A Phase 1/2 Study of KSQ-001EX, Autologous Tumor Infiltrating Lymphocytes Engineered to Inactivate the SOCS1 Gene, in Patients With Select Advanced Solid Tumors

To learn if KSQ-001EX is safe to give to participants with advanced forms of solid tumors.

开始日期2024-01-31

申办/合作机构

The University of Texas MD Anderson Cancer Center

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100 项与 KSQ-001 相关的临床结果

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100 项与 KSQ-001 相关的转化医学

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100 项与 KSQ-001 相关的专利（医药）

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项与 KSQ-001 相关的文献（医药）

2023-12-15·The Journal of clinical investigation

Rational design of a SOCS1-edited tumor-infiltrating lymphocyte therapy using CRISPR/Cas9 screens

Article

作者: Keegan, Sean ; Schlabach, Michael R. ; Cadzow, Louise ; Colletti, Nicholas ; Bullock, Caroline P. ; Thompson, Frank ; Wong, Karrie ; Stegmeier, Frank ; Shenker, Sol ; Kryukov, Gregory V. ; Le Mercier, Isabelle ; Wrocklage, Christopher ; Gannon, Hugh S. ; Sofjan, Katri ; Collester, Zachary R. ; Merkin, Jason J. ; Xu, Tianlei ; Tubo, Noah J. ; Williams, Leila J. ; Lin, Sharon ; LaMothe, Robert A. ; Garrett, Tracy E. ; Calnan, Conor ; Dunbar, Paul R. ; Dugopolski, Caroline ; Hohmann, Anja F. ; Sharp, Fiona A. ; Benson, Micah J.

Cell therapies such as tumor-infiltrating lymphocyte (TIL) therapy have shown promise in the treatment of patients with refractory solid tumors, with improvement in response rates and durability of responses nevertheless sought. To identify targets capable of enhancing the antitumor activity of T cell therapies, large-scale in vitro and in vivo clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 screens were performed, with the SOCS1 gene identified as a top T cell-enhancing target. In murine CD8+ T cell-therapy models, SOCS1 served as a critical checkpoint in restraining the accumulation of central memory T cells in lymphoid organs as well as intermediate (Texint) and effector (Texeff) exhausted T cell subsets derived from progenitor exhausted T cells (Texprog) in tumors. A comprehensive CRISPR tiling screen of the SOCS1-coding region identified sgRNAs targeting the SH2 domain of SOCS1 as the most potent, with an sgRNA with minimal off-target cut sites used to manufacture KSQ-001, an engineered TIL therapy with SOCS1 inactivated by CRISPR/Cas9. KSQ-001 possessed increased responsiveness to cytokine signals and enhanced in vivo antitumor function in mouse models. These data demonstrate the use of CRISPR/Cas9 screens in the rational design of T cell therapies.

项与 KSQ-001 相关的新闻（医药）

2024-10-28

·PRNewswire

Treg Cell-based Therapies Clinical Trial Pipeline Appears Robust With 51+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

Treg Cell-based Therapies refer to therapeutic approaches that utilize regulatory T cells (Tregs) to modulate the immune system. Innovations in cell expansion and engineering techniques have greatly enhanced the ability to isolate, expand, and deliver functional Tregs. Ex vivo expansion technologies and genetic engineering (such as CAR-Tregs) have opened up new possibilities for enhancing the stability, specificity, and effectiveness of Treg therapies. LAS VEGAS, Oct. 28, 2024 /PRNewswire/ -- DelveInsight's ' Treg Cell-based Therapies Pipeline Insight 2024 ' report provides comprehensive global coverage of available, marketed, and pipeline Treg cell-based therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Treg cell-based therapies pipeline domain. Key Takeaways from the Treg Cell-based Therapies Pipeline Report DelveInsight's Treg cell-based therapies pipeline report depicts a robust space with 50+ active players working to develop 55+ pipeline therapies for various indications. Key Treg cell-based therapies companies such as Orca Bio, Nektar Therapeutics, Quell Therapeutics, Cugene, Sangamo Therapeutics, Abata Therapeutics, HCW Biologics, GentiBio, Tr1X, PolTREG, Cellenkos, VT BIO, Miltenyi Biotec GmbH, KSQ Therapeutics, TeraImmune, Tract Therapeutics, and others are evaluating new Treg cell-based therapies to improve the treatment landscape. Promising Treg cell-based therapies in the pipeline in various stages of development include Orca-T, NKTR-358, QEL-001, CUG-252, TX200, ABA-101, HCW9302, GNTI-122, TRX-103, PTG-020, CK-0804, VT-301, TR-004, KSQ 001, TI-620, TRK 001, and others. In October 2024, Quell Therapeutics hired eXmoor Pharma to make supplies of its pipeline of novel immunosuppressive cell therapies—called CAR-Tregs—at its new cell and gene therapy facility. Quell recently teamed with fellow U.K.-based eXmoor in a strategic partnership that will see the contractor make its candidate autoimmune disease cell therapies for early-phase clinical studies. In September 2024, Abata Therapeutics announced that ABA-101, an autologous regulatory T-cell (Treg) therapy for the treatment of patients with progressive multiple sclerosis (MS), has received fast-track designation from the US FDA. In September 2024, PolTREG S.A. announced that China's National Intellectual Property Administration, PRC (CNIPA) had issued a notification to grant a patent for the intrathecal administration of the company's cellular therapies in patients with multiple sclerosis (MS). The intrathecal administration is an established method used to deliver therapy across the blood-brain barrier via injection into the subarachnoid space. In June 2024, Orca Bio announced that it had completed enrollment in the pivotal Precision-T Phase III clinical study. The primary endpoint of the Precision-T study is the rate of survival free from moderate-to-severe chronic graft versus host disease (GvHD). In March 2024, PolTREG announced that the company is planning to launch Phase II trials testing its Tregs therapy — the T-cell-based treatment PTG-007 in people with multiple sclerosis (MS). In February 2024, Cellenkos announced encouraging safety data from its innovative CK0803 neurotrophic T regulatory (Treg) cell therapy, being developed to help treat individuals with Amyotrophic Lateral Sclerosis (ALS). The company was given the green light to move ahead following a review of safety data by the Data Safety Monitoring Board (DSMB). In August 2023, BlueRock Therapeutics LP, a wholly owned, independently operated subsidiary of Bayer AG, and bit.bio, the company coding human cells for novel cures announced a collaboration and option agreement for the discovery and manufacture of iPSC-derived regulatory T cells (Tregs) for use in creating therapeutics. Request a sample and discover the recent advances in Treg cell-based therapies @ Treg Cell-based Therapies Pipeline Outlook The Treg cell-based therapies pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Treg cell-based therapies, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Treg cell-based therapies pipeline landscape. Treg Cell-based Therapies Overview Treg cell-based therapies represent a promising frontier in immunotherapy, focusing on harnessing regulatory T cells (Tregs) to modulate the immune response in various clinical settings. Tregs play a crucial role in maintaining immune homeostasis and preventing autoimmune reactions by suppressing excessive immune responses. In the context of autoimmune diseases, these therapies aim to restore balance by expanding or enhancing Tregs to dampen inappropriate immune activity. For instance, in diseases like type 1 diabetes or multiple sclerosis, Treg cell-based treatments seek to prevent or slow disease progression by reestablishing immune tolerance. Beyond autoimmune disorders, Treg cell-based therapies are also being explored in cancer treatment. Tumors often exploit Tregs to evade immune detection, and by manipulating these cells, it's possible to enhance the anti-tumor immune response. Strategies include expanding Tregs ex vivo and reinfusing them to boost their numbers or modifying them to more effectively target tumor-associated antigens. These approaches hold the potential to improve the efficacy of existing immunotherapies and offer new avenues for treating cancers that are currently resistant to conventional treatments. Find out more about Treg cell-based therapies @ Treg Cell-based Therapies in Development A snapshot of the Treg Cell-based Therapies Pipeline Drugs mentioned in the report: Learn more about the emerging Treg cell-based therapies @ Treg Cell-based Therapies Clinical Trials Treg Cell-based Therapies Therapeutics Assessment The Treg cell-based therapies pipeline report proffers an integral view of the emerging Treg cell-based therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Treg Cell-based Therapies Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intravenous, Subcutaneous, Oral, Intramuscular Therapeutics Assessment By Molecule Type: Monoclonal antibody, Small molecule, Peptide Therapeutics Assessment By Mechanism of Action: Immunologic cytotoxicity, T lymphocyte replacements, Interleukin 2 replacements, Cell replacements Key Treg Cell-based Therapies Companies: Orca Bio, Nektar Therapeutics, Quell Therapeutics, Cugene, Sangamo Therapeutics, Abata Therapeutics, HCW Biologics, GentiBio, Tr1X, PolTREG, Cellenkos, VT BIO, Miltenyi Biotec GmbH, KSQ Therapeutics, TeraImmune, Tract Therapeutics, and others. Key Treg Cell-based Therapies in Pipeline: Orca-T, NKTR-358, QEL-001, CUG-252, TX200, ABA-101, HCW9302, GNTI-122, TRX-103, PTG-020, CK-0804, VT-301, TR-004, KSQ 001, TI-620, TRK 001, and others. Dive deep into rich insights for Treg cell-based therapies assessment, visit @ Treg Cell-based Therapies Therapeutics Assessment Table of Contents For further information on the Treg cell-based therapies pipeline therapeutics, reach out @ Treg Cell-based Therapies Pipeline Landscape Related Reports Type 1 Diabetes Market Type 1 Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key type 1 diabetes companies, including Histogen , Novo Nordisk, Prevention Bio, among others. 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免疫疗法细胞疗法快速通道临床3期

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·药明康德

100%患者肿瘤细胞完全消失并维持1年的组合细胞疗法；使失聪儿童双耳听力均恢复的基因疗法…… | 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。BioMarin Pharmaceutical公司用于治疗严重血友病A的基因疗法Roctavian在一项3期临床试验中表现亮眼，73.6%的患者在接受治疗后的4年内出血次数为零。白血病患者在接受造血干细胞移植后接受一剂INB-100治疗，100%可评估的患者于今年5月31日时仍然存活，并且维持完全缓解（CR）达到1年。用于治疗遗传性耳聋的基因疗法AAV1-hOTOF使5名患儿的双侧耳朵都恢复了听力。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✦——— ◇ Ultragenyx Pharmaceutical公司宣布，该公司与美国FDA在会议中一致认为脑脊液（CSF）中的硫酸乙酰肝素（HS）是一个合理的替代终点，可用以支持其在研基因疗法UX111治疗IIIA型黏多糖贮积症（MPS IIIA）的生物制品许可申请（BLA）的加速批准。今年2月，Ultragenyx公布了UX111的1/2期临床试验Transpher A的更新数据，UX111能迅速和显著降低患者CSF中的HS水平。HS水平的持久降低与患者的长期认知发育具有相关性。该公司将在与FDA的BLA前会议中最终确认BLA的细节，并计划在今年晚些时候或明年年初提交申请。 MPS IIIA是一种常染色体隐性遗传病，由编码N-磺基葡萄糖胺磺基水解酶（SGSH）的基因发生突变所引起。SGSH酶可分解并回收HS。HS调控着一系列重要的生理过程，但是当编码SGSH的基因发生突变而导致酶失活时，HS会在器官中积聚并破坏器官的正常功能，特别是造成中枢神经系统的功能损伤，患者可能出现发育迟缓、行为障碍、癫痫发作等认知或生理异常状况。UX111旨在使用腺相关病毒9（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。 ◇ BioMarin Pharmaceutical公司公布了其基因疗法Roctavian（valoctocogene roxaparvovec）用以治疗严重血友病A患者的3期临床试验的长期安全性和有效性最新数据。分析显示，患者在接受Roctavian输注后4年，展现长期持续的出血控制和凝血因子VIII（FVIII）表达。在第4年，还在研究中的110例受试者中有73.6%（81例）的出血次数为零。 ▲GENEr8-1试验4年随访结果摘要（图片来源：参考资料[1]）血友病A是由于缺乏FVIII而导致的一种罕见遗传性出血性疾病，以反复出血及其相关并发症为主要临床表现。Roctavian是使用AAV5病毒载体递送表达FVIII的转基因。它的优势在于患者可能只需要接受一次治疗，肝细胞就可以持续表达FVIII，从而不再需要长期接受预防性凝血因子注射。 ◇ 近日，《自然-医学》杂志上刊登了由复旦大学附属眼耳鼻喉科医院和哈佛大学医学院的研究团队领衔的一项单臂研究的积极结果。该研究旨在评估一种在研基因疗法AAV1-hOTOF在遗传性耳聋儿童患者中的安全性和有效性。此前公布的试验结果显示，在6名患有常染色体隐性遗传性耳聋9（DFNB9）的儿童中，5名患儿在接受单侧耳的AAV1-hOTOF治疗后听力获得显著改善。基于这些积极数据，研究人员将方案进行了调整并获得了批准，允许对患者的双侧耳进行基因治疗。此次公布的数据显示，5名接受双耳AAV1-hOTOF治疗的患儿均恢复了双侧听力，比先前接受单耳治疗时获得了更多的益处。所有5名患者的言语感知和声源定位能力均得到了恢复，两名孩子获得了欣赏音乐的能力。新闻稿指出，该试验是全球首个对遗传性耳聋患儿的双耳进行基因疗法治疗的临床试验。 DFNB9患者由于OTOF基因的突变，导致表达的耳铁蛋白（otoferlin）功能失常，无法正常将声音刺激信号从耳朵传向大脑，从而导致耳聋。AAV1-hOTOF由复旦大学附属眼耳鼻喉科医院、哈佛大学医学院、和上海鼎新基因科技有限公司联合研发，使用AAV载体，将表达正常功能的耳铁蛋白的转基因递送到患者内耳毛细胞，从根源改善患者听力。由于OTOF转基因的大小超过单个AAV载体的载荷限制，研究团队采用了双载体递送技术，利用两个AAV载体递送OTOF转基因。 ◇ 星汉德生物宣布，其自主研发的乙型肝炎病毒（HBV）特异性T细胞受体（TCR）T细胞疗法SCG101入选了2024年度欧洲肝脏研究协会年会（EASL Congress 2024）的焦点精选报告。研究结果显示，SCG101在晚期HBV相关肝细胞癌患者中展现出抗病毒和抗肿瘤双重活性，显著延长了患者的生存期。至稿件截止日，患者的客观缓解率为33%，获得部分缓解（PR）的患者均维持缓解超过6个月，其中1例实现了靶病灶完全缓解（pCR），且持续缓解超过27个月。此外，患者的肿瘤缓解与SCG101的抗病毒活性高度相关。4例血清乙型肝炎病毒表面抗原（HBsAg）显著下降并维持在低水平患者的肿瘤缩小，且无进展生存期和总生存期显著延长至5.9个月和19.0个月。 SCG101是一种针对HBsAg特异性的自体T细胞受体（TCR）T细胞疗法。星汉德生物依托自主研发的独有GianT技术平台，筛选高亲力且高活性的天然TCR，可以有效靶向实体肿瘤中通过主要组织相容性复合体（MHC）表达的细胞内抗原。临床前和临床研究数据显示，SCG101具有肿瘤抑制和HBV共价闭合环状DNA（cccDNA）根除作用，可实现抗肿瘤和抗病毒功能。 ◇ Affimed公司在第一季度财报中公布了其先天细胞衔接蛋白acimtamig与Artiva Biotherapeutics公司开发的同种异体自然杀伤（NK）细胞AlloNK联用，治疗难治性霍奇金淋巴瘤的试验结果。临床试验LuminICE-203的初步数据显示，acimtamig与AlloNK联用，在7名患者中达到约86%（6/7）的客观缓解率，CR率超过50%（4/7）。 ◇ IN8bio公司公布了其在研现货型γδ T细胞疗法INB-100的最新1期临床试验结果。INB-100是一款来自健康供体的同种异体γδ T细胞，在体外经过扩增和激活后，被输注到白血病患者体内。在这项研究者发起的1期临床试验中，白血病患者在接受造血干细胞移植后，接受一剂INB-100的治疗。这些患者包括高风险和复发急性髓系白血病（AML）患者，他们此前已接受过多种疗法治疗，包括嵌合抗原受体（CAR）T细胞疗法。截至2024年5月31日的数据，100%可评估的白血病患者仍然存活并且维持CR达到1年。此前发表的历史数据显示，高达50%的血液癌症患者在接受造血干细胞移植和减轻强度预处理（reduced intensity conditioning）的情况下会出现复发，并且在复发后不久去世。在这10名患者中，两名接受INB-100治疗的患者已经无癌三年半，另一名患者接近3年无癌。数据同时显示，接受单次治疗后的365天里，同种异体γδ T细胞能够长期持久地在体内扩增。新闻稿指出，这是首次同种异体细胞疗法表现出持续的扩增和持久性。安全性方面，INB-100没有出现剂量限制性毒性，未发现细胞因子释放综合征（CRS），神经毒性或免疫效应细胞相关神经毒性综合征（ICANS），未发现严重感染。 ◇ 近日，《自然-医学》杂志上刊登了由希望之城（City of Hope）的研究人员开发的前列腺干细胞抗原（PSCA）靶向CAR-T细胞疗法用于治疗晚期前列腺癌的1期临床试验的初步结果。14名接受该CAR-T细胞疗法治疗的患者中有5人出现了1级或2级的CRS。14名患者中有4人的前列腺特异性抗原（PSA）下降>30%，放射学检查结果也有所改善。1名患者的PSA水平下降了95%，并获得了大约8个月的缓解，其骨骼和软组织中的肿瘤也有所减少。注射后28天后，研究人员观察到该CAR-T细胞的持久性有限，他们计划在后续的试验中增加CAR-T细胞的数量，以提高治疗的有效性。 ◇ KSQ Therapeutics公司宣布，其工程化肿瘤浸润淋巴细胞（eTIL）疗法KSQ-001EX的1/2期临床研究已完成了首例患者给药。这项开放标签研究将在黑色素瘤、头颈部鳞状细胞癌和非小细胞肺癌患者中评估KSQ-001EX的安全性和抗癌活性。KSQ-001EX由SOCS1基因被CRISPR/Cas9基因编辑失活的TIL组成。KSQ的CRISPRomics平台确定SOCS1是调控TIL抗肿瘤效力和持久性的关键基因，这一改造为KSQ-001EX在实体瘤治疗中提供了”best-in-class“潜力。 ◇ Beacon Therapeutics公司宣布，其用于治疗X连锁色素性视网膜炎（XLRP）的在研基因疗法AGTC-501的关键性2/3期临床试验VISTA已完成首例患者给药。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。该公司计划使用VISTA试验产生的数据，结合1/2期HORIZON和2期SKYLINE研究的数据，包括所有可用的长期数据，来支持该候选基因疗法在美国和欧洲的上市申请。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ 致力于开发治疗眼部遗传性和慢性疾病的基因治疗创新公司朗信生物宣布完成B+轮融资，所获资金将进一步加速朗信生物在基因治疗创新药物领域的研发和产业化进程。此轮融资由上海生物医药基金领投，联和投资等股东跟投。目前，朗信生物共推进5个候选分子进入临床，在3项致盲性眼病适应症中率先开展基因治疗临床研究，包括RPE65突变相关遗传性视网膜营养不良（IRD）、湿性年龄性黄斑变性（nAMD）和X连锁视网膜劈裂症（XLRS）。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] BioMarin Presents New Phase 3, Four-Year Data Underscoring Long-Term Safety and Efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2024 Congress. Retrieved June 14, 2024 from https://www.prnewswire.com/news-releases/biomarin-presents-new-phase-3-four-year-data-underscoring-long-term-safety-and-efficacy-of-roctavian-valoctocogene-roxaparvovec-rvox-at-international-society-on-thrombosis-and-haemostasis-2024-congress-302166583.html [2] New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears. Retrieved June 14, 2024 from https://www.eurekalert.org/news-releases/1046533 [3] Wang, H., Chen, Y., Lv, J. et al. Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results. Nat Med (2024). https://doi-org.libproxy1.nus.edu.sg/10.1038/s41591-024-03023-5 [4] 重磅｜EASL 最新突破性数据：星汉德生物TCR-T细胞治疗晚期肝癌患者总生存期显著延长并伴随乙肝表面抗原清除，获选最佳精选报告. Retrieved June 14, 2024 from https://www.prnasia.com/story/450060-1.shtml [5] City of Hope CAR T Cell Therapy for Advanced Prostate Cancer Demonstrates Positive Results in Phase 1 Clinical Trial. Retrieved June 14, 2024, from https://www.businesswire.com/news/home/20240612751963/en [6] Dorff, T.B., Blanchard, M.S., Adkins, L.N. et al. PSCA-CAR T cell therapy in metastatic castration-resistant prostate cancer: a phase 1 trial. Nat Med (2024). https://doi-org.libproxy1.nus.edu.sg/10.1038/s41591-024-02979-8 [7] KSQ Therapeutics Announces First Patient Dosed in Clinical Development Program for KSQ-001EX, a CRISPR/Cas9 Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapy. Retrieved June 14, 2024, from https://ksqtx.com/news-events/ksq-therapeutics-announces-first-patient-dosed-in-clinical-development-program-for-ksq-001ex-a-crispr-cas9-engineered-tumor-infiltrating-lymphocyte-etil-therapy/ [8] Beacon Therapeutics Treats First Patient in VISTA Registrational Trial for AGTC-501. Retrieved June 14, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-treats-first-patient-in-vista-registrational-trial-for-agtc-501-302170126.html [9] 朗信生物完成B+轮融资，推进基因治疗药物研发与产业化. Retrieved June 14, 2024, from https://mp.weixin.qq.com/s/EW_6gXRyp_FvXRNae6LFSA [10] Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA). Retrieved June 13, 2024 from https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-plans-file-accelerated-approval-ux111 [11] IN8bio Presents Positive Data Demonstrating Durable 1-year Complete Remission in 100% of Evaluable Patients in Phase 1 Trial of INB-100. Retrieved June 13, 2024, from https://www.globenewswire.com/news-release/2024/06/13/2898625/0/en/IN8bio-Presents-Positive-Data-Demonstrating-Durable-1-year-Complete-Remission-in-100-of-Evaluable-Patients-in-Phase-1-Trial-of-INB-100.html [12] AFFIMED REPORTS FIRST QUARTER 2024 FINANCIAL RESULTS & BUSINESS UPDATE. Retrieved June 12, 2024, from https://www.affimed.com/affimed-reports-first-quarter-2024-financial-results-business-update/ 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

基因疗法临床3期临床结果

2024-06-14

·GlobeNewswire-Health Care

KSQ Therapeutics Announces First Patient Dosed in Clinical Development Program for KSQ-001EX, a CRISPR/Cas9 Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapy

June 12, 2024 -- KSQ Therapeutics, Inc. (KSQ), a clinical-stage biotechnology company developing treatments for solid tumors, today announced the first patient dosed in the Phase 1/2 clinical study of KSQ-001EX, a novel edited TIL therapy. KSQ-001EX consists of TIL in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing, giving it the potential to be a best-in-class treatment for a variety of solid tumor indications. KSQ's CRISPRomics® platform identified SOCS1 as a key gene inhibiting T cell growth, survival, and differentiation by negatively regulating cytokine signaling in the tumor microenvironment. "TIL have emerged as a powerful new treatment modality, and we believe both of our eTIL® programs, KSQ-001EX and KSQ-004EX, have the potential to significantly advance the field by improving the potency of TIL therapy for the treatment of a variety of solid tumor types. In preclinical studies, KSQ-001EX demonstrated enhanced anti-tumor activity, polyclonality, persistence, and memory formation in multiple solid tumor models compared to unmodified TIL. We also saw robust anti-tumor activity in models insensitive to PD-1 inhibition,” said Qasim Rizvi, Chief Executive Officer of KSQ. “We look forward to evaluating KSQ-001EX in patients with difficult-to-treat solid tumors, including melanoma, head and neck squamous cell carcinoma, and non-small cell lung cancer.” The first patient treated with KSQ-001EX was enrolled by Rodabe Amaria, M.D., professor of Melanoma Medical Oncology and principal investigator of the study at The University of Texas MD Anderson Cancer Center. The Phase 1/2 clinical trial is an open-label, safety lead-in study for patients with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC). The primary objective of the Phase 1 portion of the trial is to evaluate the safety and tolerability of KSQ-001EX. In the safety lead-in portion, a cohort of patients will be initially dosed without IL-2. The primary objective of Phase 2 is to evaluate antitumor activity in indication-specific cohorts. KSQ’s lead eTIL® cell therapy program, KSQ-001EX, in which TIL are edited to inactivate the SOCS1 gene, has the potential to revolutionize the treatment of solid tumors. In preclinical studies, KSQ-001EX demonstrated enhanced anti-tumor function in solid tumor models refractory to PD-1 inhibition, as well as enhanced persistence and memory formation. KSQ Therapeutics is advancing a pipeline of novel drug candidates to treat cancer across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ's proprietary CRISPRomics® discovery engine enables genome-scale insights driving novel therapeutic discovery. For more information, please visit the company's website at www.ksqtx.com. The content above comes from the network. if any infringement, please contact us to modify.

免疫疗法基因疗法细胞疗法

100 项与 KSQ-001 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
晚期恶性实体瘤	临床2期	美国	KSQ Therapeutics, Inc.初创企业	2024-01-31
头颈部肿瘤	临床2期	美国	KSQ Therapeutics, Inc.初创企业	2024-01-31
肺癌	临床2期	美国	KSQ Therapeutics, Inc.初创企业	2024-01-31
黑色素瘤	临床2期	美国	KSQ Therapeutics, Inc.初创企业	2024-01-31
非小细胞肺癌	临床申请批准	美国	KSQ Therapeutics, Inc.初创企业	2023-11-29
头颈部鳞状细胞癌	临床申请批准	美国	KSQ Therapeutics, Inc.初创企业	2023-11-29
实体瘤	临床前	美国	Takeda Pharmaceutical Co., Ltd.	-

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分期

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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


SITC2022	N/A	SOCS1 gene	11	KSQ-001	遞襯蓋願築鹽醖衊遞遞(襯糧積餘鑰範糧鹹簾範) = KSQ-001 manufactured using ExRPESS exhibited heightened anti-tumor function when compared to un-engineered TIL 膚範夢網鹽獵築窪製壓 (鏇醖築願蓋獵構鏇積遞 )	积极	2022-11-07
SITC2022	N/A	头颈部鳞状细胞癌	8	KSQ-001	簾構淵簾鹹願顧窪構遞(廠選鹹廠網簾窪鏇艱選) = enhanced against 3D tumor spheroids in vitro 鬱淵範襯積廠鑰鬱觸網 (糧鏇壓壓齋憲選鹽壓簾 ) 更多	积极	2022-11-07
SITC2021	N/A	实体瘤	-	KSQ-001	鬱網觸憲齋醖廠獵齋鬱(鏇齋廠觸鹹顧艱範觸壓) = exhibited a hypersensitivity to IL-12 signaling 鏇繭範簾鬱醖選遞廠憲 (顧構構艱糧壓鏇鹹觸鬱 ) 更多	-	2021-11-10