Thomas J. Bell, PhD Director of Scientific Service

MISSION STATEMENT To serve the research community and to advance research and discovery through the procurement and distribution of organs, tissues and other biospecimens.

Serving more than 5,000 researchers with over 300,000 biospecimens resulting in 2,500 publications NDRI Program Since 1980

Funded by NIH for~30 Consecutive Years NIH Office of the Director DPCPSI NCI OD NIDDK NIAID NEI ORDR NHGRI NIMH NHLBI NIAMS

Corporate Funding Pharma/biotech industry Provided tissues to > 330 for-profit entities in last 5 years Special Initiative Funding Cystic Fibrosis Foundation Collaboration PA State Formula Grant Funding Joslin Medalist Project Additional Funding

Flow of Biospecimens to Scientists NDRI Team 24/7 ~41,000 Offered Tissues Nationally ~3,700 Diseased Tissues 82 Types ~1,311 Tissues Banked ~13,003 Tissues Matched & Placed

NDRI Researchers ~700 USA Researchers Hawaii 1 Alabama 2 Arkansas 4 California 109 Arizona 3 Colorado 2 Connecticut 10 DC 1 New Jersey 16 Delaware 3 Florida 13 Georgia 10 Indiana 8 Illinois 20 Iowa 3 Kansas 3 Kentuck y 4 Louisiana 4 Mass. 77 Maryland 57 Maine 1 Michigan 11 Minnesot a 13 Missouri 8 Montana 1 North Carolina 16 Nebraska 4 New Hampshir e 1 New Mexico 2 New York 50 Ohio 27 Oklahoma 3 Oregon 4 Pennsylvania 51 Rhode Island 2 South Carolina 4 Tennesse e 10 Texas 27 Utah 2 Virginia 10 Vermont 1 Washington 13 Wisconsin 10 Miss. 1 West Virginia 1

NDRI Global Researchers 101 International Researchers 18 Countries 2012 Australia 5 Austria 1 Belgium 2 Canada 37 Denmark 1 Finland 1 France 3 Germany 5 India 1 Ireland 1 Israel 4 Italy 2 Japan 3 Netherlands 1 Scotland 1 Switzerland 4 Taiwan 1 United Kingdom 27 United States 640

NDRI National Networks- 240 Sites 56 Organ Procurement Organizations 37 Eye Banks 23 Tissue Banks 18 Remote Sites 60 Recovery Personnel 46 Hospital Donor Sites

Researcher Application Process 1. Application - IRB document - Project synopsis - CV/Biosketch - Signed applicant’s agreement 2. Internal Feasibility Committee Review 3. External Feasibility Committee Review 4. Customized protocol development - Researcher-specified parameters

NDRI program approved by UPenn IRB Research – unrestricted Permission to use: - For profit - Therapeutics or diagnostics - Genome sequencing Potential access to complete health record Consent and Authorization

Researchers Diversity Digestive Medical Devices Neuroscience Ocular Endocrine Cell Biology Cardiovascular Immunology Rare Disease Musculoskeletal Renal Reproductive Respiratory Blood Cancer Dorsal Root Ganglia

~120 Peer-reviewed Publications - Cell - PNAS - Molecular Cell ~849 authors ~89 research centers/ institutes Publications Diversity

Low-PMI Biospecimens Normal and Diseased– Common and Rare High Quality Non-transplantable Organs Cancer Tumors/Normal Adjacent Tissues Cord Blood/Placenta/Umbilical Cord HIV+ Tissues Biospecimen Diversity

Source Diversity* Standard Requests Ocular Tissue Transplant Organs Autopsy Specimens Surgical Specimens Project-specific Requests Specialized Sourcing Special Initiatives Eye Slide Program Sympathetic and Dorsal Root Ganglia *All Source Partners are AATB or AOPO Accredited

Pre- and post-opt blood and fluid samples Medical questionnaire and chart review Rapid preservation intervals(<1 hour) Rapid delivery intervals (<24 hours) Musculoskeletal sample highlight s

Two new drugs developed in collaboration with the Cystic Fibrosis Foundation and Vertex Pharmaceuticals NDRI provided 166 CF lungs for R&D Program  Kalydeco FDA approved, and  VX809/Kalydeco in Clinical Trials “Almost every primary cell used in the Vertex discovery was from an NDRI lung” Chris Penland PhD Director of Research, Cystic Fibrosis Foundation Bench to Bedside Success

 Project-driven service to scientists  Nationwide tissue source sites  Worldwide distribution capabilities  Diseased and normal biospecimens available from multiple donor types  SOPs in place for consent, recovery, & distribution to academic and corporate scientists NDRI Summary

Please stop booth #421! Thomas Bell, PhD Director of Scientific Services NDRI Mike Salvatore Director of Site Management NDRI Thank you