Gene and Virus Therapy Shared Resource

基因与病毒治疗资源共享项目总结 梅奥诊所长期致力于将前景看好的癌症疗法转化为使患者受益的疗法 它的临床实践。新药的翻译，包括有希望的基于病毒的疗法，从周期 从基础和临床前研究到临床试验需要专业知识和设施，这在学术上是不常见的 布景。因此，临床试验过程的启动可能会显著延迟或完全受阻 学术研究员/临床医生。学术界可以采取的途径很少。 研究人员/临床医生尝试并获得必要的专业知识和设施。梅奥诊所 分子医学部(DMM)在现场建立了必要的核心资源来翻译病毒- 以梅奥诊所的研究为基础，进行一期和二期临床试验。梅奥诊所癌症中心(MCCC) 该资源的组成部分，基因和病毒治疗共享资源(GVTSR)是在 与MCCC基因和病毒治疗计划相结合，为MCCC成员提供最广泛的 灵活地获取翻译基于病毒的 以高效和经济的方式将治疗转化为临床试验。GVTSR已经开发了资源 制造大规模临床级病毒产品的专业知识，进行毒理学和 病毒治疗药物在动物模型中的药理学特征，并确保这些药物的质量 活动。GVTSR签名设施是2000平方英尺。英国《金融时报》生产病毒载体的制造设施 实验室，拥有一个可运行多达75次L病毒生产的GMP套件，以及一个用于大型病毒生产的临床前套件 规模化病毒生产流程开发。额外的实验室空间专门用于毒物/制药实验室 和QC职能，以及梅奥诊所全面的动物设施，以支持GVTSR 需要。这些GVTSR活动必须使用联邦授权的实践(良好实验室实践 和良好的制造规范)，需要特定的人员培训、设施和质量控制，以及 质量保证计划。GVTSR还与FDA建立了牢固的关系，FDA 联邦政府对这些活动的授权。GVTSR可以提供IND的两个主要部件 用途：产品制造和安全试验说明，毒理学和 在适当的动物模型中对该产品进行药理学表征。同样重要的还有 GVTSR开发大规模病毒生产和纯化工艺的能力，以支持 临床前研究以及临床级产品的制造。GVTSR密切合作 与临床医生一起开发和监管批准临床试验方案。这一全面的 方法导致MCCC成员将多种基于病毒的新疗法转变为13个阶段I和2 治疗15种不同类型肿瘤的II期临床试验。

GENE AND VIRUS THERAPY SHARED RESOURCE PROJECT SUMMARY Mayo Clinic has a long-standing commitment to translating promising cancer therapies that benefit its patients to its clinical practice. The translation of new drugs, including promising virus-based therapies, from the cycles of basic and preclinical research to clinical trials requires expertise and facilities not often found in an academic setting. Thus, the initiation of the clinical trial process can be significantly delayed or completely blocked for the academic researcher/clinician. There are very few avenues that can be taken by the academic researcher/clinician to try and gain access to the necessary expertise and facilities. The Mayo Clinic Department of Molecular Medicine (DMM) established the necessary core resources on-site to translate virus- based research at the Mayo Clinic to Phase I and II clinical trials. The Mayo Clinic Cancer Center (MCCC) component of this resource, the Gene and Viral Therapy Shared Resource (GVTSR), was developed in conjunction with the MCCC Gene and Virus Therapy Program and provides MCCC members the broadest flexibility to access the complete range of expertise and services required for translation of a virus-based therapy to a clinical trial in an efficient and cost-effective manner. The GVTSR has developed the resources and expertise to manufacture large-scale clinical grade viral products, to conduct toxicological and pharmacological characterization of viral therapeutics in animal models, and to assure the quality of these activities. The GVTSR signature facility is the 2000 sq. ft. manufacturing facility of the Viral Vector Production Laboratory, with one GMP suite capable of up to 75 L virus production runs, and a preclinical suite for large- scale virus production process development. Additional laboratory space is dedicated to the Tox/Pharm Lab and QC functions, as well the availability of Mayo Clinic comprehensive animal facilities to support GVTSR needs. These GVTSR activities must be done using federally mandated practices (Good Laboratory Practices and Good Manufacturing Practices) that require specific personnel training, facilities, and quality control and quality assurance programs. The GVTSR has also developed a strong relationship with the FDA who has federal authority over these activities. The GVTSR can supply 2 of the major components of an IND application: the descriptions of the product manufacturing and safety testing, and the toxicology and pharmacology characterization of the product in appropriate animal models. Of equal importance are the capabilities of the GVTSR to develop large-scale viral production and purification processes to support preclinical studies as well as the manufacture of clinical grade product. The GVTSR works in close partnership with a clinician in the development and regulatory approval of a clinical trial protocol. This comprehensive approach has resulted in MCCC members translating multiple novel virus-based therapies to 13 Phase I and 2 Phase II clinical trials to treat 15 different tumor types.