Regulatory Core
监管核心
基本信息
- 批准号:10668165
- 负责人:
- 金额:$ 22.97万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2023
- 资助国家:美国
- 起止时间:2023-05-16 至 2028-04-30
- 项目状态:未结题
- 来源:
- 关键词:AddressAnimal ModelAnimalsAuthorization documentationBiological ProductsCell TherapyCellular AssayClinical TrialsClinical assessmentsClustered Regularly Interspaced Short Palindromic RepeatsCollaborationsCommunicationConsensusDataDevelopmentDocumentationElementsEligibility DeterminationEnsureFeedbackFoundationsGenerationsGenomeGoalsHumanImmune responseIndividualInheritedKnowledgeMeasuresMedicalMonitorOutcomePharmacologic SubstancePreclinical TestingRare DiseasesRecommendationRegenerative MedicineRegulatory AffairsReportingResearch Project GrantsResourcesRetinaRiskSafetySomatic CellStandardizationStrategic PlanningSystemTechnologyTestingTherapeuticToxicologyTranslational ResearchVisionWorkauthorityclinical translationexperiencefirst-in-humangene therapygenome editinghuman pluripotent stem cellimprovedmeetingspre-Investigational New Drug meetingpre-clinicalpreclinical developmentproduct developmentprogramsresearch clinical testingrisk mitigationsmall moleculestem cell modelsuccesssynergismtherapeutic developmenttherapy developmenttimeline
项目摘要
PROJECT SUMMARY/ABSTRACT - REGULATORY CORE
The objectives of this Core are to develop appropriate project-specific regulatory strategies, define project
milestones relative to the critical regulatory issues, and support execution of those strategies to meet the requisite
milestones. Genome editing technologies have several unique regulatory challenges within a rapidly changing
regulatory landscape. While the FDA provides resources to guide preclinical development, these materials
generally require a degree of applicable experience for effective and appropriate interpretation and use. The
Core staff’s substantial development experience, spanning discovery through clinical evaluation of advanced
therapeutics like cell and gene therapies, is integral to project success. The Core will leverage the Forward BIO
Institute’s Catapult Program, which employs a milestone-driven approach to support regulatory-informed
decisions and improve the pace and quality of preclinical development. Catapult focuses on navigating the
regulatory landscape during preclinical development and provides a workflow with an actionable toolkit to identify
and develop measures to address risk. The Core’s Tasks are: 1) identify critical regulatory-related deficiencies,
2) develop a comprehensive project-specific technology maturation pipeline in collaboration with the other Cores
and individual Project teams, 3) monitor progress related to regulatory milestones, and 4) develop documentation
needed to advance a therapy towards IND submission. Successful completion of these Tasks will bridge critical
knowledge gaps, mitigate risk, address regulatory concerns, and facilitate effective communication with the FDA.
Outcomes include a regulatory plan that delineates specific actions, identification of applicable FDA Expedited
Programs, and generation of documents including the INTERACT and pre-IND briefing packages and the IND
submission. The Regulatory Core will capitalize on the CRISPR Vision Program’s unique opportunity to advance
clinical translation of these modular editing and delivery systems by standardizing preclinical testing approaches,
including use of human pluripotent stem cell-based models, and by recognizing the opportunities afforded to
academic translational science by the recent FDA guidance on gene therapy for rare disease. This is possible
due to the high degree of regulatory synergy that exists between the Projects and Cores, in particular safety
aspects such as toxicology, immune response, and on/off-target effects. Numerous elements of one Project
directly inform development of the other, and sequential FDA engagement enables Projects 2 and 3 to build on
the regulatory foundation established from the feedback FDA provides to Project 1. The synergy with the Human
Cell Assay and Large Animal Cores is even more pronounced, as no suitable animal model exists to evaluate
safety for these and other somatic cell genome editors. Furthermore, a modular regulatory approach leverages
the regulatory commonalities and clarifies the unique considerations, serving to expedite the path to clinical
assessment of nonviral genomic editing technologies for rare diseases and the field as a whole.
项目摘要/摘要-监管核心
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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Cathy Rasmussen其他文献
Cathy Rasmussen的其他文献
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{{ truncateString('Cathy Rasmussen', 18)}}的其他基金
StrataScreenSENS, Skin Sensitizer Detection in Luminescent 3D Human Skin Models
StrataScreenSENS,发光 3D 人体皮肤模型中的皮肤致敏剂检测
- 批准号:
8393318 - 财政年份:2012
- 资助金额:
$ 22.97万 - 项目类别:
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