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Company Presentation

Company Presentation. December 2013. Forward Looking Statement.

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Company Presentation

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  1. CompanyPresentation December 2013

  2. Forward Looking Statement This presentation includes certain estimates and other forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, including statements with respect to anticipated operating and financial performance, clinical results, potential partnerships, licensing opportunities and other statements of expectation. Words such as “expects,”“anticipates,”“intends,”“plans,”“believes,”“assumes,”“seeks,”“estimates,”“should” and variations of these words and similar expressions, are intended to identify these forward-looking statements. While we believe these statements are accurate, forward-looking statements are inherently uncertain and we cannot assure you that these expectations will occur and our actual results may be significantly different. These statements by the Company and its management are based on estimates, projections, beliefs and assumptions of management and are not guarantees of future performance. Important factors that could cause actual results to differ from those in the forward-looking statements include the factors described in the Company’s filings with the U.S. Securities and Exchange Commission. The Company disclaims any obligation to update or revise any forward-looking statement based on the occurrence of future events, the receipt of new information, or otherwise.

  3. Biopump™: Personalized Protein Therapeutics • Proprietary tissue-based technology for the sustained production and delivery of therapeutic proteins using ex-vivo gene therapy and the patient's own tissue for the treatment of a wide range of diseases 2

  4. New Management Team Has Proven Track Record of Success • President, Shire Specialty Pharmaceuticals- Responsible for all aspects of the $2.5 billion business e.g.., commercial, R&D, manufacturing, etc. • President, Life Sciences, Safeguard Scientifics – Lead Life Sciences PE team, responsible for both investment and management of portfolio companies • Multiple senior operational leadership positions at Astra Merck/AstraZeneca Mike Cola CEO • Responsible for >20 FDA approvals in multiple therapy areas • Corporate Vice President of Science and Technology, Johnson and Johnson - Multiple senior R&D leadership positions at Astra Merck/Merck KGa/AstraZeneca • Head of R&D, Appletree Partners – Responsible for both investment, overall R&D strategy, and management of portfolio companies in PE fund Garry A Neil, MD Global Head of R&D • Vice-President Commercial Assessment for Shire Specialty Pharmaceuticals - Responsible for both corporate and business unit M&A and white space strategies • Principal, Devon Park Bioventures - Responsible for all aspects of venture investing e.g., negotiation, board governance, financing for $125M Life Sciences VC fund • Associate Principal, McKinsey & Company – Worked on strategic issues for multiple Top 25 pharmaceutical companies; specialized in BD/M&A, and payer/reimbursement issues John H. Leaman, MD CFO

  5. The Biopump Therapeutic System DermaVac Micro-organ #1* #2 Biopump #4 #5 #3 “Gutless” adeno-viral vector Cryo Bank * Description on pg. 6

  6. Biopump Method: Autologous Tissue Therapy Harvest tissue from under patient’s skin via needle biopsy into a sealed cassette (half size of toothpick). Simple procedure with minimal pain. 1. Harvest Process tissue into a drug producing Biopump by controlled transfer of desired gene, using safe “gutless” Adeno vector technology ex-vivo. 2. Process Measure each Biopump’s continuous protein production level – patient’s personalized protein. This allows calibration of therapy. 3. Measure Implant required number of Biopumps under patient’s skin – they heal in place. 4. Implant Increase dose by adding Biopumps. Reduce dose/stop therapy by local ablation or removal - key differentiator from gene therapies. Can also retrieve implants. 5. Titrate

  7. Medgenics Biopump offers unique advantages • Safety of ex-vivo gene therapy: • No systemic exposure to viral vectors • Ability to remove the biopump in the event of a reaction • Dosing flexibility: • Ability to add or removing biopumps to adjust dose • Ability to re-treat • Flexibility of viral vectors • Gutless adenovirus allows very large payload including cytokines, enzymes, monoclonal antibodies, and peptides • Continuous autologous protein production • Native protein more potent than synthetic protein • Potentially less immunogenic • Low cost, fast preclinical proof of concept in tummy tuck and SCID mice • Cost to produce cDNA and vector are low • Preclinical studies have been predictive of performance in humans • Low capital requirements for production: • Relatively small amounts of vector needed • Patient’s tissue is the “bioreactor”

  8. EPODURE and INFRADURE Clinical Programs Have Provided Proof of Concept • Substantial in-vitro and in vivo production of therapeutic proteins • EPODURE and INFRADURE Biopumps have showed sustained protein production of human protein in vitro and in SCID mice • Clear safety and efficacy in human clinical trials • EPODURE and INFRADURE have shown safety and tolerability in >25 patients • Consistent and predictable efficacy response of serum levels of erythropoietin and interferon alpha from the Biopump • New generation Biopump longevity has been significantly extended • Promise of much longer dosing intervals • Fewer Biopumps per patient

  9. New Viral Vector and Depomedrol Have Shown Potential for > 6 months of in vivo Protein Production in SCID mice In vivoSCID Mice Results were Depo-Medrol was Applied to the Implantation Site Every Second Week • SCID mouse models have been historically predictive of human efficacy • Results are significant milestone toward a commercially viable therapeutic product profile

  10. Ideal Target Market for the Optimized BiopumpWould Have the Following Characteristics: Validated Target • Chronic Disease • Established treatment paradigm • Acceptable and Measurable Clinical Endpoints • Clinical differentiation beyond drug delivery must exist • Monogenic (One gene) Gene Defect • High potency protein • Microgram quantities sufficient, e.g., • Cytokine Mediated • Hormone Mediated • Significant reimbursement potential (>$50k/year) • Small sales force ~60 reps • Small clinical trials (~100 patient) for FDA approval Amendable to Single Protein Therapy Orphan-Disease “like” commercialization

  11. Management Priorities/Milestones for Next 12 Months Validate Biopump Platform • Priorities • Explore platform’s ability to show approximately 12 months of persistency with EPODURE including: • Studying the effects of Depomedrol on Biopump survival • “New” gutless Adeno vector with additional stabilization elements • Milestones • Initiation of human EPODURE trial with new viral vector and implantation protocol (1H 2014) • EPODURE trial results including persistency of protein production (2H 2012) • Priorities • Identify and create one or more pre-clinical Biopump models of commercially attractive markets (e.g., orphan disease and niche specialty) • Milestones • Biopump Proof of Concept (POC) data in identified attractive commercial markets • In vitro (1H 2012) • In vivo (SCID mouse/human tummy tuck) (2H 2012) Identify > 1 Attractive Markets For Platform • Priorities • BD should build on company’s current expertise: Exogenous transduction of autologous tissues to produce therapeutic proteins • Examples of would include: • Technologies to produce “larger” protein factories (e.g., liver micro-organs that could be transduced to produce larger amounts of protein) • Marketed cell-based therapies that secrete therapeutic proteins Leverage BD to Expand on Company’s Strategy

  12. Key Takeaways • Experienced new management team and Chairman • Strong Biopump value proposition: Potentially better, safer, less costly • 12 month priorities include: 1) Validating Biopump platform; 2) Identifying several attractive markets for technology; 3) Business development to expand the company’s strategy • 2nd generation viral vector and improved implantation technique showing significant in vivo benefit; will be utilized in 2014 human trials • Attractive markets for the Biopump include validated targets, amenable to single gene therapies, which are “orphan-like” in commercialization • Strong balance sheet; $26+ million in cash and no debt • Potential 2014 near-term value-creating milestones 1 2 3 4 5 6 7

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