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2. Disease affecting < 1/2.000 citizens (EU) Disease affecting < 200.000 citizens (US) > 7.000 different orphan diseases < 500 drugs approved for orphan diseases (2014) 6.500+ orphan diseases have no cure 350M patients worldwide (60M US+EU) A huge therapeutic need & opportunity do not distribute

3. Genzyme (US) Alexion Pharmaceuticals (US) BioMarin Pharmaceutical (US) Orphan Europe (FR) SOBI (SE) 600+ companies involved (WW) Drug development attrition rate: similar to non-orphan Death Valley Low awareness & poor understanding Low prevalence rates Niche markets Despite incentives, VCs reluctance do not distribute

4. Individual patients initiatives Patients organizations emergence Link with scientists and clinicians Link with industry Patients lobby politicians & governments Patients and their relatives are stakeholders do not distribute

5. Association Française contre les Myopathies (France) Raised > 1.5B euros since the start of Téléthon Donations go to patients’ assistance, research and preclinical/clinical trials > 1B euros invested in R&D Started a 50M euros VC fund (with CDC) Funds start-ups do not distribute

6. RDH12 Fund for Sight (US) 2010: four families met at a Leber’s Congenital Amaurosis scientific meeting Raised >1M US$ Currently funding US scientists developing a RDH12-targeting gene therapy do not distribute

7. ALS Fund (NL) 3 Amyotrophic Lateral Sclerosis patients gathered, started and funded Treeway, a biotech developing ALS cures, and Project MinE, a genetic research project 2014: launch of the ALS Fund, which aims to collect 100M Euros to fund ALS cures do not distribute

8. Barth Syndrome Foundation (US) Started in 2000 during an International Barth Syndrome Conference Funding BEZALIP (bezafibrate) repurposing with NHLBI Orphan Designation Holder (2013) do not distribute

9. AB Science (FR) Formed in 2001 to develop a cure for Mastocytosis Association Française pour les Initiatives de Recherche sur le Mastocyte et les Mastocytoses funded 650 patients genome sequencing do not distribute

10. Solid Ventures (US) Ilan Ganot, a JPMorgan banker, starts Solid Ventures following his 3-year old son Duchenne diagnosis Solid Ventures raised 17M US$ in January 2014 Solid Ventures is focused on acquiring and developing disease-modifying therapies for Duchenne Muscular Dystrophy do not distribute

11. Abeona Therapeutics (US) Formed in early 2013 to develop gene therapies for SanFilippo A and B Raised 750K US$ in a seed round in December 2013 Investors: The Children’s Medical Research Foundation, Inc., Team Sanfilippo, Stop Sanfilippo (Spain) and Fondation Sanfilippo (Switzerland) do not distribute

12. Targeted projects: early-stage (lead optimization, IND-enabling preclinical, Phase I) orphan drug candidates Targeted investors: « Patient-Centric » investors (ie. patients, families and friends) do not distribute

13. Early-stage projects (late discovery – IND- enabling studies) = Death Valley Highest risk level 10.000 preclinical compounds = 1 marketed (not orphan-specific, but still applies) Who wants to invest here? do not distribute

14. « Patient-Centric » investors = highly motivated potential investors Eagerly looking for a therapeutic return Financial return expected, but secondary Who may invest: patients, families, relatives + patients associations Snowball effect expected: 1 patient = 2 parents = 4 grand-parents = siblings = friends do not distribute

15. Early-stage, orphan drugs projects sourcing (worldwide deal-flow) Projects assessment (Go, No-Go, Go-If) Patients associations targeting International, syndicated equity- crowdfunding campaigns strategy design Equity-crowdfunding platforms selection Investor deck material production Social media buzz-making (key to success) do not distribute

17. Contact: Christian Girard christian@abcrowdfunding.com +33 (0)6 67 26 60 92 do not distribute