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

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)

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ monograph.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ sciAawards.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ conferencePapers.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ patent.updateTime }}

Cathy Rasmussen其他文献

Cathy Rasmussen的其他文献

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

{{ 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:

相似海外基金

Quantification of Neurovasculature Changes in a Post-Hemorrhagic Stroke Animal-Model
出血性中风后动物模型中神经血管变化的量化
  • 批准号:
    495434
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
Small animal model for evaluating the impacts of cleft lip repairing scar on craniofacial growth and development
评价唇裂修复疤痕对颅面生长发育影响的小动物模型
  • 批准号:
    10642519
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
Bioactive Injectable Cell Scaffold for Meniscus Injury Repair in a Large Animal Model
用于大型动物模型半月板损伤修复的生物活性可注射细胞支架
  • 批准号:
    10586596
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
A Comparison of Treatment Strategies for Recovery of Swallow and Swallow-Respiratory Coupling Following a Prolonged Liquid Diet in a Young Animal Model
幼年动物模型中长期流质饮食后吞咽恢复和吞咽呼吸耦合治疗策略的比较
  • 批准号:
    10590479
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
Diurnal grass rats as a novel animal model of seasonal affective disorder
昼夜草鼠作为季节性情感障碍的新型动物模型
  • 批准号:
    23K06011
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Longitudinal Ocular Changes in Naturally Occurring Glaucoma Animal Model
自然发生的青光眼动物模型的纵向眼部变化
  • 批准号:
    10682117
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
A whole animal model for investigation of ingested nanoplastic mixtures and effects on genomic integrity and health
用于研究摄入的纳米塑料混合物及其对基因组完整性和健康影响的整体动物模型
  • 批准号:
    10708517
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
A Novel Large Animal Model for Studying the Developmental Potential and Function of LGR5 Stem Cells in Vivo and in Vitro
用于研究 LGR5 干细胞体内外发育潜力和功能的新型大型动物模型
  • 批准号:
    10575566
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
Elucidating the pathogenesis of a novel animal model mimicking chronic entrapment neuropathy
阐明模拟慢性卡压性神经病的新型动物模型的发病机制
  • 批准号:
    23K15696
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
    Grant-in-Aid for Early-Career Scientists
The effect of anti-oxidant on swallowing function in an animal model of dysphagia
抗氧化剂对吞咽困难动物模型吞咽功能的影响
  • 批准号:
    23K15867
  • 财政年份:
    2023
  • 资助金额:
    $ 22.97万
  • 项目类别:
    Grant-in-Aid for Early-Career Scientists
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了