# Core 2: Therapeutic Viral Vector Design and Development Core

> **NIH NIH P50** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2020 · $184,236

## Abstract

Project Summary - Core 2 
The goal of the Therapeutic Viral Vector Design and Development Core (Core2) is to guide viral vector design, 
produce high quality viral vectors, and support Investigator initiated product development for novel vector 
based biologics with the goal of moving them to an IND. Significantly, the services of the Vector D & D Core 
represent a fundamental necessity to the defined and developmental projects within this CORT application as 
well as the larger identified Research Base. The center now holds 6 INDs for AAV gene therapy in various 
muscular dystrophies. We have gained valuable experience through our interactions with the FDA regarding 
the preclinical and toxicology/biodistribution studies required for IND approval. As part of this Core, Dr. 
Rodino-Klapac and her team will provide guidance for each project on the proper design of viral vectors and 
implementation of IND enabling preclinical studies. Understanding the translational pathway facilitates 
coordination between the PI's research team and the Vector Core for the production and testing of rAAV and 
other viral vectors in a manner consistent with FDA expectations at all phases of product development. This 
translates into a uniform vector product as a result of using consistent processes from early pre-clinical preps, 
through tox vector generation and ultimately early phase clinical grade vector produced using phase 
appropriate cGMPs. Through the demonstrated support of the Research Base within the Center for Gene 
Therapy at NCH and outside external collaborations, the NCH Vector Core has garnered such experience. 
The infrastructure support supplied by this P50 mechanism will allow for production of pre-clinical grade 
vectors for the three projects as well as guidance on vector design and preclinical implementation for each 
project in addition to the defined Research Base. Moreover, continued refinement of critical production 
processes and test methods as well as feasibility testing of novel AAV vector modalities will provide greater 
value to research base Investigators by reducing cost and time to completion with better quality and enhanced 
capacity. Our Specific Aims build upon the existing Vector Core infrastructure at NCH and will fulfill the 
primary goal of this CORT application - to accelerate the translation of basic science projects into genetic 
therapies for muscular dystrophies. In summary, direct interaction between the Core and Investigative team 
from each of the three projects will leverage product and regulatory experience to accelerate timelines for 
defined products and provide guidance where it was lacking to enable new laboratories to move proof of 
concept studies rapidly into translational research. The ultimate effect of this aligns perfectly with P50 CORT 
program mission, which includes the development of “tangible products or deliverables” for the “development 
of more effective treatments” for human disease in which ca...

## Key facts

- **NIH application ID:** 10017019
- **Project number:** 5P50AR070604-05
- **Recipient organization:** RESEARCH INST NATIONWIDE CHILDREN'S HOSP
- **Principal Investigator:** Louise R. Rodino-Klapac
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $184,236
- **Award type:** 5
- **Project period:** 2016-09-14 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10017019

## Citation

> US National Institutes of Health, RePORTER application 10017019, Core 2: Therapeutic Viral Vector Design and Development Core (5P50AR070604-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10017019. Licensed CC0.

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