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From Idea to Impact

Targeted Elastin Restoration Therapy
Lisa Perpall MS MBA eperpal@clemson.edu

Clemson University Innovations

Introduction
• Clemson University
– Top 25 public university – Legacy in bioengineering and biomaterials research

• Clemson University Research Foundation (CURF)
– Bridges research and industry – Access to emerging technology – Access to research resources

Clemson Biomedical Research Resources
Greenville, SC Translational Research From Bench to Bedside
• Education • Incubation

Clemson, SC

• Basic Research • Technology Development (BS, MS, PhD)

Charleston, SC
Bioengineering Alliance •Clinical Research •Research Training

CU-MUSC

Featured Technology
Targeted Elastin Restoration Therapy

Clemson Inventors:
Dr. Naren Vyavahare Aditi Sinha

Background: Elastin
• Elastin provides elasticity and resilience to connective tissue • Degrades with age or disease
– Reduced elasticity in skin – Weakened alveoli in lungs – Weakened blood vessel walls

• Exposed elastin fragments = hallmark of elastin degenerative disorders

(www.celergenswiss.com)

Invention

File: Aditi_vcam0000 DataType: AmplitudeRetrace Date: 2011-10-07 ImageNote: VCAM, 50x diluted on freshly cleaved Mica ImagingMode: AC Mode ScanLines: 512 ScanPoints: 512 ScanRate: 0.50 Hz ScanSize: 5.00 µm Time: 6:46:23 PM

Modified nanoparticles that target elastin fragments & deliver therapeutic agents to stabilize and regenerate elastin

File: Aditi_vcam0000 DataType: ZSensorRetrace Date: 2011-10-07 ImageNote: VCAM, 50x diluted on freshly cleaved Mica PEG-PLA AC nanoparticles with ImagingMode: Mode ScanLines: 512 surface bound antibody ScanPoints: 512 ScanRate: 0.50 Hz ScanSize: 5.00 µm Time: 6:46:23 PM

Application Example: Aneurysm (AAA)
• Elastin degradation causes weakened vessel walls • Abdominal Aortic Aneurysm (AAA)
– 15,000 deaths per year1 – 13th largest cause of death1

• Agents to prevent or treat early stage AAA – Statins, anti-inflammatories, MMP inhibitors, etc.

drug

The Science
• Nanoparticles injected into aneurysmal rats • Calliper IVIS used for real time imaging of NPs in vivo

Control PEG-PLA NP 24h

Elastin Targeting NP 24h

The Science
Aneurysmal aorta imaging @24 hrs
Elastin Targeting NPs 24h

Control PEG-PLA NPs 24h

Targeted sustained drug delivery of agents for prevention, treatment, or regeneration
– MMP inhibitors – Anti-inflammatories – Elastin regeneration compounds (i.e. PGG)

Application Opportunities
Topical, Respiratory, or Injection Delivery COPD
• 3rd leading cause of death2 • 2010 cost of COPD annually $49.9B2 • Treat/prevent loss of elastin in alveoli
(image from: www.beltina.org)

Skin Care – Cosmetic
• Anti-aging segment of facial skin care market $1.2B 20093 • Topical product for skin rejuvenation
1- http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1312219/#B4 2- American Lung Association www.lung.org 3- Frost and Sullivan 2011

(www.celergenswiss.com)