1. North Carolina Federal Advanced Technologies Symposium
May 9, 2013
Biotechnologies Panel
Hosted by:
Office of Senator Richard Burr
NC Military Business Center
NC Military Foundation
Institute for Defense & Business
University of North Carolina System
Reception Sponsor:
Bronze Sponsor:
2. Portable Nanodevice for mTBI
You simply need a drop of blood to diagnosis mTBI
Collaborators:
Dr. Ralf Lenigk
GE Global Research
Senior Microfluidic Specialist
Dr. Hiram Brownell
Boston College University
3. Protein Normal Values Cut-off Values
found in TBI
Time Course Biomarker
indication
S100B 0.2 ng/ml 1.13ng/mL;
~1fmol/µL
6 hours after injury
(Savola et al., 2004)
Breakdown of
blood-brain barrier
GFAP 0.49 μg/L 1.5 ng/mL;
~27 amol/µL
24 hours after injury
(Vos et al., 2004)
Damage to glial cells
NSE <12.5 ng/mL
(Mondello et al.,
2011)
21.7 ng/mL;
265 amol/µL
6 hours after injury
(Berger et al., 2007a)
Breakdown of
blood-brain barrier
UCH-L1 2.7 ng/mL(Papa et
al., 2010)
44.2 ng/mL (+/-
7.9) in severe TBI
group; ~1.8
fmol/µL
6 hours after injury (Svetlov et
al., 2010)
Cell death
C-Tau
Proteins
Undetectable (< 12
pg/mL) (Berger et
al., 2007b)
1600
ng/mL;~140
fmol/µL
6 hours after injury
(Liliang et al., 2010)
Neuronal
degeneration
C-Reactive
Protein
< 1 µg/ml 50 µg/ml; ~ 33
pmol/µL
24 hours after injury
(Hergenroeder et al.,2008)
Inflammation
Blood-Based Biomarkers for TBI
4. Enhanced SPRi Detection of ssDNA
to 1fM Concentration
Malic, L.; Sandros, MG; Tabrizian M Anal Chem 2011, 83, 5222–5229.* HDFT- Heptadecafluoro-1-decanethiol
5. A brief primer on aptamers
SELEX
↓
MiSeq sequencing
↓
Analysis of sequences
obtained
http://www.nanotemper-technologies.com/applications/list-all-
applications/article/aptamer-interaction-with-thrombin/
7. Environmental
Biotechnology
A safe, effective and lower cost cleanup for persistent organic
chemical pollutants in soil, sediment and groundwater
Exceeds mandates of Executive Order 13423
Strengthening Federal Environmental, Energy, and Transportation Management
9. Why do some chemical pollutants
degrade naturally while other
chemical pollutants persist for
decades or longer?
10. The Answer
Persistent chemical pollutants prevent bacterial genes from
expressing messenger proteins (genetic impairment)
Messenger proteins are essential for bacterial enzyme
secretion
Bacteria secrete enzymes to reduce all organic material as a
food source for growth & reproduction
Impaired genes → no messenger proteins →no enzyme
secretion →no enzymatic reduction in target pollutants
11. Environmental Gene Signaling
Gene signaling is the capacity to manipulate gene
performance (enzyme secretion) through the insertion of
deficient proteins
Analog proteins restore gene function and restore the
natural bacteria’s capacity to secrete reductive enzymes
Environmental cleanup can be completed for a fraction of
conventional cleanup costs
Environmental biotech can reduce DODs $100 billion
environmental liability by 50% to 70%
12. Lifespan: Reproduce as often as every 20 minutes. Up to 1,000 trillion per square
meter (5 billion in one tablespoon of soil).
13. The Factor Biotechnology is not genetic engineering or creating a
Super-bug. Treatments simply restore an innate function that is
disrupted in the presence of these man-made chemical compounds
21. U.S. Army Research, Development and
Engineering Command
Dr. Stephanie McElhinny
Program Manager, Biochemistry
Army Research Office
stephanie.a.mcelhinny.civ@mail.mil
919-549-4240
Biotechnology Research Opportunities
in the Department of Defense
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
22. Utilize the vast intellectual capital of our nation to:
Create and Exploit Scientific Opportunities for
Revolutionary New Army Capabilities
Drive Science to Develop Solutions to
Existing Army Technology Needs
Accelerate Transition of Basic Research
Leverage S&T From Outside Sources
Create and Strengthen University, Industry,
Government Partnerships
Unbiased expert assessments for HQs
Educate and Train the Future S&E
Workforce for the Army
Prevent Technological Surprise Chemistry
Computing and
Info Science
Electronics
Environmental
Life Sciences
Materials
Mathematics
Mechanics
Network Science
Physics
Research Domains
• 270+ Institutes of Higher Learning
• 1,121 individual Investigators
• 47 Research Centers
Research Funding
by State
More Funding
Less Funding
Army Research Office
Goals
www.aro.army.mil
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
23. Biotechnology Research
at the Army Research Office
www.aro.army.mil
Biotic/Abiotic interface between
nanostructures and biomolecules to
generate advanced materials
Reactive Chemical Systems
Genetics, genomics, epigenetics,
systems biology to optimize
performance and enhance protection
Genetics
Biomolecular assembly, biomolecular
specificity and regulation, biological
fibers, adhesives, composites
Biochemistry
Mechanisms of microbial adaptation
and survival, systems and synthetic
biology of microbes
Microbiology
Neural codes of human perception
and behavior, neuronal computation,
optimized human-machine interfaces
Neuroscience
Surface and interface engineering
(e.g., bio/organic/semiconductor) to
provide multifunctional capabilities
Materials Design
Bio-inspired mathematical methods
underlying automated sensing and
target tracking
Information Processing & Fusion
Force-activated materials, lightweight
ballistic protection, adhesives,
nondestructive material evaluation
Mechanical Behavior of Materials
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
24. Department of Defense
Biotechnology Funding Agencies
www.onr.navy.mil
www.afosr.af.mil• Synthetic Biology of living organisms
• Biomaterials – protein-based materials,
biomimetic materials, adhesives
• Bionanotechnology – biomimetic systems,
bio-directed synthesis/patterning of
nanomaterials
• Bioenergy Harvesting – Microbial Fuel Cells
• Metabolic Engineering for chemical
biosynthesis
• Marine Anti-Biofouling/Fouling Release
Coatings
• Autonomous In Vivo Medical Devices
• Biomimetics – mimic and control organism
sensors and sensor denial systems
(camouflage), natural chromophores and
photoluminescent materials
• Biomaterials – using organisms to
synthesize materials, mimicking natural
materials, using existing
materials/organisms as novel materials
• Biotic/Abiotic Interfaces – bio-directed
assembly to create new catalysts and
templates for optical or electronic materials
Office of Naval Research Air Force Office of
Scientific Research
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
25. Department of Defense
Biotechnology Funding Agencies
www.darpa.mil
• Biowarfare defense – advanced diagnostics,
decontamination, medical therapies
• Tactical Biomedical Technologies – mobile
trauma stabilization, novel therapeutics,
generation and storage of blood products
• Restorative Biomedical Technologies – restore
complex tissues after traumatic injury, neural-
controlled prostheses
• Bio-inspired Platforms and Systems – mimic
locomotion and chemical/visual/aural sensing
• Microphysiological Systems – organs-on-chip to
mimic human physiological systems
• In Vivo Nanoplatforms for diagnostics and
therapeutics
• Living Foundries – create engineering
framework for synthetic biology
www.dtra.mil
• Reduce, eliminate, counter, mitigate
weapons of mass destruction
• WMD sensing and recognition
• Threat containment, filtering, shielding
• Decontamination
• Forensics
• Neutralization of CBRNE materials
Defense Advanced
Research Projects Agency
Defense Threat
Reduction Agency
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
26. SBIR STTR
Phase I:
Project feasibility
up to 10 months
up to $150,000
6 months
up to $150,000
Phase II:
Project development
to Prototype
2 years
up to $1,000,000
2 years
up to $1,000,000
Phase III:
Commercialization
Commercialize, with non-SBIR/non-
STTR funds, the technology in
military and/or private sector markets
www.armysbir.army.mil
www.acq.osd.mil/osbp/sbir
DoD and Army
SBIR/STTR Programs
DoD FY13 SBIR/STTR Solicitation Schedule
Solicitation Pre-Release Open Close
DoD SBIR 2013.2 24 Apr 2013 24 May 2013 26 Jun 2013
DoD SBIR 2013.3 26 Jul 2013 26 Aug 2013 25 Sep 2013
DoD STTR 2013.B 26 Jul 2013 26 Aug 2013 25 Sep 2013
UNCLASSIFIED // APPROVED FOR PUBLIC RELEASE
27. 32
NC Federal Advanced Technology Symposium
5/15/2013
Jay Strum, PhD
President
Development of G1T28-1 as a Radiomitigant for
the Treatment of Acute Radiation Sickness
28. G1’s PHARMACOQUIESCENCE™ (PQ™) Approach
5/15/2013
PharmacoQuiescence™
Mechanism
Inhibition of CDK4/6 to
modulate the cell cycle
for multiple uses:
Chemo-protection
Renal-protection
Radiomitigation
Niche antineoplastic
S
G2
G0
G1
M
Specifically, cell cycle
traversal from G1 to S is
arrested.
33
29. G1T28-1 protects hematopoietic stem and progenitor cells (HSPC)
5/15/2013
34
UNMETUNMET*
Stem
Cell
Multi-potent
Progenitors
Common
Myeloid
Progenitor
Common
Lymphoid
Progenitor
Granulocyte/
Monocyte
Progenitor
Megakaryocyte/
Erythroid
Progenitor
Erythrocytes Platelets Granulocytes Macrophages T-cells B-cells
CDK4/6 dependent HSPC’s
CDK4/6 independent cells
Quadrilineage protection of blood cells from radiation exposure
30. Radiomitigation (Federally funded)
5/15/2013
Radiomitigation
o Phase II SBIR grant
funding through NIAID
o Lead molecule G1T28-1
o $0.5B+ mkt. opportunity
Grants $Amount Status
NIAID
Phase I
$600K Complete
NIAID
Phase II
$3M Ongoing
BARDA
POC
$4.5M
Under
review
A single, oral dose of G1T28-1 12 hours after ionizing
radiation significantly improves survival
Development funded by grants (BARDA app. to be submitted May 29)
35
31. RADIOMITIGATION PATH MAY SPEED APPROVAL
5/15/2013
36
• Acute Radiation Sickness (Hematologic Syndrome)
• U.S. Government stockpile
• 200,000+ doses of radiation mitigant, $500 million market
• Other markets: Japan, Israel, etc.
• U.S. military for soldiers
• Civilian Populations
• Significant US government financing (NIAID, BARDA)
1. KNOWN MECHANISM
OF ACTION
2. NON-HUMAN PRIMATE
EFFICACY STUDY
Performed in
government-sponsored
facility
3. PHASE 1 SAFETY
In this case, CDK4/6
inhibition
Timeline to approval: 24+ months
Approval Based on FDA Animal Rule – 3 Criterion:
Use existing trial or
conduct new trial
42. N O R T H C A R O L I N A B I O T E C H N O L O G Y C E N T E R
Bio Technology Panel Session
Moderator: Mary Beth Thomas, PhD
43. N O R T H C A R O L I N A B I O T E C H N O L O G Y C E N T E R
North Carolina
Biotechnology Center
Supporting biotechnology research, business and
education statewide since 1984
44. N O R T H C A R O L I N A B I O T E C H N O L O G Y C E N T E R
45. Purpose of the conference:
- To address the future advanced technology needs of
the Dept. of Defense and other federal agencies.
- Projects that are:
• Mission-driven
• Solution-oriented
• Application-focused
N O R T H C A R O L I N A B I O T E C H N O L O G Y C E N T E R
Why Biotech?
Biotech can deliver this across a highly diverse
spectrum of interest areas.
46. • “The Need” – Dr. Stephanie McElhinny,
Army Research Office (ARO)
• “The Idea” – Dr. Vince Henrich, UNC-Greensboro/Joint
School for Nanosci & NanoEngineering
• “The Development Engine” – Industry partners
– Brandon Conover Bennett Aerospace
– David Ellis SmartGene
– Christopher Young Biotech Restorations
– Dr. Jay Strum G1 Therapeutics
N O R T H C A R O L I N A B I O T E C H N O L O G Y C E N T E R
Biotech Panelists: