PROJECT SUMMARY / ABSTRACT The Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats (CASEPR) program comprises three Research Projects (RPs) and three Resource Cores (RCs), which the Administrative Core (AC) will coordinate to advance one RP through IND submission, and the other two RPs through early regulatory review. The AC will provide rigorous, project management and day-to-day administrative program coordination, planning, and evaluation, while developing and ensuring compliance with IND-grade practices for assessing molecular potency, disease-modifying efficacy, and safety. The AC will oversee the total budget, collect invoices, manage payments, and coordinate inter-campus charges. The AC will adopt a collaborative process, working with PIs to develop a shared vision, formulate guidance, provide oversight, ensure compliance, proactively resolve issues, initiate early and frequent FDA contact, and maintain overall continuity. Dedicated AC staff (1 full time equivalent) will develop detailed Gantt charts in close consultation with RP PIs, and will provide industry gold-standard project management support within each RP and across the entire effort. The AC Project Manager (PM) will monitor CASEPR activities daily to remove obstacles, coordinate in-person and virtual meetings, record minutes, manage action items, facilitate communication, manage travel, allocate resources responsive to CASEPR needs, and prepare technical summary and financial reports for leadership. With the goal of ensuring a consistent interface between the RPs and RCs, and leveraging the unique infrastructure and expertise of the IGI Clinical Laboratory, the AC will coordinate the instantiation of a dedicated Laboratory Information Management System (LIMS) that will be used for requisition, accession, and resulting of specimens submitted by RP personnel to RCs. The AC PI and PM will play a central and enabling role in all aspects of ensuring timely advance of RP1 (the trailblazer project) through every stage of regulatory review (INTERACT, pre-IND, IND), and RP2 and RP3 through INTERACT and pre-IND. To this end the AC will work with the RC PIs to ensure timely and consistent representation of key data in study reports for submission to the Agency. Further, leveraging institutional depth in developing experimental therapeutics for neurodegeneration at the UCSF and OHSU, the AC will prepare drafts of key documentation (clinical protocol, investigator’s brochure) required for IND submission. The AC PI will ensure implementation of the research plan and will play a critical role facilitating communication among all program components, monitoring progress across Gantt-specified timelines, milestones, and endpoints, and ensuring effective use and integration of all proposed shared resources. To help create a shared learning, training, and education hub, the AC will facilitate and participate in the learning and dissemination programs of the Transla...