A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in Pediatric and Young Adult (<21year) Patients With High-Risk and Very High-Risk Myeloid Malignancies

Cord blood (CB) transplants are an option for patients lacking an HLA identical donor but are hampered by low cell dose, prolonged aplasia and high transplant related mortality. UM171, a novel and potent agonist of hematopoietic stem cell self renewal could solve this major limitation, allowing for CB's important qualities as lower risk of chronic GVHD and relapse to prevail. In previous trials (NCT02668315, NCT03913026, NCT04103879, and NCT03441958), the CB expansion protocol using the ECT-001-CB technology (UM171 molecule) has proven to be technically feasible and safe in adults.
UM171 expanded CB was associated with a prompt (D+17), robust (98%) and durable neutrophil recovery. Amongst patients who received a single UM171 CB transplant with a median follow-up of 18 months, risk of TRM (10%), grade 3-4 acute GVHD (13%) and moderate-severe chronic GVHD (2%) was low at 1 year post-transplant. Incidence of severe viral and bacterial infections was reduced and immunosuppression could be discontinued in 77% of patients at 1 year. Thus, PFS and GRFS were very promising, 72% and 59% at 12 months, 69% and 53% at 24 months, respectively, in particular accounting for a large proportion of very high-risk patients. By a 10-fold increase of CB accessibility, ECT-001-CB allowed access to smaller, better HLA matched CBs.
This new study seeks to test a similar strategy in a group of pediatric and young adult patients with high risk myeloid malignancies. 12 patients will be enrolled in the first stage of this 2-stage design protocol. If intervention is considered promising (<= 3 relapses in the first 12 patients), this study will open multicenter and be extended to a second stage (16 additional patients for a total accrual 28).

开始日期2021-12-01

申办/合作机构

ExCellThera, Inc.初创企业

[+1]

NCT04594031

/ Withdrawn临床1期

A Phase I Multicenter Study of Hematopoietic Stem Cell Transplant of ECT-001-Expanded Cord Blood With a Reduced Toxicity Conditioning Regimen in Patients With Severe Sickle Cell Disease

The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment, particularly in pediatric populations. Clinical studies in adults with SCD have focused on interventions that prolong survival and improve the quality of life. Unlike children, adults with SCD are much more likely to have a debilitating complication. As a result, the risk/benefit ratio of HCT is very favorable in adults, particularly if an approach to HCT that defines an acceptable level of toxicity can be established.
Whereas hematopoietic stem cell transplantation (HSCT) remains the only curative treatment currently available for patients with SCD, the morbidity, the frequent irreversible damage in target organs and the mortality reported in the natural course of patients with severe SCD are strong incentives to perform HSCTs in younger age groups. For those who lack a matched related donor, CB transplant is an appealing option, but despite been less problematic, CB accessibility related to cell dose of appropriately matched cord blood unit (CBU) remains a significant issue. Through a 7-day culture process of a CBU's hematopoietic stem cell HSCs with the UM171 compound, the total cell dose is increased mitigating this limitation.
UM171-CB expansion (ECT-001-CB) allows a greater CB accessibility, the selection of better matched cords that might translate into favourable clinical outcomes as reported in previous trials, including a lower risk of graft-versus-host disease. After CB selection and ex-vivo expansion, ECT-001-CB transplant will follow a myeloablative reduced-toxicity conditioning regimen consisting of rATG, busulfan and fludarabine with doses of all agents optimized to the individual using model-based dosing and will be followed by standard supportive care and GVHD prophylaxis consisting of tacrolimus and MMF.

开始日期2021-07-13

申办/合作机构

ExCellThera, Inc.初创企业

[+1]

NCT04103879

/ Active, not recruiting临床2期

A Phase II Open-Label Study of UM171-Expanded Cord Blood Transplantation in Patients With High and Very High Risk Acute Leukemia/Myelodysplasia

Cord blood (CB) transplants are an option for patients lacking an HLA identical donor but are hampered by low cell dose, prolonged aplasia and high transplant related mortality. UM171, a novel and potent agonist of hematopoietic stem cell self renewal could solve this major limitation, allowing for CB's important qualities as lower risk of chronic GVHD and relapse to prevail. In a previous trial (NCT02668315), the CB expansion protocol using the ECT-001-CB technology (UM171 molecule) has proven to be technically feasible and safe. UM171 expanded CB was associated with a median neutrophil recovery at day (D)+18 post transplant. Amongst 22 patients who received a single UM171 CB transplant with a median follow-up of 18 months, risk of TRM (5%) and grade 3-4 acute GVHD (10%) were low. There was no moderate-severe chronic GVHD. Thus, overall and progression free survival at 12 months were impressive at 90% and 74%, respectively. The UM171 expansion protocol allowed access to smaller, better HLA matched CBs as >80% of patients received a 6-7/8 HLA matched CB. Interestingly there were patients with high-risk hematologic malignancies and multiple comorbidities (5 patients who had already failed an allogeneic transplant and 5 patients with refractory/relapsed acute leukemia/aggressive lymphoma). Despite this high risk population, progression was 20% at 12 months.
This new study seeks to test a similar strategy in a group of patients with high risk acute leukemia/myelodysplasia.

开始日期2020-11-13

申办/合作机构

ExCellThera, Inc.初创企业

[+1]

100 项与 Stem cell therapeutics (ExCellThera) 相关的临床结果

登录后查看更多信息

100 项与 Stem cell therapeutics (ExCellThera) 相关的转化医学

登录后查看更多信息

100 项与 Stem cell therapeutics (ExCellThera) 相关的专利（医药）

登录后查看更多信息

项与 Stem cell therapeutics (ExCellThera) 相关的文献（医药）

2025-02-01·Blood Cells, Molecules, and Diseases

Further biological characterization of small molecules UM171 and SR1: In vitro effects on three hematopoietic cell populations from human cord blood

Article

作者: Flores-Guzman, Patricia ; Torres-Caballero, Aranxa ; Mayani, Hector

Small molecules UM171 and SR1 have already been taken into clinically-oriented protocols for the ex vivo expansion of hematopoietic stem (HSCs) and progenitor (HPCs) cells. In order to gain further insight into their biology, in the present study we have assessed their effects, both individually and in combination, on the in vitro long-term proliferation and expansion of HSCs and HPCs contained within three different cord blood-derived cell populations: MNCs (CD34⁺ cells = 0.8 %), LIN^- cells (CD34⁺ cells = 41 %), and CD34⁺ cells (CD34⁺ cells >98 %). Our results show that when added to cultures in the absence of recombinant stimulatory cytokines, neither molecule had any effect. In contrast, when added in the presence of hematopoietic cytokines, UM171 and SR1 had significant stimulatory effects on cell proliferation and expansion in cultures of LIN^- and CD34⁺ cells. No significant effects were observed in cultures of MNCs. The effects of both molecules were more pronounced in cultures with the highest proportion of CD34⁺ cells, and the greatest effects were observed when both molecules were added in combination. In the absence of small molecules, cell numbers reached a peak by days 25-30, and then declined; whereas in the presence of UM171 or/and SR1 cell numbers were sustained up to day 45 of culture. Our results indicate that besides CD34⁺ cells, LIN^- cells could also be used as input cells in clinically-oriented expansion protocols, and that using both molecules simultaneously would be a better approach than using only one of them.

2025-01-01·Transplantation and Cellular Therapy

A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients

Article

作者: Veilleux, Olivier ; Bernard, Léa ; Delisle, Jean-Sébastien ; Lachance, Silvy ; Terra, Rafik ; Bambace, Nadia ; Cohen, Sandra ; LeBlanc, Richard ; Ahmad, Imran ; Kiss, Thomas ; Sauvageau, Guy ; Fournier, Valentyn ; Thauvette, Gabrielle ; Roy, Jean ; Thiant, Stéphanie ; Roy, Denis Claude ; Caudrelier, Pierre

Multiple myeloma (MM) remains associated with a poor outcome, particularly in patients with advanced disease and high-risk (HR) cytogenetics. To date, the only curative treatment is allogeneic (allo) hematopoietic cell transplantation (HCT), but high incidences of graft versus host disease (GVHD), nonrelapse mortality (NRM) and disease progression remain important obstacles. Cord blood (CB) transplantation has been associated with low rates of relapse and chronic (c) GVHD, but its use has declined because of high incidences of infections, severe acute GVHD and high NRM. In other hematologic malignancies, UM171-expanded CB transplants have led to improved outcomes, allowing for the selection of smaller, better HLA-matched units. We aimed to investigate the safety and feasibility of single UM171-expanded single CB unit transplantation in frontline tandem auto/allo HCT for HR/ultra-HR MM patients. Newly diagnosed MM patients ≤ 65 years with an ISS stage II/III and del(17p), t(4;14), t(14;16), t(14;20), del(1p) or +1q, R-ISS 3, ≥ 2 cytogenetic abnormalities, or plasma cell leukemia without a sibling donor and availability of a 5-7/8 matched CB graft with ≥ 0.5 x 10⁵ CD34+/kg and ≥ 1.5 x 10⁷ TNCs/kg were eligible to this phase I/II prospective study (ClinicalTrials.gov NCT03441958). After induction and autologous HCT, patients received a reduced intensity conditioning regimen and were infused with 7-day UM171-expanded CD34+ cells, along with the lymphocytes contained in the CD34-negative fraction. The primary endpoints were feasibility of UM171 expansion, safety, kinetics of engraftment, incidences and maximum grades of acute and cGVHD at 1 and 2 years, assessment of measurable residual disease (MRD) and quality of life (QoL). Between 05/2018 and 11/2021, 20 patients were enrolled. One patient had an unsuccessful CB expansion with UM171, leaving 19 patients with a median age of 56 years. Median CD34+ cell dose infused after expansion was 4.62 x 10⁶/kg (range: 0.79 to 5.76). Median times to achieve absolute neutrophil counts of 0.1 and 0.5 x 10⁹/L were D+6 and D+10.5; median time to reach ≥ 20 x 10⁹/L platelets was D+36. Full donor chimerism was achieved in all cell lineages by D+120 in recipients of reduced intensity conditioning. Cumulative incidences of grade II-IV, grade III-IV acute GVHD and moderate/severe cGVHD at 12 months were 68.4% (95% CI: 46 to 90), 5.3% (95% CI: 0% to 16%), and 10.5% (95% CI: 0% to 25%), respectively. With a median follow-up of 2.9 years (range: 0.46 to 5.3), cumulative incidences of relapse, PFS, OS and NRM at 3 years were 36.8% (95% CI: 14 to 59), 47.4% (95% CI: 29 to 76), 68.4% (95% CI: 50 to 93) and 15.8% (95%CI: 0 to 33), respectively. Median time to complete immunosuppression discontinuation was D+238. No unexpected adverse events were observed. Only one of 7 patients alive at 2 years with negative MRD at transplant has relapsed. Non-relapsing patients had a QoL after transplant similar to the general population. UM171-expanded CB transplant in HR/ultra-HR myeloma patients is feasible and allows the use of single CB units with a low risk of cGVHD. Patients with negative pretransplant MRD might benefit most from a UM171-expanded CB transplant.

2024-12-01·Regenerative Therapy

Synergistic effect and molecular mechanism of nicotinamide and UM171 in ex vivo expansion of long-term hematopoietic stem cells

Article

作者: Cui, Yanni ; Ren, Fanggang ; Ren, Yan ; Wang, Hongwei ; Zhang, Yaofang

IntroductionSeveral approaches to expand human hematopoietic stem cells (HSCs) have been reported, but the ability of these methods to expand long-term hematopoietic stem cells (LT-HSCs) remains to be improved, which limits the application of HSCs-based therapies.MethodsCD34+ cells were purified from umbilical cord blood using MacsCD34 beads, and then cultured for 12 d in a serum-free medium. Flow cytometry was used to detect phenotype, cell cycle distribution, and apoptosis of the cultured cells. Colony-forming cell (CFC) assays can evaluate multi-lineage differentiation potential of HSCs. Real-time polymerase chain reaction was employed to detect the expression of genes related to self-renewal programs and antioxidant activity. DCFH-DA probes were used to evaluate intracellular production of reactive oxygen species (ROS). Determination of the effect of different culture conditions on the balance of cytokine by cytometric bead array.ResultsHere, we show a combination, Nicotinamide (NAM) combined with pyrimidoindole derivative UM171, can massively expand LT-HSCs ex vivo, and the expanded cells maintained the capability of self-renewal and multilineage differentiation. Additionally, our data indicated that UM171 promoted self-renewal of HSCs by inducing HSCs entry into the cell cycle and activating Notch and Wnt pathways, but the infinite occurrence of this process may lead to mitochondrial metabolism disorder and differentiation of HSCs. NAM kept HSCs in their primitive and dormant states by reducing intracellular ROS levels and upregulating the expression of stemness related genes, so we believed that NAM can act as a brake to control the above process.ConclusionsThe discovery of the synergistic effect of NAM and UM171 for expanding LT-HSCs provides a new strategy in solving the clinical issue of limited numbers of HSCs.

项与 Stem cell therapeutics (ExCellThera) 相关的新闻（医药）

2024-12-23

·药明康德

突破实体瘤难关，明年更多疗法有望获批｜细胞和基因疗法年度盘点（附下载）

▎药明康德内容团队编辑细胞和基因疗法在过去10年里长足发展，在即将过去的2024年再次迎来重要里程碑——两类细胞疗法纷纷突破实体瘤难关；同时，从获批数量来看，2024年不仅保持了过去3年中的稳健趋势，即将到来的2025年还有望开启“加速”模式，超过10款疗法可能获批。今天的这篇文章中，药明康德内容团队将和各位读者朋友分享今年细胞和基因疗法领域新药获批和研发的一些趋势，并展望未来有望获批的多款疗法。扫描文中二维码或点击文末“阅读原文”可申请获得完整报告，包括已获批疗法及2025年即将获批疗法盘点、大型药企管线列表、融资新锐介绍等详尽信息。 ▲长按扫描二维码或点击文末“阅读原文/Read more”，即可填写问卷，申请获取细胞和基因疗法研发进展完整报告获批速度稳健 2024年全球共7款细胞和基因疗法首次获批上市，其中2款为干细胞疗法。除外干细胞疗法后的细胞和基因疗法获批速度依然保持了近年来这一领域的积极趋势。 ▲历年全球范围内首次批准的细胞和基因疗法数量（非干细胞类）（药明康德内容团队制图，数据截至2024年12月20日） 2024年全球首批的1款基因疗法用于治疗罕见病，4款免疫细胞疗法均治疗癌症，尤为值得关注的里程碑突破是首款肿瘤浸润淋巴细胞（TIL）疗法，以及首款T细胞受体（TCR）-T细胞疗法问世，T细胞疗法和工程化T细胞疗法均实现治疗实体瘤的突破。 ▲2024年全球首次获批的细胞和基因疗法（药明康德内容团队制图，数据截至2024年12月20日）大型药企临床管线趋势全球20家大型药企的管线布局中，目前共29款细胞疗法和16款基因疗法处于临床开发阶段。 ▲全球20家大型药企的细胞疗法（左）和基因疗法（右）管线分布；扫描文中二维码可申请获取完整管线列表（药明康德内容团队制图；数据来源为各药企公开信息，截至2024年12月；20家大型药企参考行业媒体Fierce Pharma 2024年榜单）尽管细胞疗法仍以嵌合抗原受体（CAR）T细胞疗法为主，但近1/3采取双靶点策略，同时在热门靶点CD19/CD20/BCMA之外，早期管线更多针对新兴靶点如GPRC5D、GPC3、CLDN18.2、STEAP2，后3个靶点也是攻克实体瘤的策略。早期管线中也有更多新型细胞疗法，如阿斯利康正在开发3款TCR-T疗法以治疗实体瘤；武田正在开发一款γδ T细胞疗法，治疗急性髓系白血病。基因疗法的应用领域正在从罕见病拓展至更多相对常见的疾病，多款已进入临床中后期研发阶段的疗法有望为帕金森病、痴呆等中枢神经系统（CNS）疾病，以及常见眼病、心力衰竭、遗传性耳聋等疾病带来全新希望。早期融资回暖，细胞疗法新锐融资总额超越2023 2024年细胞和基因疗法领域共发生50起早期融资事件，整体呈现回暖趋势。 2024年共28家聚焦细胞疗法的新锐获得共约12.1亿美元的早期融资，超越2023年（11.54亿美元），且下半年呈现逐步回暖趋势。 ▲近3年细胞疗法研发新锐早期投融资趋势（药明康德内容团队制图，数据截至2024年12月20日）单笔超过1亿美元的大额融资聚焦下一代CAR-T细胞疗法，如体内CAR-T、可控CAR-T、针对实体瘤的CAR-T等。其他受关注的技术方向包括基于TCR-T、调节性T细胞（Treg）、树突状细胞、巨噬细胞、胸腺干细胞、诱导多能干细胞（iPSC）等更广泛细胞类型治疗实体瘤、自身免疫和炎症性疾病、神经系统疾病等更多种疾病。 2024年共23家聚焦基因疗法或基因编辑的新锐获得共约8.29亿美元的早期融资，从各季度数据来看也正在回暖。 ▲近3年基因疗法研发新锐早期投融资趋势（药明康德内容团队制图，数据截至2024年12月20日）这些融资新锐的技术创新包括：设计下一代载体如新型腺相关病毒（AAV）衣壳、免疫屏蔽慢病毒（ISLV）载体，开发下一代编辑工具以插入大段DNA、消除脱靶突变等。基因编辑领域先驱张锋教授联合创建Moonwalk Biosciences，采取靶向表观遗传组的新颖编辑策略。罕见病之外，不少基因疗法新锐剑指眼科疾病、肾病、神经退行性病变等更常见疾病。注：早期融资定义为B轮及之前的融资轮次，且融资金额超过1000万美元。如果融资事件的货币单位不是美元，则依据当时汇率进行换算。未符合以上条件的公司（如获得融资，但未公布金额/轮次）未被纳入。 2025年超10款细胞和基因疗法有望获批基于当前公开研发与监管审评进展信息，2025年全球有11款在研细胞和基因疗法有望迎来首次批准，获批数量有望创下新高。其中不乏技术创新，例如基因校正表皮片，以及基于光遗传学开发、治疗时无需考虑基因突变类型的基因疗法。从监管风向来看，美国FDA支持采用替代终点来审评生物制品许可申请（BLA）的加速批准，以加快疗法上市。基因疗法prademagene zamikeracel（pz-cel）：治疗隐性营养不良大疱性表皮松解症（RDEB） Pz-cel是一种自体、基因校正表皮片，旨在将功能性、产生胶原蛋白VII的COL7A1转基因递送到患者自身的皮肤细胞中，稳定整合到分裂的靶细胞DNA中，在伤口部位实现长期基因表达。基于多项临床研究长达8年随访的疗效和安全性数据，Abeona Therapeutics更新递交了pz-cel的BLA，PDUFA日期为2025年4月29日，此前该疗法也已获得优先审评资格。基因疗法RP-L102：治疗范可尼贫血 RP-L102通过LV对患者的自体造血干细胞进行基因工程化改造，递送FANCA基因的功能性拷贝，以预防范可尼贫血相关骨髓衰竭（BMF）。全球1/2期关键研究中，RP-L102显示出持续的基因校正、全面的表型校正和血液学稳定性。EMA已于2024年4月接受RP-L102的上市许可申请（MAA），2024年11月，Rocket Pharmaceuticals宣布也已启动向美国FDA滚动递交BLA。干细胞疗法dorocubicel（UM171）：用于造血干细胞移植 UM171由诱导扩增产生的造血干细胞（HSCs）组成。UM171可通过降解组蛋白去乙酰化酶1/2和赖氨酸特异性去甲基化酶1来防止细胞培养过程中出现细胞分化，从而增强造血干细胞的更新。2024年6月，该疗法的MAA已被EMA接受并予以快速审评资格。基因疗法rebisufligene etisparvovec（UX111）：治疗IIIA型Sanfilippo综合征（MPS IIIA） UX111使用AAV9载体递送编码N-磺基葡萄糖胺磺基水解酶的SGSH基因到中枢神经系统和外周器官。SGSH酶可分解并回收硫酸乙酰肝素（HS），HS水平与长期认知发育相关。1/2期临床试验显示，UX111能迅速和显著降低患者脑脊液中的HS水平。2024年6月，Ultragenyx Pharmaceutical与美国FDA达成一致HS可作为替代终点支持BLA，该公司已于2024年12月递交BLA。根据新闻稿，如果获得批准，UX111将成为获美国FDA批准用以治疗MPS IIIA的首款疗法。基因疗法RGX-121：治疗黏多糖贮积症II型（MPS II） RGX-121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。临床试验显示，接受关键剂量RGX-121治疗的患者，关键生物标志物——脑脊液中HS D2S6水平降低85%且持续长达两年，80%患者无需静脉输注酶替代疗法。2024年11月，REGENXBIO宣布已启动向美国FDA滚动递交RGX-121的BLA，预计2025年第一季度完成，其新闻稿指出，RGX-121有潜力成为在美国获批的首个MPS II基因疗法。干细胞疗法deramiocel（CAP-1002）：治疗杜氏肌营养不良（DMD） CAP-1002由同种异体、心脏来源细胞（CDCs）组成，通过释放包含微RNA、非编码RNA和蛋白的外泌体，改善DMD患者肌肉瘢痕或纤维化以及心脏功能。HOPE-2研究显示，DMD患者心脏功能的多项指标均有所改善，心脏和骨骼肌功能在三年期间趋于稳定。2024年10月，Capricor Therapeutics宣布已启动向美国FDA滚动递交BLA。目前尚无获批的DMD心肌病疗法，而心肌病是DMD患者死亡的主要原因。此外，关键试验已获得积极结果、已明确计划或有望在近期递交上市申请的还有： AAV基因疗法sonpiretigene Isteparvovec（MCO-010），由Nanoscope Therapeutics开发，治疗视网膜色素变性； AAV基因疗法pariglasgene brecaparvovec（DTX401），由Ultragenyx Pharmaceutical开发，治疗Ia型糖原贮积病； AAV基因疗法giroctocogene fitelparvovec，由辉瑞和Sangamo Therapeutics共同开发，治疗血友病A； LV基因疗法marnetegragene autotemcel，由Rocket Pharmaceuticals开发，治疗严重I型白细胞粘附缺陷；靶向CLDN18.2的CAR-T细胞疗法舒瑞基奥仑赛注射液（CT041），由科济药业开发，治疗胃癌。 ▲长按扫描二维码或点击文末“阅读原文/Read more”，即可填写问卷，申请获取细胞和基因疗法研发进展完整报告 ▲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

细胞疗法基因疗法免疫疗法上市批准

2024-06-26

·GlobeNewswire-Health Care

ExCellThera Announces EMA’s Acceptance under Accelerated Assessment of Market Authorisation Application (MAA) for UM171 Cell Therapy for Patients with Hematological Malignancies who Lack a Readily Available Suitable Donor

- Market Authorisation Application (MAA) for UM171 Cell Therapy accepted under an accelerated assessment procedure by the European Medicines Agency (EMA), for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor June 25, 2024 -- ExCellThera Inc. (ExCellThera), a world leader in enhanced blood stem cell expansion and therapies, announced today that the Market Authorisation Application (MAA) for UM171 Cell Therapy (INN-dorocubicel) has been accepted under an accelerated assessment procedure by the European Medicines Agency (EMA), for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor. The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted UM171 Cell Therapy accelerated assessment as it was deemed of major interest from a public health point of view. Accelerated assessment aims to reduce the timeframe for the CHMP to review a MAA compared to the standard procedure and follows the EMA granting Orphan Medicinal Product designation and access to the Priority Medicines scheme (PRIME) in 2020. “We are excited that the EMA has accepted this regulatory submission under an accelerated assessment procedure, as UM171 Cell Therapy has immense potential to help address a high unmet medical need for patients with hematological malignancies who require a stem cell transplant but lack a readily available suitable donor,” said David Millette, CEO of ExCellThera. “We now look forward to working closely with the EMA in the coming months to bring this therapy to the patients who need it.” UM171 Cell Therapy (INN-dorocubicel), developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients to treat hematologic malignancies in clinical trials in the United States, Europe and Canada. UM171 Cell Therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA. UM171 Cell Therapy has successfully completed Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with refractory or active disease, patients requiring a second transplant, and patients with TP53 mutations or other genetic abnormalities. The initiation of a Phase 3 trial in this patient population is planned for the second half of 2024. The use of UM171 Cell Therapy in other patient populations, including patients with multiple myeloma, pediatric patients, and patients with non-malignant hematological diseases, is also being explored. UM171 Cell Therapy is an investigational cell therapy, and its safety and efficacy have not been established by the FDA, EMA, Health Canada or any other health authority. ExCellThera is a world leader in enhanced blood stem cell expansion. ExCellThera’s proprietary EnhanceTM platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding HSCs from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the EnhanceTM platform, including the proprietary molecule UM171 which has a first- and best-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs. For additional information, visit excellthera.com, and follow us on LinkedIn. The content above comes from the network. if any infringement, please contact us to modify.

细胞疗法孤儿药加速审批

2024-01-02

·BioSpace

ExCellThera to Conduct Investor Meetings During the J.P. Morgan 42nd Annual Healthcare Conference and to Participate in Upcoming Investor Conferences

MONTREAL, Jan. 02, 2024 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell therapies, announced today that its senior leaders will be holding investor meetings during the 42nd Annual J.P. Morgan Healthcare Conference, January 8-11, 2024, in San Francisco, CA, and will be participating in the following investor conferences in January: 2024 Biotech Showcase (Presentation) Presentation Date and Time: January 8, 2024 at 4:45 PM PT Speaker: David Millette, Chief Executive Officer Location: San Francisco, CA, Ballroom Franciscan D, Hilton Union Square ExCellThera will also hold one-on-one investor meetings during the 42nd Annual J.P. Morgan Healthcare Conference, January 8-11, 2024, in San Francisco, CA. To request a one-on-one meeting with ExCellThera, please contact sellinwood@kendallir.com. B Riley 4th Annual Oncology Conference (Fireside Chat) Presentation Date and Time: January 18, 2024 at 10:00 AM ET Speakers: Guy Sauvageau, Founder and Chief Scientific Officer and David Millette, Chief Executive Officer Location: Virtual About UM171 Cell Therapy UM171 Cell Therapy has been evaluated in over 100 patients to treat hematologic malignancies in clinical trials in the United States, Europe and Canada. UM171 Cell Therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designation from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA. High Unmet Medical Need UM171 Cell Therapy Patients with high and very high-risk acute leukemias and myelodysplasias, including patients with refractory or active disease, patients requiring a second transplant, and patients with TP53 mutations or other genetic abnormalities, have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care. UM171 Cell Therapy has successfully completed Phase 2 trials in high and very high-risk acute leukemias and myelodysplasias. A Phase 3 trial is planned in 2024 for UM171 Cell Therapy for the treatment of high and very high-risk acute leukemias and myelodysplasias. Patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor, including familial, unrelated or haploidentical donor, or cord blood of sufficient quantity. A Market Authorization Application (MAA) submission was completed and is under accelerated assessment with the European Medicines Agency (EMA) for the licensure of UM171 Cell Therapy for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor. The use of UM171 Cell Therapy in other patient populations, including pediatric patients and patients with non-malignant hematological diseases, is also being explored. UM171 Cell Therapy is an investigational cell therapy, and its safety and efficacy have not been established by the FDA, EMA, Health Canada or any other health authority. About ExCellThera and UM171 Technology ExCellThera is a world leader in enhanced blood stem cell therapies. ExCellThera’s proprietary EnhanceTM platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding HSCs from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the EnhanceTM platform, including the proprietary molecule UM171 which has a first-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs. For additional information, visit excellthera.com, and follow us on LinkedIn. Investor and Media Contact Sarah Ellinwood Kendall Investor Relations sellinwood@kendallir.com

细胞疗法孤儿药临床2期临床3期

100 项与 Stem cell therapeutics (ExCellThera) 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
血液肿瘤	申请上市	欧盟	ExCellThera, Inc.初创企业	2024-06-25
髓系肿瘤	临床2期	美国	ExCellThera, Inc.初创企业	2021-12-01
骨髓增生异常综合征	临床2期	加拿大	ExCellThera, Inc.初创企业	2019-04-01
难治性急性白血病	临床2期	加拿大	ExCellThera, Inc.初创企业	2019-04-01
浆细胞白血病	临床2期	加拿大	ExCellThera, Inc.初创企业	2018-03-07
造血干细胞移植	临床前	欧盟	ExCellThera, Inc.初创企业	2020-12-16
移植物抗宿主病	临床前	美国	ExCellThera, Inc.初创企业	2018-12-13
镰状细胞血症	药物发现	美国	ExCellThera, Inc.初创企业	2021-07-13

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04103879 \| NCT03913026 (GlobeNewswire) 人工标引	临床2期	白血病 \| 骨髓增生异常综合征	50	UM171	(構鏇鬱獵鑰鏇築鑰壓壓) = 選窪鬱簾齋壓廠鏇築築蓋願鬱鏇齋壓醖顧夢齋 (製願憲築壓廠蓋艱構築 ) 更多	积极	2023-12-12
ASGCT2023	N/A	镰状细胞血症 CD34+ \| CD38- \| CD45RA ... 更多	-	UM171	齋遞襯壓廠積齋膚衊鏇(壓構構夢鏇製製網衊繭) = 餘艱糧醖網顧遞願艱糧憲壓構壓觸鹹餘選襯構 (淵築積獵憲鏇繭選壓範 ) 更多	积极	2023-05-01
ASGCT2023	N/A	镰状细胞血症 CD34+ \| CD38- \| CD45RA ... 更多	-	UM171 (Control)	獵選鏇願遞繭顧遞廠蓋(蓋艱範築繭壓醖製鏇壓) = 鬱餘壓廠夢糧醖遞顧夢衊鑰膚鑰繭蓋鹹蓋餘繭 (簾壓餘膚憲廠製繭鹹艱 )	积极	2023-05-01
Tandem Meetings ASTCT and CIBMTR2023	N/A	骨髓肿瘤	-	UM171 (Expanded Cord Blood Grafts)	鏇構鹹鏇衊壓憲繭鹽觸(餘窪網膚淵範願鏇範簾) = 餘壓衊獵夢襯鏇艱廠蓋廠鬱繭餘獵醖齋鹹壓艱 (積製蓋餘餘糧襯簾憲鑰 ) 更多	-	2023-02-01
Tandem Meetings ASTCT and CIBMTR2020	临床1期	造血干细胞移植	22	UM171 (Myeloablative conditioning regimen + single CB transplant)	鑰顧範淵選鹹願蓋襯淵(築構鑰淵醖憲網鏇淵構) = 夢範築餘廠淵繭積糧築製糧艱構齋網鹹鏇觸淵 (簾憲鏇齋築衊壓壓憲鹽 ) 更多	积极	2020-03-01
ASGCT2019	N/A	-	-	UM171	夢鑰積艱構鏇蓋糧範選(網選壓繭艱淵艱壓簾構) = 遞簾簾衊淵積築繭艱淵範膚餘壓網餘蓋鏇繭襯 (製範廠顧鑰積願鏇膚網 )	-	2019-04-22
Tandem Meetings ASTCT and CIBMTR2018	N/A	-	17	UM171 expanded cord blood (eCB)	(鏇鏇鹹繭醖構簾願夢齋) = 廠淵餘鹽窪鑰衊鹽構積獵製憲膚膚蓋夢廠獵製 (鬱簾網淵網遞廠夢鬱鏇 ) 更多	积极	2018-03-01
Tandem Meetings ASTCT and CIBMTR2018	N/A	-	17	UM171 (Peripheral blood or marrow)	(鏇鏇鹹繭醖構簾願夢齋) = 齋餘鑰夢構選窪鹽願夢獵製憲膚膚蓋夢廠獵製 (鬱簾網淵網遞廠夢鬱鏇 )	积极	2018-03-01
ASGCT2014	N/A	-	-	UM171	繭築願積廠鹹憲艱襯憲(夢憲衊構顧構齋夢選艱) = 鏇簾齋鹹蓋廠願繭網淵積醖夢築夢夢觸窪範製 (糧築餘憲選襯衊選淵艱 ) 更多	-	2014-05-01
ASGCT2014	N/A	-	-	UM171 (SR1)	繭築願積廠鹹憲艱襯憲(夢憲衊構顧構齋夢選艱) = 繭繭積淵構淵網襯鹽淵積醖夢築夢夢觸窪範製 (糧築餘憲選襯衊選淵艱 ) 更多	-	2014-05-01