COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ NAME - PDF document

COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ NAME OF THE CEO MISSION Karen AIACH Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A pivotal clinical trial in MPS IIIA is expected to start in 2018, a phase 1-2 clinical trial in GM1 Gangliosidosis in 2019, while we are currently collaborating to define the clinical development path for the treatment of Fragile X syndrome. TECHNOLOGY Lysogene is a gene therapy company using AAV vectors and addresses monogenic diseases. COMPETITION Lysogene is the most advanced company in discovering gene therapies for MPS III A, ADRESS GM1 ganliosidosis and Fragile X; competition in MPS IIIA is including ABEONA (US) and ESTEVE (Private Spain). There is no identified competition other than publicly -funded 18/20 rue Jacques Dulud, research for GM1-Gangliosidosis and gene therapy in Fragile X. 92200 Neuilly-sur-Seine _____________________ ALLIANCES/PARTNERSHIPS EMAIL karen.aiach@lysogene.com University of Massachusetts Medical School Auburn University www.lysogene.com SAHMRI (South Australian Health and Medical Research Institute) University of Strasbourg, SATT Conectus MANAGEMENT TEAM UPCOMING CATALYSTS CEO: Karen Aiach COO: Philippe Mendels-Flandre • Start of pivotal trial in MPS IIIA : H2 2018 • Start of phase 1/2 for LYS-GM101: 2019 CSO: Ralph Laufer • Phase 3 results for MPS IIIA : 2020 CTO: Mark Plavsic • Proof of concept for Fragile X : early 2020 CMO: Sophie Olivier MARKET FIGURES (listed companies) VP of Regulation and Quality Affairs: Sean O’Bryan Revenues 2017: 0 Date of IPO: February 2017 TARGETED Ticker: EPA.LYS (ISIN: FR0013233475) MARKET Exchange: Euronext Currency: Euro ____________ Market cap: 25 millions € Price: 2,08 52-weeks-high: 5,94 € Orphan CNS disease (MPS IIIA, GM1 52-weeks-low: 1,98 € Gangliosidosis and Fragile X) and looking for Average daily volume: 4536 expanding the portfolio. KEY FIGURES € m 2016 2017 Growth (%) LISTED COMPANY Sales 0 0 0 EBIT - - - CREATION DATE Net Income (7,5) (17,8) - 2009 Cash Position 6,3 14,1 -

COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ ANALYST COVERAGE NAME OF THE CEO Reco. Broker Date Target Price Potential (%) Karen AIACH (buy/neutral/h old) Société 24/06/2017 BUY 7 336.54% Générale Gilbert Dupont 13/04/2018 BUY 6 288.46% Average: - - 6,5 312.5% SHAREHOLDERS (percentage) ADRESS Sofinnova Partners: 29.41% 18/20 rue Jacques Dulud, BPI France: 20.62% 92200 Neuilly-sur-Seine Novo: 17.45% _____________________ KGA: 10.37% EMAIL Others: 22.15% karen.aiach@lysogene.com PIPELINE www.lysogene.com MANAGEMENT TEAM CEO: Karen Aiach COO: Philippe Mendels-Flandre CSO: Ralph Laufer CTO: Mark Plavsic CMO: Sophie Olivier VP of Regulation and Quality Affairs: Sean O’Bryan TARGETED MARKET ____________ Rare CNS disease (MPS IIIA, GM1 Gangliosidosis and Fragile X) and looking for expanding the portfolio. LISTED COMPANY CREATION DATE 2009