LIST OF EXHIBITS
EXHIBIT 1 SEGMENTATION OF US GENE THERAPY MARKET
EXHIBIT 2 MARKET SIZE CALCULATION APPROACH 2021
EXHIBIT 3 CLASSIFICATION OF REGENERATIVE MEDICINE
EXHIBIT 4 PAST & PRESENT GENERATIONS OF GENE THERAPY
EXHIBIT 5 PROCESS OF GENE THERAPY
EXHIBIT 6 HISTORY OF GENE THERAPY NIH-FDA OVERSIGHT
EXHIBIT 7 PROCESS OF CLINICAL TRIALS FOR GENE THERAPY PRODUCTS
EXHIBIT 8 FDA-APPROVED COMMERCIALIZED GENE THERAPY PRODUCTS IN THE US
EXHIBIT 9 IMPACT OF INCREASING MARKET ACCESS FOR GENE THERAPIES
EXHIBIT 10 IMPACT OF INTRODUCTION OF UNIVERSAL CAR-T THERAPY
EXHIBIT 11 IMPACT OF INCREASE IN STRATEGIC ACQUISITIONS
EXHIBIT 12 IMPACT OF INCREASED FUNDING FOR R&D ACTIVITIES OF GENE THERAPIES
EXHIBIT 13 IMPACT OF INCREASE IN CMOS TO MANUFACTURE GENE THERAPIES
EXHIBIT 14 MAJOR ADVANTAGES BY ENGAGING WITH CMOS
EXHIBIT 15 FACTORS CONSIDERED ON PARTNERSHIP WITH CONTRACT MANUFACTURERS
EXHIBIT 16 IMPACT OF INCREASING TARGET PATIENT POPULATION
EXHIBIT 17 IMPACT OF PRODUCT LAUNCHES & REGULATORY APPROVALS
EXHIBIT 18 IMPACT OF REGULATORY SUPPORT & SPECIAL DESIGNATIONS FOR GENE THERAPIES
EXHIBIT 19 CUMULATIVE NUMBER OF RMAT REQUESTS RECEIVED BY FDA 2017–2022
EXHIBIT 20 IMPACT OF HIGH COST OF GENE THERAPY PRODUCTS
EXHIBIT 21 IMPACT OF LIMITATIONS OF GENE THERAPY PRODUCTS
EXHIBIT 22 IMPACT OF AVAILABILITY OF ALTERNATIVE THERAPEUTICS & WITHDRAWAL OF PRODUCTS
EXHIBIT 23 US GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 24 US GENE THERAPY MARKET BY VECTOR TYPE: MARKET SHARE 2021 & 2027
EXHIBIT 25 US GENE THERAPY MARKET BY PRODUCT: MARKET SHARE 2021 & 2027
EXHIBIT 26 US GENE THERAPY MARKET BY INDICATION: MARKET SHARE 2021 & 2027
EXHIBIT 27 US GENE THERAPY MARKET BY GENE TRANSFER: MARKET SHARE 2021 & 2027
EXHIBIT 28 US GENE THERAPY MARKET BY END-USERS: MARKET SHARE 2021 & 2027
EXHIBIT 29 FIVE FORCES ANALYSIS 2021
EXHIBIT 30 INCREMENTAL GROWTH BY VECTOR TYPE 2021 & 2027
EXHIBIT 31 US GENE THERAPY MARKET BY VECTOR TYPE
EXHIBIT 32 TYPES OF VECTORS USED IN GENE THERAPY
EXHIBIT 33 US GENE THERAPY MARKET BY VECTOR TYPE: INCREMENTAL GROWTH
EXHIBIT 34 US GENE THERAPY MARKET BY VECTOR TYPE: ABSOLUTE GROWTH
EXHIBIT 35 US RETROVIRAL GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 36 US RETROVIRAL GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 37 NON-VIRAL VECTOR SYSTEMS USED FOR GENE TRANSFER
EXHIBIT 38 US OTHER VECTORS GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 39 US OTHER VECTORS GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 40 INCREMENTAL GROWTH BY PRODUCT TYPE 2021 & 2027
EXHIBIT 41 US GENE THERAPY MARKET BY PRODUCT TYPE
EXHIBIT 42 US GENE THERAPY MARKET BY PRODUCT TYPE: INCREMENTAL GROWTH
EXHIBIT 43 US GENE THERAPY MARKET BY PRODUCT TYPE: ABSOLUTE GROWTH
EXHIBIT 44 US CAR-T GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 45 US CAR-T GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 46 US NON-CAR-T GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 47 US NON-CAR-T GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 48 INCREMENTAL GROWTH BY INDICATION 2021 & 2027
EXHIBIT 49 US GENE THERAPY MARKET BY INDICATION TYPE
EXHIBIT 50 US GENE THERAPY MARKET BY INDICATION: INCREMENTAL GROWTH
EXHIBIT 51 US GENE THERAPY MARKET BY INDICATION: ABSOLUTE GROWTH
EXHIBIT 52 US ONCOLOGY GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 53 US ONCOLOGY GENE THERAPY MARKET 2021-2027 ($ MILLION)
EXHIBIT 54 US OTHER INDICATIONS GENE THERAPY MARKET: INCREMENTAL GROWTH & ABSOLUTE GROWTH
EXHIBIT 55 US OTHER INDICATIONS GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 56 INCREMENTAL GROWTH BY GENE TRANSFER 2021 & 2027
EXHIBIT 57 US GENE THERAPY MARKET BY GENE TRANSFER TYPE
EXHIBIT 58 US GENE THERAPY MARKET BY GENE TRANSFER: INCREMENTAL GROWTH
EXHIBIT 59 US GENE THERAPY MARKET BY GENE TRANSFER: ABSOLUTE GROWTH
EXHIBIT 60 US EX-VIVO GENE THERAPY MARKET: INCREMENTAL & ABSOLUTE GROWTH
EXHIBIT 61 US EX-VIVO GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 62 US IN-VIVO GENE THERAPY MARKET: INCREMENTAL & ABSOLUTE GROWTH
EXHIBIT 63 US IN-VIVO GENE THERAPY MARKET 2021–2027 ($ MILLION)
EXHIBIT 64 INCREMENTAL GROWTH BY END-USERS 2021 & 2027
EXHIBIT 65 US GENE THERAPY MARKET BY END-USERS
EXHIBIT 66 US GENE THERAPY MARKET BY END-USERS: INCREMENTAL GROWTH
EXHIBIT 67 US GENE THERAPY MARKET BY END-USERS: ABSOLUTE GROWTH
EXHIBIT 68 US GENE THERAPY MARKET BY HOSPITALS: INCREMENTAL & ABSOLUTE GROWTH
EXHIBIT 69 US GENE THERAPY MARKET BY HOSPITALS 2021–2027 ($ MILLION)
EXHIBIT 70 US GENE THERAPY MARKET BY CANCER CARE CENTERS: INCREMENTAL & ABSOLUTE GROWTH
EXHIBIT 71 US GENE THERAPY MARKET BY CANCER CARE CENTERS 2021–2027 ($ MILLION)
EXHIBIT 72 US GENE THERAPY MARKET BY ACADEMIC & RESEARCH INSTITUTES: INCREMENTAL & ABSOLUTE GROWTH
EXHIBIT 73 US GENE THERAPY MARKET BY ACADEMIC & RESEARCH CENTERS 2021–2027 ($ MILLION)
EXHIBIT 74 PURE & DIVERSIFIED VENDORS IN US GENE THERAPY MARKET
EXHIBIT 75 AMGEN: TOTAL REVENUE 2018−2020 ($ MILLION)
EXHIBIT 76 AMGEN: R&D EXPENDITURE 2018−2020 ($ MILLION)
EXHIBIT 77 BLUEBIRD BIO: TOTAL REVENUE 2018−2020 ($ MILLION)
EXHIBIT 78 BLUEBIRD BIO: R&D EXPENDITURE 2018−2020 ($ MILLION)
EXHIBIT 79 BRISTOL MYERS SQUIBB: TOTAL REVENUE 2019−2021 ($ MILLION)
EXHIBIT 80 BRISTOL MYERS SQUIBB: R&D EXPENDITURE 2019−2021 ($ MILLION)
EXHIBIT 81 BRISTOL MYERS SQUIBB: REVENUE BY GEOGRAPHIC REGIONS 2021 ($ MILLION)
EXHIBIT 82 GILEAD SCIENCES: TOTAL REVENUE 2018−2020 ($ MILLION)
EXHIBIT 83 GILEAD SCIENCES: R&D EXPENDITURE 2018−2020 ($ MILLION)
EXHIBIT 84 NOVARTIS: TOTAL REVENUE 2018−2020 ($ MILLION)
EXHIBIT 85 NOVARTIS: R&D EXPENDITURE 2018−2020 ($ MILLION)
EXHIBIT 86 NOVARTIS: REVENUE BY BUSINESS SEGMENT 2019−2020 ($ MILLION)
EXHIBIT 87 NOVARTIS: REVENUE BY GEOGRAPHY 2020 ($ MILLION)
EXHIBIT 88 F. HOFFMANN-LA ROCHE: TOTAL REVENUE 2019−2021 ($ BILLION)
EXHIBIT 89 F. HOFFMANN-LA ROCHE: R&D EXPENDITURE 2019−2021 ($ BILLION)
LIST OF TABLES
TABLE 1 KEY CAVEATS
TABLE 2 CURRENCY CONVERSION 2015−2021
TABLE 3 MAJOR FINANCING CHALLENGES EXIST FOR PAYERS
TABLE 4 EXAMPLES OF GENE THERAPIES LAUNCHED IN US ASSOCIATED WITH HIGH COSTS
TABLE 5 EXEMPLARS OF INNOVATIVE PAYMENT MECHANISMS USED FOR GENE THERAPIES IN US
TABLE 6 GENE THERAPY CLINICAL TRIAL STATUS IN US MARKET
TABLE 7 GENE THERAPY CLINICAL TRIALS IN DIFFERENT PHASES IN US MARKET
TABLE 8 GENE THERAPY CLINICAL TRIALS ENROLLMENT STATUS IN US MARKET
TABLE 9 GENE THERAPY PRODUCTS IN PIPELINE & EXPECTED YEAR OF LAUNCH
TABLE 10 PHARMACEUTICAL COMPANIES & NON-PROFIT ORGANIZATIONS FOR DEVELOPMENT AND UNLOCKING ACCESS TO GENE THERAPIES
TABLE 11 RECENT STRATEGIC ACQUISITIONS IN GENE THERAPY MARKET
TABLE 12 FUNDING OF GENE THERAPY CLINICAL TRIALS LISTED IN 2020
TABLE 13 MAJOR INVESTMENTS BY CMOS IN GENE THERAPY DEVELOPMENT
TABLE 14 TARGET POPULATION & RESPECTIVE GENE THERAPY IN US AS OF 2021
TABLE 15 PRODUCT APPROVALS & LAUNCH DETAILS
TABLE 16 OVERVIEW OF ALL FOUR FDA’S EXPEDITED PROGRAMS
TABLE 17 LIST OF PRODUCTS THAT RECEIVED RMAT DESIGNATION
TABLE 18 COST OF KYMRIAH IN DIFFERENT REGIONS
TABLE 19 FDA APPROVED CAR-T THERAPIES IN THE US
TABLE 20 FDA APPROVED NON-CAR-T THERAPIES IN US
TABLE 21 GENE THERAPY PRODUCTS AVAILABLE FOR CANCER IN US
TABLE 22 GENE THERAPY PRODUCTS AVAILABLE FOR GENETIC DISORDERS IN US
TABLE 23 TOP CANCER CARE CENTERS ACROSS US
TABLE 24 VENDORS OFFERING COMMERCIAL GENE THERAPY PRODUCTS IN US: MARKET DOMINANCE ANALYSIS
TABLE 25 VENDORS OFFERING COMMERCIAL GENE THERAPY PRODUCTS IN US: CAPABILITIES & EXPERTISE ASSESSMENT
TABLE 26 US GENE THERAPY MARKET: VENDOR RANKING
TABLE 27 AMGEN: MAJOR PRODUCT OFFERINGS
TABLE 28 BLUEBIRD BIO: MAJOR PRODUCT OFFERINGS
TABLE 29 BRISTOL MYERS SQUIBB: MAJOR PRODUCT OFFERINGS
TABLE 30 GILEAD SCIENCES: MAJOR PRODUCT OFFERINGS
TABLE 31 NOVARTIS: MAJOR PRODUCT OFFERINGS
TABLE 32 F. HOFFMANN-LA ROCHE: MAJOR PRODUCT OFFERINGS
TABLE 33 US GENE THERAPY MARKET BY VECTOR TYPE 2021−2027 ($ MILLION)
TABLE 34 US GENE THERAPY MARKET BY VECTOR TYPE 2021−2027 (%)
TABLE 35 US GENE THERAPY MARKET BY PRODUCT TYPE 2021−2027 ($ MILLION)
TABLE 36 US GENE THERAPY MARKET BY PRODUCT TYPE 2021−2027 (%)
TABLE 37 US GENE THERAPY MARKET BY INDICATION 2021−2027 ($ MILLION)
TABLE 38 US GENE THERAPY MARKET BY INDICATION 2021−2027 (%)
TABLE 39 US GENE THERAPY MARKET BY GENE TRANSFER 2021−2027 ($ MILLION)
TABLE 40 US GENE THERAPY MARKET BY GENE TRANSFER 2021−2027 (%)
TABLE 41 US GENE THERAPY MARKET BY END-USER 2021−2027 ($ MILLION)
TABLE 42 US GENE THERAPY MARKET BY END-USER 2021−2027 (%)