Key Dates

Related Announcements

• November 9, 2021 - Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional). See NOFO PAR-22-030.

Issued by

National Institute of Neurological Disorders and Stroke (NINDS)

Purpose

The purpose of this notice is to inform applicants of a change to the allowable Appendix Materials for PAR-22-030 "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)". This notice of funding opportunity (NOFO) has been modified to allow the full FDA meeting minutes as an Appendix document. Changes to the NOFO are shown below. This notice is effective for the October 10, 2023 application due date and subsequent due dates.

Currently Reads:

Section IV. Application and Submission Information

PHS 398 Research Plan

Appendix

Only limited Appendix materials are allowed. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

The following modifications apply:

If any FDA meetings have been held, documentation of meeting outcomes, agreements, disagreements, and action items should be summarized and included as an Appendix document. These meetings can include pre-IND meetings and, earlier in development, INitial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT) meetings. Early, nonbinding regulatory advice can be obtained from the FDA through an INTERACT meeting, which can be used to discuss issues such as a product’s early preclinical program, and/or through a pre-IND meeting prior to submission of the IND.

Modified to Read:

Section IV. Application and Submission Information

PHS 398 Research Plan

Appendix

Only limited Appendix materials are allowed. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

The following modifications apply:

If any FDA meetings have been held, documentation of meeting minutes, outcomes, agreements, disagreements, and action items should be included as an Appendix document. These meetings can include pre-IND meetings and, earlier in development, INitial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT) meetings. Early, nonbinding regulatory advice can be obtained from the FDA through an INTERACT meeting, which can be used to discuss issues such as a product’s early preclinical program, and/or through a pre-IND meeting prior to submission of the IND.

All other aspects of this NOFO remain unchanged.

Inquiries

Please direct all inquiries to:

Chris Boshoff, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-1779
Email: chris.boshoff@nih.gov