REDWOOD CITY, Calif. & SOUTH SAN FRANCISCO, Calif.---- Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, and Graphite Bio, Inc., a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced a research and clinical collaboration agreement to evaluate JSP191, Jasper’s first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen for Graphite Bio’s investigational GPH201 gene replacement therapy for severe combined immune deficiency in patients with IL2RG deficiency, known as x-linked SCID .
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XSCID is a severe, inherited disorder of the immune system with symptoms often presenting in early infancy, including persistent infections and failure to thrive. Without treatment, XSCID is typically fatal to patients in the first two years of life.
Graphite Bio is focused on the development of potentially curative therapies for patients suffering from serious diseases, using its targeted gene integration platform to harness the natural cellular process of homology directed repair (HDR) in order to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Jasper Therapeutics’ JSP191 is a first-in-class humanized monoclonal antibody that depletes hematopoietic stem cells from bone marrow and acts as a conditioning agent in patients prior to receiving a hematopoietic stem cell transplant. JSP191 is currently being evaluated in multiple trials as a stem cell depleting conditioning agent, including a Phase 1/2 trial to achieve donor stem cell engraftment in SCID patients undergoing hematopoietic cell transplant and a separate Phase 1/2 trial in AML/MDS patients undergoing hematopoietic cell transplant.
“This collaboration with Jasper demonstrates our shared commitment to pioneering novel therapeutic approaches with the potential to significantly improve the treatment experiences of individuals with devastating conditions who stand to benefit from gene replacement therapies, initially for patients with XSCID,” said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. “GPH201 harnesses our targeted gene integration platform to precisely target the defective gene that causes XSCID and replace it with a normal copy. We are impressed by the initial positive clinical results demonstrated by JSP191 when used as a conditioning regimen, and look forward to collaborating with the Jasper team to explore how our novel technologies can be brought to more patients with XSCID and other indications.”
“Our collaboration with Graphite Bio is an exciting opportunity to further advance the field of curative gene correction by combining a targeted gene integration platform with our first-in-class targeted CD117 antibody, JSP191, that has already demonstrated preliminary clinical efficacy and safety as a conditioning agent in XSCID patients and those with blood cancers undergoing allogeneic hematopoietic stem cell transplant,” said Bill Lis, executive chairman and CEO, Jasper Therapeutics.
Graphite Bio and Jasper will collaborate on research, and potentially a clinical study, evaluating JSP191 as a conditioning agent for GPH201. Each company will retain commercial rights to their respective technologies.
JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.
Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.
JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit www.clinicaltrials.gov (NCT02963064 and NCT04429191).
Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.
GPH201 is a first-in-human investigational hematopoietic stem cell treatment that will be evaluated as a potentially curative therapy for patients suffering from XSCID. GPH201 is generated using Graphite Bio’s precise and efficient targeted gene integration platform technology to directly replace the defective IL2RG gene, maintain normal IL2RG regulation and expression, and ultimately lead to the production of fully functional adaptive immune cells.
About Jasper Therapeutics
Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company’s lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.
About Graphite Bio, Inc.
Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The company’s targeted gene integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients’ lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit graphitebio.com.
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Source: Jasper Therapeutics, Inc.
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加州红木城。Jasper Therapeutics，Inc.一家专注于造血细胞移植疗法的生物技术公司和Graphite Bio，Inc.一家专注于利用靶向基因整合治疗或治愈严重疾病疗法的新一代基因编辑公司，今天宣布了一项研究和临床合作协议，以评估Jasper公司一流的抗CD117单克隆抗体JSP191，作为Graphite Bio公司研究的GPH201基因替代疗法的靶向，无毒调理方案，用于治疗IL2RG缺乏症患者的严重联合免疫缺陷，称为X连锁SCID。
Graphite Bio致力于为患有严重疾病的患者开发潜在的治疗方法，利用其靶向基因整合平台，利用同源定向修复（HDR）的天然细胞过程，从源头上有效修复遗传缺陷，精确地传递基因货物，并设计新的细胞效应功能。Jasper TherapeuticsJSP191是一种一流的人源化单克隆抗体，可从骨髓中清除造血干细胞，并在接受造血干细胞移植前作为患者的调理剂。JSP191目前正在多项试验中作为干细胞耗尽调理剂进行评估，包括在接受造血细胞移植的SCID患者中实现供体干细胞植入的1/2期试验，以及在接受造血细胞移植的AML/MDS患者中单独进行的1/2期试验。
Graphite Bio公司首席执行官Josh Lehrer博士说:“与Jasper公司的合作表明，我们共同致力于开拓新的治疗方法，这些方法有可能显著改善患有破坏性疾病的患者的治疗经验，这些患者将从基因替代疗法中获益，首先是XSCID患者。”GPH201利用我们的靶向基因整合平台，精确地靶向导致XSCID的缺陷基因，并用正常拷贝替换它。当使用JSP191作为调理方案时，我们对最初的积极临床结果印象深刻，并期待与Jasper团队合作，探索如何将我们的新技术带给更多XSCID和其他适应症的患者。
Jasper Therapeutics执行董事长兼首席执行官Bill Lis说:我们与Graphite Bio的合作是一个激动人心的机会，通过将靶向基因整合平台与我们一流的靶向CD117抗体JSP191相结合，进一步推进治疗性基因矫正领域，JSP191已在XSCID患者和接受异基因造血干细胞移植的血癌患者中作为一种调理剂证明了初步的临床疗效和安全性。
石墨生物是一家下一代基因编辑公司，致力于为患有严重疾病的患者开发具有潜在疗效的疗法。该公司的靶向基因整合平台利用同源定向修复（HDR）的天然细胞过程，从源头上有效修复基因缺陷，精确地传递基因货物，并设计新的细胞效应器功能。Graphite Bio正在利用其差异化平台（最初专注于造血干细胞的离体工程）来推进一系列具有挽救和显著改善患者生命潜力的变革性治疗。该公司由基因编辑和基因治疗领域的学术先驱共同创立，其中包括Maria Grazia Roncarolo医学博士和Matthew Porteus医学博士和PhD，并得到Versant Ventures和Samsara Biocapital的支持。欲了解更多信息，请访问Graphitebio.com。