药品管理局（EMA）发布杜氏肌营养不良症（DMD）创新药物Duvyzat（givinostat）“有条件上市许可”建议。该组蛋白去乙酰化酶（HDAC）抑制剂在120例6岁以上患者的18个月研究中显著延缓台阶攀爬时间，虽次要终点未全部显著，但整体数据朝积极方向发展。获得“有条件上市许可”，这是欧盟的监管机制之一，旨在促进早期上市药物，以满足未满足医疗需求的。EMA要求申请人后续进行一项随机、安慰剂对照研究，并进行长期随访将补足盲点。上市许可人为Italfarmaco S.p.A.，是是一家总部位于意大利米兰的制药跨国公司。图：杜氏肌营养不良症（DMD）创新药物获“有条件上市许可”https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy2. 甲状腺眼病首创疗法Tepezza获积极审评意见4月25日，EMA对甲状腺眼病（TED）首款靶向IGF1受体的单抗Teprotumumab（Tepezza）给出积极评审意见。三项RCT临床显示活动期患者24周内眼球突出度平均减少2.0–2.3mm，临床活动评分大幅改善；慢性期亦有约1.5mm降低。基于肌肉痉挛、脱发等可管理风险，EMA将附加风险最小化措施并转交欧委会审批。申请人为Amgen Europe B.V.，是全球领先的生物技术公司安进在欧洲的子公司。图：甲状腺眼病首创疗法Tepezza获积极审评意见https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease3. CHMP会议要点：新药、扩展、再审与撤回4月22–25日的EMA人药委员会（CHMP）会议，推荐16款新药（包含Vertex囊性纤维化三联疗法、差异化适应症的多款孤用药及九款生物类似药），推动10项适应症扩展，如武田制药的Adcetris多淋巴瘤适应等；同时，礼来的阿尔茨海默药物Kisunla申请再审，Biohaven和辉瑞的两款产品适应失利撤回。整体来看，EMA通过CMA平衡未满足需求与证据要求，也显示出对成熟产品生命周期管理策略的开放态度。图：2025年4月22日至25日人药委员会( CHMP )会议要点https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025欧洲EDQM高通量测序入典，提升生物制品安全4月23日，EDQM欧洲药典委员会（EPC）正式采纳通则“2.6.41病毒外源性因子检测的高通量测序（HTS）”，自2026年4月1日起生效。新章节涵盖非靶向与靶向HTS方法学、基因组/转录组数据分析和验证指南，驱动与ICH Q5A(R2)修订及WHO国际参考组协同，助力生物制品从传统动物实验迈向更高灵敏度的分子检测。图：高通量测序入欧洲药典https://www.edqm.eu/en/-/epc-adopts-cutting-edge-hts-chapter-to-enhance-viral-contaminant-detection-in-biological-products英国MHRA创新疗法齐发1. 4月拜耳新药Acoramidis获批，用于心肌病4月25日，MHRA批准拜耳制药的Acoramidis（Beyonttra），用于治疗因变异型或野生型转甲状腺素蛋白淀粉样变性（ATTR-CM）引起的心肌病（心肌损伤）成年患者。，632例国际多中心研究显示30个月心血管住院率和死亡率显著降低，6分钟步行距离改善。口服每日两次，通过IRP机制加速审评。图：拜耳新药Acoramidis获批，用于心肌病https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy2. 白血病CAR-T疗法Obecabtagene autoleucel获有条件上市批准4月25日，obecabtagene autoleucel (Aucatzyl) 这一CART疗法获条件上市许可，用于治疗复发或难治性B 细胞前体急性淋巴细胞白血病 (ALL) 的成年患者。FELIX研究153例数据显示12个月总生存率81%、完全缓解55%，后续需提供更多数据满足全面上市要求。上市许可人为Autolus Therapeutics，是一家总部位于英国的生物制药公司，该公司于2014 年从伦敦大学学院（UCL）分拆成立。图：白血病CAR T疗法Obecabtagene autoleucel获有条件上市批准https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia3. 百健新药在英获批4月23日，MHRA批准了 omaveloxolone（Skyclarys），这是英国首个针对 16 岁及以上患者的治疗药物，用于治疗一种名为弗里德赖希共济失调的罕见神经退行性运动障碍。弗里德赖希共济失调是最常见的遗传性共济失调（由遗传基因引起）。据估计，每5万人中至少有1人患有此病。弗里德赖希共济失调的症状包括平衡和运动障碍，并会随着时间的推移逐渐恶化。该药物为口服胶囊剂。上市许可人为Biogen Netherlands B.V.，为全球领先的生物技术公司 Biogen Inc.（百健公司）在荷兰的全资子公司。图：百健新药在英获批https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-friedreichs-ataxia-omaveloxolone4. 辉瑞血友病药物获批4月22日，MHRA批准辉瑞药物Marstacimab（Hympavzi），用于预防或减少12岁及以上、体重至少35公斤的血友病A和B患者的出血。该药物是同类药物中第一个通过针对血液凝固过程中的蛋白质来发挥作用的药物。Marstacimab每周一次皮下注射，使用预充式注射器或注射笔。患者或护理人员经过适当培训后即可自行注射药物。图：辉瑞血友病药物获批https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b国际ISPE发布数字化验证良好实践指南国际制药工程协会（ISPE）发布了《数字化验证良好实践指南》（Good Practice Guide: Digital Validation），为制药企业提供从传统验证向数字化框架过渡提供了指导。这本90页的指导文件，旨在帮助制药企业采用数字化验证工具（Digital Validation Tools, DVT），提升数据可靠性、运营效率和监管合规性。该指南由全球专家团队开发，其核心成员来自于武田制药、葛兰素史克等全球药企和行业咨询公司。ISPE表示，指南结合真实案例和结构化方法，适用于从初创到优化现有系统的各类企业。（点击此处查看相关阅读）图：ISPE最新发布《数字化验证良好实践指南》https://ispe.org/publications/guidance-documents/good-practice-guide-digital-validation结语回望过去一周，FDA、EMA、EDQM、MHRA、PDA和ISPE等监管与行业机构的多项决策、指南与更新，彰显监管严谨，又鼓励技术革新。从食品安全到精准疗法，从分子检测到数字化治理，昭示出全球制药行业正朝着更健康、更高效、更可持续的未来协奏。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月6日 | 线上 | 新法规和应用场景下的全球包材注册策略和最佳实践5月8日 | 线上 | 双维度解锁无菌药品生产污染防控策略

孤儿药突破性疗法上市批准医药出海申请上市

2025-04-29

·PMLiVE

MHRA approves Autolus Therapeutics’ Aucatzyl to treat acute lymphoblastic leukaemia

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Autolus Therapeutics’ Aucatzyl (obecabtagene autoleucel) to treat acute lymphoblastic leukaemia (ALL). The CAR T-cell therapy has been specifically authorised for use in adults with relapsed or refractory (R/R) B-cell precursor ALL. Approximately 765 new cases of ALL, an aggressive blood cancer, are diagnosed in the UK every year. Up to 50% of patients will relapse following frontline treatment, and survival rates remain very poor in adults with R/R disease. Administered as an intravenous infusion, Aucatzyl works by taking patients’ T cells and transforming them into CD19-targeting CAR T cells. When transferred back into the body, these modified cells can recognise and destroy the cancer cells. The MHRA’s decision on the therapy was supported by data from the open-label FELIX trial, in which 52 of the 94 R/R B-cell ALL patients who were given at least one infusion of Aucatzyl showed complete remission of the disease with an 81% probability of overall survival at 12 months. Julian Beach, MHRA interim executive director, healthcare quality and access, said: “We are committed to making innovative treatment options, like CAR T-cell therapy, available to patients as quickly as possible, ensuring our approval is underpinned by robust evidence of efficacy alongside the highest standards of safety. “We are assured that the appropriate regulatory standards for the approval of this medicine have been met.” In line with its conditional marketing authorisation pathway, the MHRA will review new efficacy and safety information for Aucatzyl at least once every year. The therapy is now being evaluated by the National Institute for Health and Care Excellence (NICE) for potential use on the NHS. FELIX lead investigator, Claire Roddie, University College London Cancer Institute, said: “Having treated a number of patients with Aucatzyl as part of the FELIX clinical trial, I am delighted that we have moved closer to eligible R/R B-cell ALL patients being able to access Aucatzyl. “We now look forward to NICE completing its assessment of the medicine to potentially make it an option for eligible patients on the NHS.”

细胞疗法免疫疗法临床结果临床研究上市批准

2025-04-15

·GlobeNewswire-Health Care

Remix Therapeutics Appoints Linda C. Bain to Board of Directors

WATERTOWN, Mass., April 15, 2025 (GLOBE NEWSWIRE) -- Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced the appointment of life science executive, Linda C. Bain, to its Board of Directors. Ms. Bain is a Venture Partner at Atlas Venture and currently serves as a Board Advisor to Remix Therapeutics. “Linda brings more than 20 years of financial, operational and executive leadership success at innovative life science companies,” said Matt Patterson, Chairman of Remix Therapeutics Board of Directors. “Her substantial knowledge and experience successfully managing organizations through significant periods of growth will be invaluable to support Remix as it advances its innovative clinical-stage and discovery programs.” “Having partnered with Linda in her role as Board Advisor to Remix, we are excited to have her join our Board of Directors,” said Pete Smith, PhD, Co-Founder and Chief Executive Officer of Remix Therapeutics. “Linda’s impressive track record of leading financial and operational strategy for both private and publicly held biopharmaceutical companies will be beneficial to Remix’s evolution and achieving our goals.” Prior to Atlas Venture, Ms. Bain served as Chief Operating Officer (COO) and Chief Financial Officer (CFO) of Mariana Oncology and oversaw the company’s acquisition by Novartis in 2024 for $1B upfront and up to $750M in potential milestone payments. She has raised nearly $1B in capital for public and private life science companies over the past 10+ years. Prior to Mariana Oncology, Ms. Bain served as CFO of Codiak BioSciences, CFO of Avalanche Biotechnologies, and Vice President of Finance, Business Operations, and Treasurer at bluebird bio. She previously held senior roles at Genzyme, Fidelity Investments, AstraZeneca and Deloitte & Touche. She is currently on the Board of Directors of Arvinas, Autolus Therapeutics and Hemab Therapeutics.“I am thrilled with the opportunity to contribute my skills and experience to a Company at the forefront of tackling disease drivers with a novel approach, modulating RNA processing with small molecules,” said Ms. Bain. “I look forward to joining the Remix Board of Directors and working with the team to best position the Company to deliver on this important mission and advancing new options for patients in great need of new treatments.” About Remix TherapeuticsRemix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and address disease drivers at their origin. Remix's REMaster™ technology platform leverages cutting-edge data science, biomolecular sciences and chemistry approaches to identify orally administered compounds that modulate gene expression. Remix's innovative therapeutic approach led to the discovery of REM-422, a first-in-class RNA processing modulator in oncology, now being evaluated in Phase 1 clinical studies to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (HR-MDS) and adenoid cystic carcinoma (ACC). For more information visit www.remixtx.com. Contacts: Media Contact:Lisa BuffingtonBuffington Commslbuffington@remixtx.com Investor Contact:Will O'Connor Precision AQWill.OConnor@precisionaq.com

高管变更临床1期

100 项与 Autolus Therapeutics Plc 相关的药物交易

登录后查看更多信息

100 项与 Autolus Therapeutics Plc 相关的转化医学

登录后查看更多信息

组织架构

使用我们的机构树数据加速您的研究。

或

查看完整样例数据

管线布局

2025年05月08日管线快照

管线布局中药物为当前组织机构及其子机构作为药物机构进行统计，早期临床1期并入临床1期，临床1/2期并入临床2期，临床2/3期并入临床3期

临床前

临床1期

临床2期

临床3期

批准上市

其他

登录后查看更多信息

当前项目

药物(靶点)	适应症	全球最高研发状态

欧贝妥基奥仑赛 ( CD19 )	前体B细胞急性淋巴细胞白血病更多	批准上市
AUTO-3 ( CD19 x CD22 )	肿瘤更多	临床3期
AUTO-4(University College London) ( TRBC1 )	间变性大细胞淋巴瘤更多	临床2期
AUTO-8 ( BCMA x CD19 )	多发性骨髓瘤更多	临床1期
AUTO-1-NG ( CD19 x CD22 )	急性淋巴细胞白血病更多	临床1期