Immunotherapy With CD19/22 CAR Redirected T-cells for High Risk, Relapsed Paediatric CD19+ and/or CD22+ Acute Lymphoblastic Leukaemia and Other Haematological Malignancies

This study aims to evaluate the safety, efficacy and duration of response of CD19/22 Chimeric Antigen Receptor (CAR) redirected autologous T-cells in children with high risk, relapsed CD19+ and/ or CD22+ haematological malignancies.

开始日期2016-04-01

申办/合作机构

University College London

100 项与 CD19/22 CAR T-cells(University College, London) 相关的临床结果

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100 项与 CD19/22 CAR T-cells(University College, London) 相关的转化医学

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100 项与 CD19/22 CAR T-cells(University College, London) 相关的专利（医药）

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项与 CD19/22 CAR T-cells(University College, London) 相关的文献（医药）

2024-08-01·Cellular oncology (Dordrecht)

Efficacy of programmed cell death 1 inhibitor maintenance after chimeric antigen receptor T cells in patients with relapsed/refractory B-cell non-Hodgkin-lymphoma

Article

作者: Zhu, Xiaojian ; Meng, Fankai ; Wei, Jia ; Wang, Na ; Yang, Yang ; Jiang, Lijun ; Xin, Xiangke ; Zhang, Yicheng ; Wang, Jue ; Xu, Jinhuan ; Huang, Liang ; Cao, Yang ; Zheng, Miao ; Li, Chunrui ; Xiao, Yi

INTRODUCTION:

Chimeric antigen receptor (CAR)-T cells obtained long-term durability in about 30% to 40% of relapsed/refractory (r/r) B-cell non-Hodgkin lymphoma (B-NHL). Maintenance therapy after CAR-T is necessary, and PD1 inhibitor is one of the important maintenance therapy options.

METHODS:

A total of 173 r/r B-NHL patients treated with PD1 inhibitor maintenance following CD19/22 CAR-T therapy alone or combined with autologous hematopoietic stem cell transplantation (ASCT) from March 2019 to July 2022 were assessed for eligibility for two trials. There were 81 patients on PD1 inhibitor maintenance therapy.

RESULTS:

In the CD19/22 CAR-T therapy trial, the PD1 inhibitor maintenance group indicated superior objective response rate (ORR) (82.9% vs 60%; P = 0.04) and 2-year progression-free survival (PFS) (59.8% vs 21.3%; P = 0.001) than the non-maintenance group. The estimated 2-year overall survival (OS) was comparable in the two groups (60.1% vs 45.1%; P = 0.112). No difference was observed in the peak expansion levels of CD19 CAR-T and CD22 CAR-T between the two groups. The persistence time of CD19 and CD22 CAR-T in the PD1 inhibitor maintenance group was longer than that in the non-maintenance group. In the CD19/22 CAR-T therapy combined with ASCT trial, no significant differences in ORR (81.4% vs 84.8%; P = 0.67), 2-year PFS (72.3% vs 74.9%; P = 0.73), and 2-year OS (84.1% vs 80.7%; P = 0.79) were observed between non-maintenance and PD1 inhibitor maintenance therapy groups. The peak expansion levels and duration of CD19 and CD22 CAR-T were not statistically different between the two groups. During maintenance treatment with PD1 inhibitor, all adverse events were manageable. In the multivariable analyses, type and R3m were independent predictive factors influencing the OS of r/r B-NHL with PD1 inhibitor maintenance after CAR-T therapy.

CONCLUSION:

PD1 inhibitor maintenance following CD19/22 CAR-T therapy obtained superior response and survival in r/r B-NHL, but not in the trial of CD19/22 CAR-T cell therapy combined with ASCT.

2024-05-01·Cytotherapy

Prognostic differences between carmustine, etoposide, cytarabine and melphalan (BEAM) and carmustine, etoposide, cytarabine, melphalan and fludarabine (BEAMF) regimens before autologous stem cell transplantation plus chimeric antigen receptor T therapy in patients with refractory/relapsed B-cell non-Hodgkin-lymphoma

Article

作者: Zhu, Xiaojian ; Wei, Jia ; Meng, Fankai ; Lin, Li ; Wang, Na ; Yang, Yang ; Xin, Xiangke ; Zhang, Yicheng ; Xu, Jinhuan ; Wang, Jue ; Huang, Liang ; Cao, Yang ; Zheng, Miao ; Xiao, Yi

BACKGROUND AIMS:

The combination therapy of autologous hematopoietic stem cell transplantation (ASCT) and chimeric antigen receptor T-cell (CART) therapy has been employed to improve outcomes for relapsed or refractory (R/R) B-cell non-Hodgkin-lymphoma (B-NHL). The widely used conditioning regimen before ASCT plus CART therapy reported in the literature was carmustine, etoposide, cytarabine and melphalan (BEAM). However, whether adding fludarabine to the BEAM regimen (BEAMF) can improve the survival of patients with R/R B-NHL remains unknown.

METHODS:

In total, 39 and 19 patients with R/R B-NHL were enrolled to compare clinical outcomes in the BEAM and BEAMF regimens before ASCT plus CD19/22 CART therapy, respectively.

RESULTS:

The objective response (OR) rates at 3 months to BEAM and BEAMF regimens before ASCT plus CD19/22 CART therapy were 71.8% and 94.7%, respectively (P = 0.093). The BEAMF regimen showed a trend towards a superior duration of response compared with the BEAM regimen (P = 0.09). After a median follow-up of 28 months (range: 0.93-51.9 months), the BEAMF regimen demonstrated superior 2-year progression-free survival (PFS) (89.5% versus 63.9%; P = 0.048) and 2-year overall survival (OS) (100% vs 77.3%; P = 0.035) compared with the BEAM regimen. In the multivariable Cox regression analysis, OR at month 3 (responders) was remarkably correlated with better OS (hazard ratio: 0.112, P = 0.005) compared with OR (non-responders).

CONCLUSIONS:

For patients with R/R B-NHL, the BEAMF regimen before ASCT plus CD19/22 CART therapy was correlated with superior PFS and OS than the BEAM regimen, and the BEAMF regimen is a promising alternative conditioning regimen for ASCT plus CAR-T therapy.

2024-05-01·International immunopharmacology

Combination of chidamide and PD-1 blockade in Refractory/Relapsed aggressive large B-cell lymphomas with high risk of failing CAR-T therapy

Article

作者: Huang, Liang ; Yang, Zhuming ; Han, Zhiqiang ; Cao, Yang ; Xu, Hao ; Zhang, Yicheng ; Xiao, Min ; Mei, Yu ; Hong, Zhenya ; Wang, Zhenhao ; Zheng, Miao

BACKGROUND:

Refractoriness and relapse after chimeric antigen receptor T-cell therapy have emerged as major challenges for immunotherapy of aggressive large B-cell lymphoma. Thus far, there is no consensus on how to address treatment failure and whether to administer maintenance therapy following CAR-T cell therapy.

METHODS:

From August 2017 through November 2022, 52 patients with refractory/relapsed aggressive LBCL who had a high risk of resistance to CAR-T cell therapy were given chidamide in combination with a PD-1 inhibitor as maintenance therapy following either CAR19/22 T-cell cocktail therapy or CAR19/22 T-cell cocktail therapy plus autologous stem cell transplantation (ASCT). Another 52 aggressive LBCL patients who had comparable baseline characteristics and received similar therapeutic regimens but did not receive any interventions following CAR-T cell therapy or CAR-T cell therapy plus ASCT were regarded as the control group to evaluate the efficacy and safety of the combination of chidamide and a PD-1 inhibitor.

RESULTS:

Among the 52 patients who received chidamide and a PD-1 inhibitor as maintenance therapy, with a median follow-up of 26.5 months (range: 1.1-53.8), neither the median progression-free survival (PFS) nor overall survival (OS) was reached, and the expected 2-year OS and PFS rates were 89 % and 77 %, respectively, which were superior to those of the control group (p < 0.001). Long-term chidamide administration and a specific genetic subtype of EZB were strongly associated with a better response after chidamide plus PD-1 blockade therapy. Additionally, long-term chidamide administration was significantly associated with prolonged persistence and reactivation of CD19-directed CAR-T cells in the peripheral blood. Adverse effects (AEs) were moderate and reversible, and no treatment-related deaths occurred.

CONCLUSION:

Our results indicate that the combination of chidamide and PD-1 blockade as maintenance therapy could improve the outcomes of aggressive LBCL patients at high risk of failing CAR-T cell therapy.

项与 CD19/22 CAR T-cells(University College, London) 相关的新闻（医药）

2024-08-08

·PRNewswire

Nektar Therapeutics Reports Second Quarter 2024 Financial Results

SAN FRANCISCO, Aug. 8, 2024 /PRNewswire/ -- Nektar Therapeutics (Nasdaq: NKTR) today reported financial results for the second quarter ended June 30, 2024. Cash and investments in marketable securities at June 30, 2024 were $290.6 million as compared to $329.4 million at December 31, 2023. Nektar's cash and marketable securities are expected to support strategic development activities and operations into the third quarter of 2026. "We continue to make excellent progress advancing rezpegaldesleukin in Phase 2 studies in patients with atopic dermatitis and alopecia areata," said Howard W. Robin, President and CEO of Nektar. "We are particularly pleased that enrollment is on track for topline data in the first half of 2025 for atopic dermatitis and topline data in mid-2025 for alopecia areata. For our TNFR2 agonist antibody, NKTR-0165, we are conducting IND-enabling studies with the goal of preparing for an IND submission in the middle of 2025. Recent data at the 2024 EULAR Congress highlighted the potential of this unique AI-generated antibody in inflammatory disorders. Finally, we remain in a strong financial position with a cash runway extending into the third quarter of 2026." Summary of Financial Results Revenue in the second quarter of 2024 was $23.5 million as compared to $20.5 million in the second quarter of 2023. Revenue for the first half of 2024 was $45.1 million as compared to $42.1 million in the first half of 2023. Total operating costs and expenses in the second quarter of 2024 were $73.3 million as compared to $71.1 million in the second quarter of 2023. Total operating costs and expenses in the first half of 2024 were $130.3 million as compared to $227.4 million in the first half of 2023. Operating costs and expenses for the first half of 2024 decreased due to a decrease in restructuring, impairment and costs of terminated program and a one-time $76.5 million non-cash goodwill impairment recognized in the first quarter of 2023. R&D expense was $29.7 million in the second quarter of both 2024 and 2023. R&D expense in the first half of 2024 was $57.1 million as compared to $60.2 million for the first half of 2023. R&D expense decreased for the first half of 2024 primarily due to decreases in employee costs and related facilities costs as well as development expense for NKTR-255 offset by increases in development expenses for rezpegaldesleukin and NKTR-0165. G&A expense was $20.5 million in the second quarter of 2024 as compared to $17.9 million in the second quarter of 2023. G&A expense was $40.7 million in the first half of 2024 as compared to $39.0 million in the first half of 2023. G&A expense increased for both the second quarter and first half of 2024 primarily due to a reduction of facilities costs allocated to research and development expenses partially offset by a decrease in employee costs. Non-cash restructuring and impairment charges in the second quarter of 2024 were $13.3 million and $14.3 million in the first half of 2024. These non-cash charges are related to the declining San Francisco commercial real estate market and real estate lease obligations held by Nektar. Net loss for the second quarter of 2024 was $52.4 million or $0.25 basic and diluted loss per share as compared to a net loss of $51.1 million or $0.27 basic and diluted loss per share in the second quarter of 2023. Net loss in the first half of 2024 was $89.2 million or $0.44 basic and diluted loss per share as compared to a net loss of $188.1 million or $0.99 basic and diluted loss per share in the first half of 2023. Excluding the $13.3 million and $14.3 million in non-cash restructuring and real estate impairment charges, net loss, on a non-GAAP basis, for the second quarter and first half of 2024 were $39.1 million and $74.9 million, respectively, or $0.19 and $0.37 basic and diluted loss per share, respectively. Second Quarter 2024 and Recent Business Highlights In July 2024, our collaborators at Stanford published data from a Phase 1 trial evaluating NKTR-255 in combination with CD19/22 CAR-T cell therapy in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) in Blood, an open-access journal of the American Society of Hematology. These data showed that, at 12 months, remission-free survival for NKTR-255 was double that of historical controls (67% vs 38%). The data also showed that eight out of nine patients (89%) achieved complete remission with or without hematologic recovery, all without detectable measurable residual disease (MRD). In June 2024, Nektar presented the first preclinical data on NKTR-0165 at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress. The data presented show that NKTR-0165 is a unique antibody that selectively binds to TNFR2 on Tregs to enhance its immunosuppressive activities, and could potentially become a first-in-class treatment for various autoimmune diseases, including ulcerative colitis and vitiligo. Nektar plans to initiate first-in-human studies in the first half of 2025. Enrollment remains on track for the two Phase 2b studies of rezpegaldesleukin, one in patients with moderate-to-severe atopic dermatitis and one in patients with severe to very severe alopecia areata. Nektar expects topline data from these studies, respectively, in the first half and in the middle of 2025. Nektar also announced upcoming presentations at the following scientific congress: 2024 European Academy of Dermatology and Venereology (EADV) Congress ePoster P0662: "Serum proteomic biomarker analysis of the interleukin-2 receptor pathway agonist rezpegaldesleukin in patients with atopic dermatitis", Yu, D. ePoster P0600 (Trial in Progress): "A Phase 2b, Randomized, Double-Blinded, Parallel-Group, Placebo-Controlled, International, Multicenter, Study to Evaluate the Efficacy and Safety of Rezpegaldesleukin in Adults with Moderate-to-Severe Atopic Dermatitis", Gkalpakiotis, S. ePoster P2080 (Trial in Progress): "A Phase 2b Study Evaluating the Efficacy and Safety of Single Agent Rezpegaldesleukin, an Interleukin-2 Receptor (IL-2R) Pathway Agonist, in the Treatment of Severe to Very Severe Alopecia Areata", Reich, A. Conference Call to Discuss Second Quarter 2024 Financial Results Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, August 8, 2024. This press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the Home Page and Investors section of the Nektar website: . The web broadcast of the conference call will be available for replay through September 8, 2024. To access the conference call, please pre-register at Nektar Earnings Call Registration. All registrants will receive dial-in information and a PIN allowing them to access the live call. About Nektar Therapeutics Nektar Therapeutics is a clinical-stage biotechnology company focused on developing treatments that address the underlying immunological dysfunction in autoimmune and chronic inflammatory diseases. Nektar's lead product candidate, rezpegaldesleukin (NKTR-358), is a novel, first-in-class regulatory T cell stimulator being evaluated in two Phase 2b clinical trials, one in atopic dermatitis and one in alopecia areata. Our pipeline also includes a preclinical candidate NKTR-0165, which is a bivalent tumor necrosis factor receptor type II agonist antibody. Nektar, together with various partners, is also evaluating NKTR-255, an investigational IL-15 receptor agonist designed to boost the immune system's natural ability to fight cancer, in several ongoing clinical trials. Nektar is headquartered in San Francisco, California. For further information, visit and follow us on LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements which can be identified by words such as: "will," "expect," "develop," "potential," "advance," "anticipate," and similar references to future periods. Examples of forward-looking statements include, among others, statements regarding the therapeutic potential of, and future development plans for, rezpegaldesleukin, NKTR-0165, and NKTR-255. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, anticipated events and trends, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause our actual results to differ materially from those indicated in the forward-looking statements include, among others: (i) our statements regarding the therapeutic potential of rezpegaldesleukin, NKTR-0165, and NKTR-255 are based on preclinical and clinical findings and observations and are subject to change as research and development continue; (ii) rezpegaldesleukin, NKTR-0165, and NKTR-255 are investigational agents and continued research and development for these drug candidates is subject to substantial risks, including negative safety and efficacy findings in future clinical studies (notwithstanding positive findings in earlier preclinical and clinical studies); (iii) rezpegaldesleukin and NKTR-255 are in clinical development and the risk of failure is high and can unexpectedly occur at any stage prior to regulatory approval; (iv) the timing of the commencement or end of clinical trials and the availability of clinical data may be delayed or unsuccessful due to challenges caused by health epidemics, including the COVID-19 pandemic, regulatory delays, slower than anticipated patient enrollment, manufacturing challenges, changing standards of care, evolving regulatory requirements, clinical trial design, clinical outcomes, competitive factors, or delay or failure in ultimately obtaining regulatory approval in one or more important markets; (v) we may not achieve the expected cost savings we expect from our 2022 corporate restructuring and reorganization plan or our 2023 cost restructuring plan and we may undertake additional restructuring and cost-saving activities in the future, (vi) patents may not issue from our patent applications for our drug candidates, patents that have issued may not be enforceable, or additional intellectual property licenses from third parties may be required; and (vii) certain other important risks and uncertainties set forth in our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 10, 2024. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Contact: For Investors: Vivian Wu of Nektar Therapeutics 628-895-0661 For Media: David Rosen of Argot Partners (212) 600-1902 [email protected] SOURCE Nektar Therapeutics

临床结果临床2期临床1期财报ASH会议

2024-02-10

·药明康德

基因疗法关键性临床结果积极，睿健医药帕金森病细胞疗法完成首例患者给药… | CGT周报

▎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。REGENXBIO公司宣布，在研基因疗法RGX-121在治疗II型黏多糖贮积症（MPS II）的关键性临床试验中达到主要终点，显著降低患者脑脊液中的生物标志物水平。这些结果支持该公司在2024年使用加速批准通道向美国FDA递交生物制品许可申请。睿健医药宣布，该公司用于治疗帕金森病的在研细胞疗法NouvNeu001在1/2期临床试验中完成首例患者给药。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✦———◇REGENXBIO公司宣布，在研基因疗法RGX-121在治疗II型黏多糖贮积症的关键性临床试验中达到主要终点，显著降低患者脑脊液中的生物标志物水平。新闻稿指出，这些结果支持该公司在2024年使用加速批准通道向美国FDA递交生物制品许可申请。在临床试验的关键性阶段，接受RGX-121治疗的MPS II患者的脑脊液D2S6水平平均降低86%，接近正常水平，D2S6是大脑疾病活动的关键生物标志物之一。此外，对在临床试验的剂量探索阶段接受治疗的患者的长期随访数据显示，在接受关键性试验阶段推荐剂量治疗的患者中，80%的患者在最近一次随访时不需要接受标准的静脉输注酶替代疗法（ERT）。RGX-121是一种一次性的基因疗法，旨在使用AAV9载体将编码人艾杜糖酸-2-硫酸酯酶（IDS）的转基因递送至中枢神经系统（CNS）。RGX-121已获得美国FDA授予的孤儿药资格、罕见儿科疾病认定、快速通道资格和再生医学先进疗法认定。此外，REGENXBIO公司宣布在研基因疗法RGX-202在治疗杜氏肌营养不良（DMD）患者的1/2期临床试验中获得积极中期结果。RGX-202在接受治疗的最初5名患者中表现出良好的安全性，未发现药物相关严重不良事件。给药3个月后，其中一名患者的微抗萎缩蛋白表达水平达到健康对照的83.4%，血清肌酐激酶（CK）水平在10周时比基线降低93%。RGX-202是一款基于AAV8载体的基因疗法，旨在递送表达微抗肌萎缩蛋白的转基因进入骨骼肌和心肌组织。目前，这一试验更高剂量组的患者注册已经完成。REGENXBIO预计将在2024年下半年启动关键性临床试验。◇Ultragenyx Pharmaceutical公司宣布，在研基因疗法UX111在治疗IIIA型黏多糖贮积症（MPS IIIA）的1/2期临床试验中，迅速和显著降低患者脑脊液中的硫酸乙酰肝素（heparan sulfate）水平。同时，硫酸乙酰肝素水平的持久降低与患者的长期认知发育具有相关性。UX111旨在使用AAV9载体，递送SGSH基因的功能性拷贝到中枢神经系统和外周器官，从而弥补SGSH酶的缺失。SGSH酶的缺失导致硫酸乙酰肝素在大脑和身体其它部位累积，引发进行性细胞损伤。◇Sangamo Therapeutics公司公布了在研基因疗法isaralgagene civaparvovec（ST-920）治疗法布里病（Fabry disease）的1/2期临床试验的最新结果。截至2023年9月19日，总计24名患者接受了治疗，isaralgagene civaparvovec表现出良好的耐受性，大部分不良事件为1/2级。疗效方面，所有12名在加入研究时使用酶替代疗法，并在接受治疗后停止ERT的患者，截至2023年9月19日仍然不需要ERT疗法。其中11名患者的α-半乳糖苷酶水平超过正常人的水平，1名患者的水平与正常人类似。在13名随访超过12个月的患者中，肾功能维持稳定，患者报告总体疾病严重性、生活质量和胃肠道症状获得显著改善。Isaralgagene civaparvovec使用重组AAV2/6载体，将表达α-半乳糖苷酶的GLA基因递送到肝脏，旨在通过一次性治疗获得持久疗效，减轻ERT输注负担。◇AvenCell公司宣布，在研同种异体CAR-T疗法AVC-201在1A期临床试验中完成首例患者给药，用于治疗复发/难治性急性髓系白血病和其它CD123阳性血液癌症患者。AVC-201是一款通过CRISPR基因编辑技术改造的同种异体CAR-T疗法，靶向CD123抗原。它的独特之处在于结合了两种不同技术。首先是AvenCell公司的UniCAR通用/可转换技术，这一技术包含两个组件，基因工程改造的T细胞表达一种“通用”的受体，它只有在与靶向生物制品分子结合时才会被激活，而靶向生物制品分子的一端可以与肿瘤抗原结合，另一端与T细胞上表达的受体结合。这种设计让靶向模块成为启动和关闭CAR-T疗法活性的开关。另一个技术平台是由Intellia Therapeutics公司开发的基因编辑平台，通过CRISPR/Cas9基因编辑防止移植物抗宿主病（GvHD）以及由患者的先天和适应性免疫系统介导的免疫排斥。◇Triumvira Immunologics公司宣布，其靶向Claudin 18.2的T细胞疗法TAC101-CLDN18.2在1/2期临床试验中完成首例患者给药。TAC101-CLDN18.2是一款创新工程化自体T细胞疗法。它表达的T细胞抗原结合子（TAC）在靶向Claudin 18.2的同时利用了天然T细胞受体复合体的内在信号通路，有利于防止T细胞的过度激活和过早耗竭。这项临床试验将在至少接受过2种前期治疗的Claudin 18.2阳性实体瘤患者中进行。◇睿健医药宣布，该公司用于治疗帕金森病的在研细胞疗法NouvNeu001在1/2期临床试验中完成首例患者给药，该患者也已经成功度过观察期。药明康德内容团队专访魏君博士NouvNeu001是一款源于诱导多能干细胞（iPSC），化学诱导分化的多巴胺能神经元前体细胞。睿健医药联合创始人兼首席执行官魏君博士早先接受药明康德内容团队采访时介绍：“秉承着‘从患者的需求出发，解决真正的临床需求’的宗旨，睿健医药以安全性和可及性作为首要原则，从源头创新，最终形成了独特的‘AI+化学诱导’体系。具体而言，我们首先利用知识图谱进行发育生物学核心基因富集分析；然后结合组学分析及互作网络拓扑结构计算锚定特定发育关键通路及其诱导化合物，从而确定最适合于治疗的细胞及化学诱导开发工艺；最终，只需要少数合适的化学小分子与基础培养基的简单组合，即可以在工业量级完成多能干细胞向目标细胞的高效精准转化。这一理念也帮助公司在研产品实现更快和更好的治疗效果。”◇Adverum Biotechnologies公司宣布，在研基因疗法ixoberogene soroparvovec（ixo-vec，曾用名ADVM-022）在治疗湿性年龄相关性黄斑变性（AMD）的2期临床试验中获得积极初步结果。在接受剂量为6E10和2E11的ixo-vec治疗的患者中，26周时患者接受抗VEGF疗法注射的频率分别降低90%和94%。该公司同时宣布完成1.275亿美元融资。Ixo-vec使用该公司独有的腺相关病毒载体AAV.7m8，将编码抗VEGF-A的融合蛋白aflibercept的转基因通过玻璃体内注射递送到视网膜中。这一疗法旨在通过一次注射，提供长期疗效，降低患者接受频繁抗VEGF疗法注射的负担。◇Beacon Therapeutics公司宣布，在研基因疗法AGTC-501在治疗X连锁色素性视网膜炎（XLRP）的2期临床试验中获得积极结果。12个月的随访数据显示，AGTC-501在表现出良好的安全性和耐受性同时，提高患者的视觉功能。接受高剂量AGTC-501治疗的眼睛应答率为63%。XLRP主要由色素性视网膜炎GTP酶调节因子（RPGR）基因突变引起。AGTC-501表达全长RPGR蛋白，因此有望挽救视杆细胞和视锥细胞的功能，使其非常适合作为改善 XLRP患者生活的潜在治疗方法。◇Krystal Biotech宣布，基因疗法beremagene geperpavec（B-VEC）作为眼药水，治疗营养不良性大疱性表皮松解症（DEB）患者结膜炎的研究发表在《新英格兰医学杂志》上。新闻稿指出，这是这款基因疗法首次用于治疗DEB患者的眼科并发症。B-VEC是一款使用1型单纯疱疹病毒（HSV-1）作为载体，递送编码VII型胶原蛋白（COL7）的转基因至患者皮肤细胞的基因疗法。通过表达COL7可以帮助因为COL7缺失导致皮肤损伤的DEB患者伤口愈合。25%的DEB患者会出现眼部并发症，包括角膜损伤，水泡和疤痕组织的产生，导致视力受损。这名患者在接受手术治疗去除疤痕组织后，使用B-VEC眼药水的滴注治疗。试验结果显示，B-VEC表现出良好的耐受性，在使用3个月后患者的角膜上皮完全愈合，接受治疗8个月时视力显著提高，接近正常水平。———✦合作、融资、M&A✦———◇Neurona Therapeutics宣布成功完成了1.2亿美元的融资。筹集的资金将用于推进公司即用型细胞疗法产品线的发展，包括其主打在研候选产品NRTX-1001。目前，NRTX-1001正在开放标签、单臂1/2期临床试验中接受评估，用于治疗药物抵抗性颞叶内侧癫痫（MTLE）。去年12月公布的试验结果显示，前两名受试者在六个月基线期间分别每月发作32次和14次癫痫，接受NRTX-1001治疗后一年多时间内，总体癫痫发作次数相比基线降低了95%以上，且影响意识的严重局灶性癫痫消失。NRTX-1001是一种源于自人类多能干细胞的再生神经细胞疗法候选产品。这些完全分化的中间神经元分泌抑制性神经递质γ-氨基丁酸（GABA）。一次给药之后，这些人类中间神经元可在大脑的癫痫区域实现靶向整合并持久地抑制癫痫活动。◇BioNTech和Autolus Therapeutics日前宣布，双方达成一项战略合作，旨在推进两家公司的自体CAR-T细胞疗法的开发和商业化。根据协议，Autolus公司将获得5000万美元的预付款，BioNTech公司还将对Autolus进行2亿美元的股份投资。BioNTech将有权使用Autolus在英国的生产能力，临床试验点以及商业化网络，加速推进BioNTech的在研疗法BNT211在更多CLDN6阳性肿瘤类型中的开发。BioNTech预计到2024年底在管线中包括10个以上潜在注册性临床试验。Autolus将领导其在研疗法AUTO1/22和AUTO6NG在任何肿瘤适应症中的开发和商业化，BioNTech有选择权支持某些开发活动并在某些地区共同商业化这两个候选产品。AUTO1/22是一款双靶点CAR-T疗法，在靶向CD19的同时还靶向CD22。AUTO6NG是一款靶向GD2的新一代T细胞疗法，它结合了额外的细胞编程元件，来克服肿瘤微环境的免疫抑制机制。Autolus还授予BioNTech独家许可，以开发和商业化利用Autolus的某些专有结合子（binders）的治疗产品。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲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：[1] Adverum Biotechnologies Announces Positive Preliminary Efficacy and Safety Data from LUNA Phase 2 Trial of Ixo-vec in Patients with Wet AMD. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/08/2825871/32452/en/Adverum-Biotechnologies-Announces-Positive-Preliminary-Efficacy-and-Safety-Data-from-LUNA-Phase-2-Trial-of-Ixo-vec-in-Patients-with-Wet-AMD.html[2] Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-12-month-data-from-phase-2-skyline-trial-of-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-302056840.html[3] Krystal Biotech Announces Publication in the New England Journal of Medicine on the Application of B-VEC to Treat Ocular Complications in Patient with Dystrophic Epidermolysis Bullosa. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/08/2825862/0/en/Krystal-Biotech-Announces-Publication-in-the-New-England-Journal-of-Medicine-on-the-Application-of-B-VEC-to-Treat-Ocular-Complications-in-Patient-with-Dystrophic-Epidermolysis-Bull.html[4] BioNTech and Autolus Announce Strategic CAR-T Cell Therapy Collaboration to Advance Pipeline and Expand Late-Stage Programs. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/08/2825810/0/en/BioNTech-and-Autolus-Announce-Strategic-CAR-T-Cell-Therapy-Collaboration-to-Advance-Pipeline-and-Expand-Late-Stage-Programs.html[5] Genenta Demonstrated Reprogramming of the Tumor Microenvironment in GBM Patients, Paving the Way for Innovative Treatments of Solid Tumors. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/08/2825783/0/en/Genenta-Demonstrated-Reprogramming-of-the-Tumor-Microenvironment-in-GBM-Patients-Paving-the-Way-for-Innovative-Treatments-of-Solid-Tumors.html[6] REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/regenxbio-announces-pivotal-trial-of-rgx-121-for-the-treatment-of-mps-ii-achieves-primary-endpoint-302056283.html[7] REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/regenxbio-announces-completion-of-enrollment-in-cohort-2-and-additional-positive-interim-data-in-affinity-duchenne-trial-302055713.html[8] Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA). Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/06/2824223/20739/en/Ultragenyx-Announces-Data-Demonstrating-Treatment-with-UX111-Results-in-Significant-Reduction-in-Heparan-Sulfate-Exposure-in-Cerebrospinal-Fluid-Correlated-with-Improved-Long-term-.html[9] Sangamo Therapeutics Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit and Differentiated Safety Profile. Retrieved February 8, 2024, from https://www.businesswire.com/news/home/20240205333085/en[10] Adverum Biotechnologies Announces $127.5 Million Private Placement Financing. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/05/2823441/32452/en/Adverum-Biotechnologies-Announces-127-5-Million-Private-Placement-Financing.html[11] Neurona Therapeutics Raises $120M to Advance Groundbreaking Pipeline of Regenerative Cell Therapy Candidates for Chronic Neurological Disorders. Retrieved February 8, 2024, from https://www.globenewswire.com/news-release/2024/02/08/2825959/0/en/Neurona-Therapeutics-Raises-120M-to-Advance-Groundbreaking-Pipeline-of-Regenerative-Cell-Therapy-Candidates-for-Chronic-Neurological-Disorders.html[12] Coeptis Therapeutics to Pursue Phase 2 Program Using Proprietary First-in-Class Allogeneic NK Cell Therapy for the Treatment of Viral Infections. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/coeptis-therapeutics-to-pursue-phase-2-program-using-proprietary-first-in-class-allogeneic-nk-cell-therapy-for-the-treatment-of-viral-infections-302055678.html[13] AvenCell Announces First Patient Dosed in a Phase IA Study with lead product candidate AVC-201, a Novel Allogeneic CD123-Directed Switchable CAR-T Investigational Therapy for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia and other CD123 Hematological Malignancies. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/avencell-announces-first-patient-dosed-in-a-phase-ia-study-with-lead-product-candidate-avc-201-a-novel-allogeneic-cd123-directed-switchable-car-t-investigational-therapy-for-the-treatment-of-relapsedrefractory-acute-myeloid-leuk-302053495.html[14] Triumvira Immunologics Announces First Patient Dosed in Phase I/II Cell Therapy Trial of TAC101-CLDN18.2 for the Treatment of Claudin 18.2+ Solid Tumors. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/triumvira-immunologics-announces-first-patient-dosed-in-phase-iii-cell-therapy-trial-of-tac101-cldn18-2-for-the-treatment-of-claudin-18-2-solid-tumors-302052750.html[15] NouvNeu001 Achieves Milestone with Successful Dosing of First Patient, Signaling Smooth Progress in iRegene Therapeutics' Multicenter Clinical Trial for Innovative Novel Parkinson's Disease Therapy. Retrieved February 8, 2024, from https://www.prnewswire.com/news-releases/nouvneu001-achieves-milestone-with-successful-dosing-of-first-patient-signaling-smooth-progress-in-iregene-therapeutics-multicenter-clinical-trial-for-innovative-novel-parkinsons-disease-therapy-302053243.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

孤儿药基因疗法快速通道临床2期临床结果

2024-02-08

·BioSpace

BioNTech and Autolus Announce Strategic CAR-T Cell Therapy Collaboration to Advance Pipeline and Expand Late-Stage Programs

Strategic alliance leverages manufacturing and commercial infrastructure as well as technology with the aim to advance both companies’ autologous CAR-T programs towards market, pending market authorization BioNTech secures the right to utilize Autolus’ manufacturing capacity in a cost-efficient set-up to accelerate the development of BNT211 into pivotal trials in CLDN6+ tumors BioNTech to support launch and expansion of development program of Autolus’ lead cell therapy candidate obe-cel and will receive a royalty on net sales BioNTech has co-commercialization options for Autolus’ AUTO1/22 and AUTO6NG programs BioNTech has the option to access a suite of Autolus target binders and cell programming technologies to support BioNTech’s development of in vivo cell therapy and antibody-drug conjugate candidates BioNTech agrees to invest $200 million in Autolus MAINZ, Germany and LONDON, United Kingdom, February 8, 2024 – BioNTech SE (Nasdaq: BNTX, “BioNTech”), a next-generation immunotherapy company pioneering novel therapies for cancer and other serious diseases, and Autolus Therapeutics plc (Nasdaq: AUTL, “Autolus”), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced a strategic collaboration aimed at advancing both companies’ autologous CAR-T programs towards commercialization, pending regulatory authorizations. In connection with the strategic collaboration, the companies entered into a license and option agreement and a securities purchase agreement. “The collaboration with Autolus enables us to expand our BNT211 program into trials for multiple cancer indications in a cost-efficient way. Autolus' state-of-the-art manufacturing facilities’ set-up for clinical and commercial supply will enhance our own capacities in addition to our existing U.S. supply network and the ongoing expansion of our site in Gaithersburg, Maryland,” said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. “Furthermore, this collaboration grants us access to Autolus' precise cell targeting tools to further support BioNTech’s development of in vivo cell therapy and antibody-drug conjugate candidates." “We see a remarkable opportunity to leverage our core capabilities, accelerate pipeline programs, realize cost-efficiencies and expand opportunities beyond autologous cell therapies,” said Dr. Christian Itin, CEO of Autolus. “We look forward to investing a portion of the capital raised on delivering on obe-cel’s path in adult acute lymphoblastic leukaemia, potentially offering another treatment option for patients where there is still an unmet medical need. This collaboration creates a path for accelerating our respective oncology pipeline programs and broadening the use of Autolus’ technology outside of autologous cell therapy applications.” BioNTech has agreed to purchase $200 million of Autolus’ American Depositary Shares in a private placement. BioNTech will have a right to appoint a director to the Board of Autolus. Under the terms of the license and option agreement, BioNTech will make a cash payment of $50 million and is granted the following rights in exchange: BioNTech is eligible to receive an up to mid-single digit royalty on obe-cel net sales. Autolus will retain full rights to and control of the development and commercialization of obe-cel. BioNTech has the option to access Autolus’ commercial and clinical site network, manufacturing capacities in the United Kingdom and commercial supply infrastructure in a cost-efficient set-up in order to accelerate the development of BNT211 in additional CLDN6+ tumor types. BioNTech plans to have 10 or more ongoing potentially registrational clinical trials in the pipeline by the end of 2024, including its fully owned CLDN6 CAR-T program BNT211 in relapsed or refractory germ cell tumors. Autolus will lead the development and commercialization for AUTO1/22 and AUTO6NG in any oncology indication with BioNTech having an option to support certain development activities and co-commercialize both candidates in certain territories. If BioNTech exercises an option, it will receive a profit share with respect to such exercised product candidate worldwide while Autolus will be eligible to receive an option exercise fee, milestone payments and co-funding of development expenses. Autolus granted BioNTech an exclusive license to develop and commercialize therapeutics incorporating certain of Autolus’ proprietary binders along with options to license binders and cell programming technology for use in BioNTech’s in vivo cell therapy development programs and investigational antibody-drug conjugates. If BioNTech exercises an option, Autolus will be eligible to receive exercise fees and milestones payments, with low-single digit royalties on net sales of the licensed products. About BioNTech’s cell and gene therapy portfolio BioNTech has been active in the development of cell and gene therapies since 2009. Today, it is a core platform technology in BioNTech’s pipeline. BioNTech is investing in multiple platform technologies with the aim to lead in the field. BioNTech’s engineered cell therapy portfolio features both chimeric antigen receptor (CAR) and T cell receptor (TCRs) or individualized T cell receptor therapeutic drug candidates. BNT211 is BioNTech’s most advanced cell therapy development program. BNT211 is an autologous Claudin-6 (CLDN6)-targeting CAR-T cell therapy candidate that is being tested alone and in combination with a CAR-T cell Amplifying RNA Vaccine (“CARVac”), encoding CLDN6. The CAR-T cells are equipped with a second-generation CAR of high sensitivity and specificity. CARVac is intended to support in vivo expansion of CAR-T cells to increase their persistence and efficacy. CLDN6 is expressed on multiple solid tumors such as ovarian cancer, sarcoma, testicular cancer, endometrial cancer and gastric cancer. BioNTech plans to initiate its first pivotal Phase 2 trial evaluating BNT211 in 2L+ germ cell tumors in 2024 and is continuing to assess additional indications for further development. About BioNTech BioNTech is a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases. BioNTech exploits a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules. Based on its deep expertise in mRNA vaccine development and in-house manufacturing capabilities, BioNTech and its collaborators are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. BioNTech has established a broad set of relationships with multiple global pharmaceutical collaborators, including Duality Biologics, Fosun Pharma, Genentech, a member of the Roche Group, Genevant, Genmab, OncoC4, Regeneron and Pfizer. For more information, please visit . BioNTech Forward-Looking Statements This press release contains “forward-looking statements” of BioNTech within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited to, statements concerning: the collaboration between BioNTech and Autolus to advance both companies’ autologous CAR-T programs towards commercialization, pending regulatory authorizations, including BNT211 in CLDN6+ tumors, obe-cel in adult acute lymphoblastic leukemia, and AUTO1/22 and AUTO6NG in any oncology indication; the expected impact of the collaboration on BioNTech’s business, including any potential benefits to BioNTech and Autolus resulting from the collaboration; BioNTech’s access to or option to access Autolus’ target binders and cell programming technologies to support development of in vivo cell therapy and ADC candidates; BioNTech’s co-commercialization options for Autolus’ AUTO1/22 and AUTO6NG programs; BioNTech’s option to enter into a future agreement to access Autolus’ commercial and clinical site network, manufacturing capacities and commercial supply infrastructure; BioNTech’s plans regarding the timing, characterization and number of potentially registrational trials, including BNT211 in relapsed or refractory germ cell tumors; BioNTech’s agreement to make an equity investment in Autolus, including BioNTech’s director appointment right; the parties’ ability to receive certain milestone, royalty, revenue sharing, and/or profit-sharing payments; the planned next steps in BioNTech’s pipeline programs, including, but not limited to, statements regarding timing or plans for initiation or enrollment of clinical trials, or submission for and receipt of product approvals with respect to BioNTech’s product candidates; the ability of BioNTech’s mRNA technology to demonstrate clinical efficacy outside of BioNTech’s infectious disease platform; the potential safety and efficacy of BioNTech’s product candidates; and BioNTech’s anticipated market opportunity and size for its product candidates. Any forward-looking statements in this press release are based on BioNTech’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond BioNTech’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the proposed transactions may not close, in whole or in part; the compliance of the proposed transactions with applicable securities laws with respect to the purchase and sale of Autolus securities, including the availability of exemptions from registration and/or the future registration of purchased securities; the reaction of third parties, including competitors, to the transactions, including BioNTech’s planned equity investment in Autolus; each party’s ability to protect and maintain its intellectual property position; Autolus’ ability to maintain its manufacturing and supply infrastructure; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, and including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing data; the nature of the clinical data, which is subject to ongoing peer review, regulatory review and market interpretation; the timing of and BioNTech’s ability to obtain and maintain regulatory approval for its product candidates; BioNTech’s and its counterparties’ ability to manage and source necessary energy resources, capital requirements, the use of capital and unexpected expenditures; BioNTech’s ability to identify research opportunities and discover and develop investigational medicines; the ability and willingness of BioNTech’s third-party collaborators to continue research and development activities relating to BioNTech’s development candidates and investigational medicines; unforeseen safety issues and potential claims that are alleged to arise from the use of products and product candidates developed or manufactured by BioNTech; BioNTech’s and its collaborators’ ability to commercialize and market, if approved, its product candidates; BioNTech’s ability to manage its development and expansion; regulatory developments in the United States and other countries; BioNTech’s ability to effectively scale its production capabilities and manufacture its products and product candidates; risks relating to the global financial system and markets; BioNTech’s ability to create long-term value for its shareholders; and other factors not known to BioNTech at this time. You should review the risks and uncertainties described under the heading “Risk Factors” in BioNTech’s Report on Form 6-K for the period ended September 30, 2023, and in subsequent filings made by BioNTech with the SEC, which are available on the SEC’s website at Except as required by law, BioNTech disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on BioNTech’s current expectations and speak only as of the date hereof. About Autolus Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer and autoimmune disease. Using a broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize target cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies, solid tumors and autoimmune diseases. For more information, please visit Autolus Forward-Looking Statements This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus’ development of its product candidates, including the obe-cel program; the pro potential application of obe-cel in additional disease settings; the future clinical development, efficacy, safety and therapeutic potential of Autolus' product candidates, including progress, expectations as to the reporting of data, conduct and timing and potential future clinical and preclinical activity and milestones; expectations regarding the initiation, design and reporting of data from clinical trials and preclinical studies; expectations regarding the regulatory approval process for any product candidates; the benefits of the collaboration between Autolus and BioNTech, including the potential and timing to receive equity investments, milestone payments, profit share payments, and/or royalties under the terms of the strategic collaboration; Autolus’ current and future manufacturing capabilities; and the completion and timing of the proposed private placement. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, the risks that Autolus’ preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results; the cost, timing and results of clinical trials; that many product candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; and possible safety and efficacy concerns. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Autolus’ actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission, or the SEC, on March 7, 2023 and in Autolus' Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Autolus' subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Autolus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law. You should, therefore, not rely on these forward-looking statements as representing Autolus’ views as of any date subsequent to the date of this press release. CONTACTS BioNTech Investor Relations Victoria Meissner, M.D. +1 617 528 8293 Investors@biontech.de Media Relations Jasmina Alatovic +49 (0)6131 9084 1513 Media@biontech.de Autolus Olivia Manser +44 (0) 7780 471 o.manser@autolus.com Julia Wilson +44 (0) 7818 430877 j.wilson@autolus.com Susan A. Noonan S.A. Noonan Communications +1-917-513-5303 susan@sanoonan.com

免疫疗法引进/卖出细胞疗法疫苗

100 项与 CD19/22 CAR T-cells(University College, London) 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
急性淋巴细胞白血病	临床1期	英国	University College London	2016-04-01
伯基特淋巴瘤	临床1期	英国	University College London	2016-04-01
CD19 表达恶性肿瘤	临床1期	英国	University College London	2016-04-01

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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


NCT02443831 (CARPALL, EBMT2023) 人工标引	临床1期	复发难治性急性淋巴细胞白血病三线	12	AUTO1/22	鏇艱遞積鏇鑰膚鬱願遞(鹹觸積顧衊淵觸齋觸鹹) = 構壓簾範壓積鏇構積築築鑰窪構鑰淵壓齋獵膚 (獵獵觸鹹遞糧廠衊餘艱 ) 更多	积极	2023-04-26
NCT02443831 (CARPALL, EBMT2023) 人工标引	临床1期	复发难治性急性淋巴细胞白血病三线	12	AUTO1/22 (responding patients)	鏇遞憲鏇鹹齋夢廠製網(壓範鏇壓築窪壓壓製鹹) = 築膚構範顧膚網窪簾鑰遞壓鑰鏇膚範觸餘齋製 (窪遞選鬱鏇簾蓋獵襯築 )	积极	2023-04-26
NCT02443831 (ASH2022) 人工标引	临床1期	复发难治性急性淋巴细胞白血病	12	AUTO1/22	顧憲鹽壓艱積遞淵獵製(繭積憲範衊醖衊築觸鑰) = 襯鬱醖膚襯鑰獵淵齋範襯構艱蓋蓋壓鹽願鏇鏇 (餘廠衊繭窪鹽膚觸範觸 ) 更多	积极	2022-11-15
NCT02443831 (CARPALL, EHA2022) 人工标引	临床1期	复发难治性急性淋巴细胞白血病三线	8	AUTO1/22	鑰鹹廠餘構築鏇衊製選(觸構廠夢衊糧鏇簾窪鹹) = 顧襯觸壓築選鹹構範鏇醖顧鬱構壓鏇積製衊繭 (襯鏇積鬱壓繭衊選繭憲 ) 更多	积极	2022-05-12