Phase I/II Trial to Determine the in Vivo Engraftment, Safety and Clinical Activity of Allogeneic CIK Cells Transduced With a Transposon CD19-chimeric Antigen Receptor (CARCIK-CD19) Gene in Adult and Pediatric Patients With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma

This is a single arm, open-label, multi-center, phase I/II study to determine the engraftment, safety and clinical activity of allogeneic CARCIK-CD19 cells in adult and pediatric patients with relapsed/refractory mature B-cell neoplasia expected to express CD19 i.e. B-cell NHL and CLL.
CARCIK-CD19 will be produced from the peripheral blood of an at least haploidentical familial donor.

开始日期2023-12-01

申办/合作机构

Fondazione Matilde Tettamanti e Menotti de March

NCT05252403

/ Recruiting临床2期IIT

Measurable Residual Disease Driven Strategy for One or Two Infusions of Non- Viral, Transposon-manipulated CARCIK (CD19) Cells: A Phase II Study in Pediatric and Adult Patients With Relapsed/Refractory B Cell Precursor ALL (BCP-ALL)

This is a single arm, open-label, multi-center, phase II study to determine the activity and the safety of a therapeutic strategy that allows a second CARCIK-CD19 cells infusion, driven by the status of disease from one month after the first infusion, in adult and pediatric patients with r/r BCP- ALL.

开始日期2021-12-01

申办/合作机构

Fondazione Matilde Tettamanti e Menotti de March

NCT03389035

/ Completed临床1/2期IIT

Phase 1-2a Trial to Determine the Feasibility and Safety of a Single Dose of Transposon-manipulated Allogeneic CARCIK-CD19 Cells in Adult and Pediatric Patients With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia, After Hematopoietic Stem Cell Transplantation

This is a single arm, open-label, multi-center, phase 1-2a study to determine the Maximum Tolerated Dose and/or the Recommended Phase 2 Dose and the safety of CARCIK-CD19 in adult and pediatric patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

开始日期2017-12-20

申办/合作机构

Fondazione Matilde Tettamanti e Menotti de March

100 项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的临床结果

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100 项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的转化医学

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100 项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的专利（医药）

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项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的文献（医药）

CANCER IMMUNOLOGY IMMUNOTHERAPY

Advancing CIK cell immunotherapy: highlights from the second international conference on DC-CIK and CAR-CIK approaches

Article

作者: Sharma, Amit ; Schmidt-Wolf, Ingo G H ; Pu, Jingjing ; Introna, Martino ; Rambaldi, Alessandro ; Ren, Xiubao ; Rosato, Antonio ; Serafini, Marta ; Fenix, Kevin Aaron ; Hou, Jian ; Sangiolo, Dario ; Tettamanti, Sarah

On April 7, 2025, the 2nd International CIK Cell Conference was held in Monza, Italy, marking the establishment of the International Society for CIK Cells (ISCC). The conference, kindly supported by Fondazione Tettamanti (Italy), featured cutting-edge advancements in cytokine-induced killer (CIK) cell immunotherapy, used alone and in combination with dendritic cells (DC-CIK) and chimeric antigen receptors (CAR-CIK). The event also provided a valuable opportunity for young investigators to engage in insightful discussions and to network within experts in the field.

Journal of Clinical Medicine

Prevention and Treatment of Acute Myeloid Leukemia Relapse after Hematopoietic Stem Cell Transplantation: The State of the Art and Future Perspectives

Review

作者: Leotta, Salvatore ; Spadaro, Andrea ; Condorelli, Annalisa ; Cupri, Alessandra ; Garibaldi, Bruno ; Schininà, Giovanni ; Milone, Giuseppe ; Milone, Giulio ; Bellofiore, Claudia ; Sciortino, Roberta

Allogeneic hematopoietic stem cell transplantation (HSCT) for high-risk acute myeloid leukemia (AML) represents the only curative option. Progress has been made in the last two decades in the pre-transplant induction therapies, supportive care, selection of donors and conditioning regimens that allowed to extend the HSCT to a larger number of patients, including those aged over 65 years and/or lacking an HLA-identical donor. Furthermore, improvements in the prophylaxis of the graft-versus-host disease and of infection have dramatically reduced transplant-related mortality. The relapse of AML remains the major reason for transplant failure affecting almost 40–50% of the patients. From 10 to 15 years ago to date, treatment options for AML relapsing after HSCT were limited to conventional cytotoxic chemotherapy and donor leukocyte infusions (DLI). Nowadays, novel agents and targeted therapies have enriched the therapeutic landscape. Moreover, very recently, the therapeutic landscape has been enriched by manipulated cellular products (CAR-T, CAR-CIK, CAR-NK). In light of these new perspectives, careful monitoring of minimal-residual disease (MRD) and prompt application of pre-emptive strategies in the post-transplant setting have become imperative. Herein, we review the current state of the art on monitoring, prevention and treatment of relapse of AML after HSCT with particular attention on novel agents and future directions.

Journal of experimental & clinical cancer research : CR

CSPG4 CAR-redirected Cytokine Induced Killer lymphocytes (CIK) as effective cellular immunotherapy for HLA class I defective melanoma

Article

作者: Ferrone, Cristina R ; Iaia, Ilenia ; Marchiò, Caterina ; Biolato, Andrea Michela ; Giraudo, Lidia ; Carnevale-Schianca, Fabrizio ; Pisacane, Alberto ; Merlini, Alessandra ; Basiricò, Marco ; Poletto, Stefano ; Sangiolo, Dario ; Caravelli, Daniela ; Centomo, Maria Laura ; Massa, Annamaria ; Dotti, Gianpietro ; Donini, Chiara ; Paruzzo, Luca ; Landoni, Elisa ; Picciotto, Franco ; Bortolot, Valentina ; Leuci, Valeria ; Gammaitoni, Loretta ; Vigna, Elisa ; Ventin, Marco ; Cattaneo, Giulia ; Berrino, Enrico ; Aglietta, Massimo

Abstract:

Background:

Even acknowledging the game-changing results achieved in the treatment of metastatic melanoma with the use of immune checkpoint inhibitors (ICI), a large proportion of patients (40–60%) still fail to respond or relapse due to the development of resistance. Alterations in the expression of Human Leukocyte Antigen class I (HLA-I) molecules are considered to play a major role in clinical resistance to ICI. Cellular immunotherapy with HLA-independent CAR-redirected lymphocytes is a promising alternative in this challenging setting and dedicated translational models are needed.

Methods:

In this study, we propose an HLA-independent therapeutic strategy with Cytokine Induced Killer lymphocytes (CIK) genetically engineered with a Chimeric Antigen Receptor (CAR) targeting the tumor antigen CSPG4 as effector mechanism. We investigated the preclinical antitumor activity of CSPG4-CAR.CIK in vitro and in a xenograft murine model focusing on patient-derived melanoma cell lines (Mel) with defective expression of HLA-I molecules.

Results:

We successfully generated CSPG4-CAR.CIK from patients with metastatic melanoma and reported their intense activity in vitro against a panel of CSPG4-expressing patient-derived Mel. The melanoma killing activity was intense, even at very low effector to target ratios, and not influenced by the expression level (high, low, defective) of HLA-I molecules on target cells. Furthermore, CAR.CIK conditioned medium was capable of upregulating the expression of HLA-I molecules on melanoma cells. A comparable immunomodulatory effect was replicated by treatment of Mel cells with exogenous IFN-γ and IFN-α. The antimelanoma activity of CSPG4-CAR.CIK was successfully confirmed in vivo, obtaining a significant tumor growth inhibition of an HLA-defective Mel xenograft in immunodeficient mice.

Conclusions:

In this study we reported the intense preclinical activity of CSPG4-CAR.CIK against melanoma, including those with low or defective HLA-I expression. Our findings support CSPG4 as a valuable CAR target in melanoma and provide translational rationale for clinical studies exploring CAR-CIK cellular immunotherapies within the challenging setting of patients not responsive or relapsing to immune checkpoint inhibitors.

项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的新闻（医药）

2024-09-11

·Pharmaceutical Technology

IBA venture PanTera gains €134m in funding to scale up actinium-225 production

PanTera aims to accelerate actinium-225 production, used in promising targeted alpha therapy for cancer. Image: Getty Images / Love Employee. PanTera, a Belgian radioisotope producer, announced today (11 September) that it had secured €93m ($102.4m) in an oversubscribed Series A financing round to accelerate production of actinium-225. The radioactive isotope is integral to several investigational cancer therapies. This is the largest Series A financing round in the Belgian life sciences sector to date. The round was led by EQT Life Sciences. Ion Beam Applications (IBA), which invested in the joint venture PanTera with SCK CEN (the Belgian Nuclear Research Centre), and SFPIM—Belgium’s sovereign wealth fund, will also convert loans into €7.2m in equity. Along with €33.8m in debt financing, PanTera will have raised a total of €134m, increasing their valuation to €280m. The deal will see IBA’s shareholding in PanTera drop from 47.8% to 31.3% but will net IBA roughly €23m in profit as a result. Following the announcement, IBA share price was up by 4% when the market opened today, bringing its market cap to €3.29bn. Actinium-225 is a crucial component of targeted alpha therapy. The isotope is conjugated with cancer antigen-targeting elements to deliver localised radiation to cancer cells while minimising damage to healthy tissue. Several investigational treatments for metastatic castration-resistant prostate cancer (mCRPC) depend on actinium-225, including Johnson & Johnson’s JNJ-6420 , Eli Lilly’s PNT-2001, and Novartis ’s AAA-802. The funds are set to support PanTera’s patented photo-nuclear ‘gamma’ process which converts radium-226 to actinium-225. Already, the company is on track to produce 1.5 – 2 Curies (Ci) of the isotope annually from early 2025, a large portion of the estimated 3 Ci per year global supply. Through the gamma process, PanTera claims they will produce over 100 Ci annually by 2029, enough to treat more than 100,000 patients. See Also: CMN-005 by CoImmune for Extranodal Marginal Zone B-Cell Lymphoma (Mucosa-Associated Lymphoid Tissue or MALT-Lymphoma): Likelihood of Approval CMN-005 by CoImmune for Nodal Marginal Zone B-Cell Lymphoma: Likelihood of Approval PanTera CEO Sven Van den Berghe said the company aims to radically improve supply in the short and long term to address what Marijn Kleijwegt, Partner at EQT Life Sciences, described as a “critical actinium-225 supply shortage”. PanTera seeks to employ SCK CEN’s large radium-226 stock and IBA’s Rhodotron electron accelerator to overcome the issues of complexity and infrastructure contributing to the global shortage. As pharmaceutical companies ramp up clinical trialling for alpha particle-targeted therapies across various cancers, demand is poised to increase further. The company has signed supply deals with pharmaceutical manufacturers, such as Bayer for their therapies BAY-3563254 and BAY-3546828, both in Phase I trials for mCRPC. It expects to arrange deals to account for over 80% of its productive capacity before production begins.

放射疗法临床1期

2024-09-10

·Pharmaceutical Technology

T-cell targeting Candid Therapeutics launches with $370m

Candid plans to develop antibody drugs for inflammatory diseases such as rheumatoid arthritis. Credit: Drazen Zigic via Shutterstock. T cell engager (TCE) startup Candid Therapeutics has debuted with $370m in its pocket to develop antibody drugs for inflammatory diseases. Candid plans to develop T cell engager antibodies to selectively target and deplete specific B lymphocyte populations that play a role in autoimmune diseases. Selective depletion of B lymphocytes has shown clinical effectiveness in patients with conditions such as rheumatoid arthritis and myasthenia gravis. Headquartered in San Diego, California, the company’s Series A funding round was co-led by Venrock Healthcare Capital Partners, Fairmount, TCGX and venBio Partners, with Foresite Capital and Third Rock Ventures also taking part, among others. As part of its debut, Candid inherited two advanced bispecific antibodies to kick off its portfolio. CND106 is a BCMAxCD3 antibody from Vignette, and CD20xCD3 is an antibody from TRC 2004. Both candidates, which have completed Phase I trials with over 130 oncology patients, show potential as therapies for autoimmune diseases, according to Candid. TCE antibodies have the most promise in becoming scalable and patient-friendly drugs that deplete B cells for the treatment of several autoimmune conditions, said Candid’s new CEO Ken Song, whose previous company RayzeBio was acquired by Bristol Myers Squibb in December 2023. See Also: CMN-005 by CoImmune for Extranodal Marginal Zone B-Cell Lymphoma (Mucosa-Associated Lymphoid Tissue or MALT-Lymphoma): Likelihood of Approval CMN-005 by CoImmune for Nodal Marginal Zone B-Cell Lymphoma: Likelihood of Approval Candid joins a number of high-profile companies that are making waves in the space. In January 2024, MSD acquired Harpoon in a $680m deal, which included Harpoon’s T cell engagers. One of the candidates, named HPN328, is being tested in a Phase I/II trial (NCT04471727) for some types of lung cancer and neuroendocrine tumours. HPN328 was developed using Harpoon’s TriTAC platform. In April, Roche shared data from a trial of its bispecific T cell engager Columvi (glofitamab). The drug, which targets CD3 on T cells and CD20 on B cells, improved survival in a Phase III lymphoma trial. The 270 participants received either Columvi or Rituxan (rituximab) on top of a regimen of two chemotherapy drugs, gemcitabine and oxaliplatin. In the announcement accompanying the launch, Song said that Candid’s drugs have the potential to “go above and beyond the clinical and commercial success of Humira and Rituxan.” AbbVie ’s Humira (adalimumab) and Biogen and Genentech’s Rituxan are blockbuster autoimmune drugs that pulled in $14.5bn and $1.8bn last year, respectively, as per the company’s financials.

临床1期免疫疗法临床3期临床结果并购

2024-09-09

·Pharmaceutical Technology

Formosa Pharma ships APP13007 ophthalmic drug to US

Eyenovia has initiated pre-launch activities and is expected to begin the commercialisation of APP13007 by late September 2024. Credit: Formosa Pharmaceuticals Inc. Formosa Pharmaceuticals has announced the first shipment of its ophthalmic asset, clobetasol propionate ophthalmic suspension 0.05% (APP13007),to the US to facilitate commercialisation. The drug, developed using Formosa’s APNT nanotechnology platform, is the first US Food and Drug Administration (FDA)-approved ophthalmic solution featuring the super-potent corticosteroid clobetasol propionate. Bora Pharmaceuticals Ophthalmic has manufactured the shipment of APP13007, which offers a twice-daily dosing regimen that is more convenient than the four doses per day required by other treatments. Eyenovia , Formosa Pharma’s US partner, initiated pre-launch activities and is expected to begin the commercialisation of APP13007 by late September 2024. In 2023, Eyenovia acquired exclusive licensing and commercialisation rights for APP13007 in the US from Formosa Pharmaceuticals in an $86m deal. See Also: CMN-005 by CoImmune for Extranodal Marginal Zone B-Cell Lymphoma (Mucosa-Associated Lymphoid Tissue or MALT-Lymphoma): Likelihood of Approval CMN-005 by CoImmune for Nodal Marginal Zone B-Cell Lymphoma: Likelihood of Approval In August 2024, Formosa obtained a drug export licence from Taiwan’s Food and Drug Administration. The licence has enabled the company to co-ordinate its first shipment to the US, a key step in its strategy for global commercialisation. In anticipation of demand within the US market, Formosa is also collaborating with partners in other regions to prepare for regulatory submissions. The goal is to make APP13007 available in international markets, expanding its reach beyond the US. Formosa Pharmaceuticals focuses primarily on ophthalmology and oncology. Its APNT technology enhances the dissolution, bioavailability and stability of active pharmaceutical ingredients, enabling the use of poorly soluble or highly potent drug agents. Formosa Pharma president and CEO Dr Erick Co said, “This first shipment to Eyenovia for the much-anticipated commercial US launch is an exclamation point on our development of APP13007. “We thank all our partners for making this journey with us and are eager to provide this formidable therapy to ocular surgery patients worldwide. We look forward to creating continued value for our stakeholders and shareholders.”

引进/卖出

100 项与 CIK-CAR CD19(Fondazione Matilde Tettamanti e Menotti de March) 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
难治性B细胞淋巴瘤	临床2期	意大利	Fondazione Matilde Tettamanti e Menotti de March	2023-12-01
里氏综合症	临床2期	意大利	Fondazione Matilde Tettamanti e Menotti de March	2023-12-01
CD19阳性急性淋巴细胞白血病	临床2期	意大利	Fondazione Matilde Tettamanti e Menotti de March	2021-12-01
难治性 B 细胞急性淋巴细胞白血病	临床2期	意大利	Fondazione Matilde Tettamanti e Menotti de March	2017-12-20
T细胞急性淋巴细胞白血病复发	临床2期	意大利	Fondazione Matilde Tettamanti e Menotti de March	2017-12-20
非霍奇金淋巴瘤	临床前	意大利	Formula Pharmaceuticals, Inc.	2020-11-01

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临床结果

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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


NCT03389035 (FT01CARCIK, ASH 12022 \| ASH 22022) 人工标引	临床1/2期	CD19阳性B细胞急性淋巴细胞白血病	27	CARCIK-CD19	淵鬱選願蓋獵淵願繭廠(遞積窪遞構製窪壓積鑰) = 淵範餘鹽遞網壓鏇製衊觸積積襯廠鑰餘夢鏇積 (淵顧願醖鏇遞蓋選憲積 ) 更多	积极	2022-11-15