The National Eye Institute's (NEI) budget for fiscal year 2016 was $695 million, which was used to fund research projects, training, clinical trials, and small business innovation grants. Major initiatives included regenerating neurons and neural connections in the eye through the Audacious Goals Initiative, contributions to the BRAIN Initiative and Precision Medicine Initiative. The NEI supports collaborative research projects, small business grants, and conducts intramural research including stem cell therapy and gene therapy clinical trials. Growing the vision research field through training and diversity programs is also a priority.

1. Ophthalmic Innovation 2015
“A View from the NEI”

2. Ophthalmic Innovation 2015:
View from the National Eye Institute
Paul A. Sieving, MD, PhD
Director, NEI, NIH

3. 2%
3%
11%
9%
6%
4%
65%
FY 2016 Budget Mechanisms
Research Training
Resrch Mgmt& Support
Intramural Research
Other Research
R&D Contracts
Research Centers
Research Projects Grants
NEI Budget in FY2016: $695 million
• NIH budget approximately $30 billion.
• NEI Research Project Grants: The biggest part of NEI budget.
• Non-diluting funding:
Money comes free, and
Grantee keeps the IP (Bayh-Dole Act).

4. Major Initiatives at NEI & NIH.
NEI Audacious Goals Initiative:
“Regenerate neurons & neural connections in the eye & visual
system.”
• Important for AMD, RP, glaucoma & other retinal diseases.
• Model for regenerative therapy beyond vision: CNS & spinal
cord.
NIH BRAIN Initiative - 2014:
The vision research community is dominating BRAIN projects.
NIH Precision Medicine Initiative – 2015:
• Medical treatments of the future.
• More than 600 genes can cause eye diseases.
• NEI is on course to implement biology
to develop novel medical treatments.

5. Collaborative research projects – currently budgeting $11M/year
Make resources available across disciplines to address scientific and
technical questions beyond the capabilities of any one research group.
• $2M/yr for 5 yrs
• Proof of concept  Preclinical development  IND/IDE submission 
 possible clinical trial support for trials.
• Leber Congenital Amaurosis (LCA) Gene Therapy
 NEI kicked off this research, using R01/R24 grant funding.
 Proof of Concept -- Demonstrated restoration of functional vision
and improved sensitivity in vision-limited subjects from RPE65
gene mutations
 15 years to get to Phase 3 clinical trial for registration.
R24 Translational Research Grants
Program Director: Neeraj Agarwal, PhD. agarwalnee@nei.nih.gov

6. Enable small companies to convert innovative ideas into ophthalmic
drugs, medical devices or assistive technologies that help people.
• NEI commits nearly $20 M/year in funding about 50 small companies.
• Regulatory Assistance Program provides support in seeking FDA
approvals.
Among projects receiving SBIR support in the past year:
• VisionQuest Biomedical, LLC: Low-priced, portable retinal camera
for large scale eye screening at sustainable cost.
• Photoswitch Biosciences: Small‐molecule therapeutic photo-
switch channel to restore vision in retinal degenerative disease.
• Novoron Bioscience, Inc: LRP1 antagonist receptor associated
protein for therapeutic regeneration of optic nerve after axon injury.
Small Business Grants (SBIR & STTR)
Program Director: Jerry Wujek, PhD, wujekjer@nei.nih.gov

7. Intramural Research at NEI (snippets)
iPSC Stem Cell Therapy Development
• Use iPS cells to create RPE patch to treat AMD.
• A major NEI intramural basic to clinical initiative.
• Stem cells into human subjects in 2017/2018.
• A leading NIH program for neural transplantation.
Human Ocular Gene Therapy
X-Linked Retinoschisis (XLRS): Phase I / IIa.
• One of first NIH in vivo gene therapy programs.
• Clinical trials with XLRS patients ongoing.
X-Linked Retinitis Pigmentosa RPGR: Pre-Clinical Phase.
• Murine animal model.
• AAV-based therapeutic rescue.

8. NEI commits nearly $50M in annual clinical trial support
Effective Treatment of Diabetic Macular Edema
• Trial found that Avastin and Lucentis performed similarly to
Eylea for mild vision loss but demonstrated treatment differences
for more significant loss.
• Conducted by NEI DRCR.Net
Extramural Clinical Trial Support
Extended daily eye patching effective at treating
stubborn amblyopia in children
• Trial found that ocular patching can be extended to 6 hours
to treat amblyopia otherwise resistant to therapy.
• Effective for children up to 17 years old.
• Conducted by PEDIG Research Group, supported by
NEI grants EY011751 and FY018810.

9. Growing the Vision Research Talent Pool
NEI trains students, fellows and clinicians
in vision research.
• Fellowship training in basic science and
clinical research engages graduate students,
baccalaureate fellows, postdoc fellows and
clinicians in vision research.
• Diversity in Vision Research and
Ophthalmology (DIVRO) recruits
students from underrepresented groups for
summer internship program.

10. End of Slides
Ophthalmic Innovation 2015:
View from the National Eye Institute.
Paul A. Sieving, MD, PhD.