Clarification of the Scope of Research Projects to be Supported by PA-13-283 "NEI Audacious Goals Initiative in Vision Research High Priority Research Area: Molecular Therapies for Eye Disease (R01)"

Notice Number: NOT-EY-13-003

Release Date: August 14, 2013

PA-13-283

National Eye Institute (NEI)

The purpose of this Notice is to clarify aspects of the scientific scope of Funding Opportunity Announcement (FOA) PA-13-283, entitled "NEI Audacious Goals Initiative in Vision Research High Priority Research Area: Molecular Therapies for Eye Disease (R01)."

The following text is hereby added to replace the second sentence in Part I. under "Funding Opportunity Purpose":

The purpose of this Funding Opportunity Announcement is to encourage submission of new, innovative projects directed at exploring this area by developing treatments to restore sight through: 1) the control, modification, and delivery of genetic information; 2) the development and/or application of small molecules with high specificity for a particular target; or 3) optogenetic approaches.

And the following text is hereby added to replace the material following the first paragraph in Part 2. Section I. Funding Opportunity Description:

This is a propitious time to stimulate development of molecular treatments. The genetic and cellular bases of many eye diseases are rapidly becoming understood. Proof of concept has been demonstrated for ocular gene therapy. New and promising technologies are emerging for precise in vivo gene correction. And the burgeoning field of optogenetics has created photosensitive reagents for manipulating the physiology of specific cells for research and ultimately for therapy.

Applications submitted for this FOA should be innovative, exploratory, and new. They may be directed to any tissue(s) of the visual system. Applications may include, but are not limited to, studies:

• exploring in vivo gene edition and correction tools for the visual system;
• defining unique markers on a large scale for specific ocular cell types;
• developing controllable delivery systems with variable cargo capacities;
• improving gene therapy approaches to treated inherited or acquired eye disorders;
• developing new therapeutic small molecules using structure-based or computer-aided design; and
• developing new classes of photoreactivatable molecules.

Highest priority for support under this FOA will be for pre-clinical and in vivo animal studies. The NEI typically only supports interventional human studies using the U10 mechanism.

All other aspects of the FOA remain unchanged.

Please direct all inquiries to:

Houmam Araj, Director, Lens and Cataract Program
National Eye Institute
Telephone: 301-451-2020
Email: arajh@mail.nih.gov
Website: www.nei.nih.gov