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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

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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

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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

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Association Franaise contre les Myopathies (France) Raised > 1.5B euros since the start of Tlthon 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

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

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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

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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

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

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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

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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 Childrens Medical Research Foundation, Inc., Team Sanfilippo, Stop Sanfilippo (Spain) and Fondation Sanfilippo (Switzerland) do not distribute

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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

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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

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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

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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

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