PART 01: EXECUTIVE SUMMARY PART 02: SCOPE OF THE REPORT • 2.1 Preface • 2.2 Preface • 2.3 Currency conversion rates for US$ PART 03: MARKET LANDSCAPE • Market ecosystem • Market characteristics • Market segmentation analysis PART 04: MARKET SIZING • Market definition • Market sizing 2018 • Market size and forecast 2018-2023 PART 05: FIVE FORCES ANALYSIS • Bargaining power of buyers • Bargaining power of suppliers • Threat of new entrants • Threat of substitutes • Threat of rivalry • Market condition PART 06: PIPELINE PART 07: MARKET SEGMENTATION BY INDICATION • Market segmentation by indication • Comparison by indication • SMA - Market size and forecast 2018-2023 • Others - Market size and forecast 2018-2023 • Market opportunity by indication PART 08: CUSTOMER LANDSCAPE PART 09: GEOGRAPHIC LANDSCAPE • Geographic segmentation • Geographic comparison • North America - Market size and forecast 2018-2023 • Europe - Market size and forecast 2018-2023 • Asia - Market size and forecast 2018-2023 • ROW - Market size and forecast 2018-2023 • Key leading countries • Market opportunity PART 10: DECISION FRAMEWORK PART 11: DRIVERS AND CHALLENGES • Market drivers • Market challenges PART 12: MARKET TRENDS PART 13: VENDOR LANDSCAPE • Overview • Landscape disruption • Competitive scenario PART 14: VENDOR ANALYSIS • Vendors covered • Vendor classification • Market positioning of vendors • Biogen • bluebird bio, Inc. • Novartis AG • Pfizer Inc. • Voyager Therapeutics PART 15: APPENDIX • Research methodology • List of abbreviations PART 16: EXPLORE TECHNAVIO   Exhibit 01: Global pharmaceuticals market Exhibit 02: Segments of global pharmaceuticals market Exhibit 03: Market characteristics Exhibit 04: Market segments Exhibit 05: Market definition - Inclusions and exclusions checklist Exhibit 06: Market size 2018 Exhibit 07: Global market: Size and forecast 2018-2023 ($ millions) Exhibit 08: Global market: Year-over-year growth 2019-2023 (%) Exhibit 09: Five forces analysis 2018 Exhibit 10: Five forces analysis 2023 Exhibit 11: Bargaining power of buyers Exhibit 12: Bargaining power of suppliers Exhibit 13: Threat of new entrants Exhibit 14: Threat of substitutes Exhibit 15: Threat of rivalry Exhibit 16: Market condition - Five forces 2018 Exhibit 17: Global gene therapy for CNS disorders market pipeline: Overview Exhibit 18: Global gene therapy for CNS disorders market pipeline: Snapshot Exhibit 19: Indication - Market share 2018-2023 (%) Exhibit 20: Comparison by indication Exhibit 21: SMA - Market size and forecast 2018-2023 ($ millions) Exhibit 22: SMA - Year-over-year growth 2019-2023 (%) Exhibit 23: Others - Market size and forecast 2018-2023 ($ millions) Exhibit 24: Others - Year-over-year growth 2019-2023 (%) Exhibit 25: Market opportunity by indication Exhibit 26: Customer landscape Exhibit 27: Market share by geography 2018-2023 (%) Exhibit 28: Geographic comparison Exhibit 29: North America - Market size and forecast 2018-2023 ($ millions) Exhibit 30: North America - Year-over-year growth 2019-2023 (%) Exhibit 31: Top 3 countries in North America Exhibit 32: Europe - Market size and forecast 2018-2023 ($ millions) Exhibit 33: Europe - Year-over-year growth 2019-2023 (%) Exhibit 34: Top 3 countries in Europe Exhibit 35: Asia - Market size and forecast 2018-2023 ($ millions) Exhibit 36: Asia - Year-over-year growth 2019-2023 (%) Exhibit 37: Top 3 countries in Asia Exhibit 38: ROW - Market size and forecast 2018-2023 ($ millions) Exhibit 39: ROW - Year-over-year growth 2019-2023 (%) Exhibit 40: Top 3 countries in ROW Exhibit 41: Key leading countries Exhibit 42: Market opportunity Exhibit 43: Impact of drivers and challenges Exhibit 44: Vendor landscape Exhibit 45: Landscape disruption Exhibit 46: Vendors covered Exhibit 47: Vendor classification Exhibit 48: Market positioning of vendors Exhibit 49: Biogen - Vendor overview Exhibit 50: Biogen - Business segments Exhibit 51: Biogen - Organizational developments Exhibit 52: Biogen - Geographic focus Exhibit 53: Biogen - Key offerings Exhibit 54: Biogen - Key customers Exhibit 55: bluebird bio, Inc. - Vendor overview Exhibit 56: bluebird bio, Inc. - Product segments Exhibit 57: bluebird bio, Inc. - Organizational developments Exhibit 58: bluebird bio, Inc. - Key offerings Exhibit 59: bluebird bio, Inc. - Key customers Exhibit 60: Novartis AG - Vendor overview Exhibit 61: Novartis AG - Business segments Exhibit 62: Novartis AG - Organizational developments Exhibit 63: Novartis AG - Geographic focus Exhibit 64: Novartis AG - Segment focus Exhibit 65: Novartis AG - Key offerings Exhibit 66: Novartis AG - Key customers Exhibit 67: Pfizer Inc. - Vendor overview Exhibit 68: Pfizer Inc. - Business segments Exhibit 69: Pfizer Inc. - Organizational developments Exhibit 70: Pfizer Inc. - Geographic focus Exhibit 71: Pfizer Inc. - Segment focus Exhibit 72: Pfizer Inc. - Key offerings Exhibit 73: Pfizer Inc. - Key customers Exhibit 74: Voyager Therapeutics - Vendor overview Exhibit 75: Voyager Therapeutics - Business segments Exhibit 76: Voyager Therapeutics - Organizational developments Exhibit 77: Voyager Therapeutics - Key offerings Exhibit 78: Voyager Therapeutics - Key customers Exhibit 79: Validation techniques employed for market sizing

Biogen bluebird bio, Inc. Novartis AG Pfizer Inc. Voyager Therapeutics