U.S. Gene Therapy Market - Industry Outlook & Forecast 2022-2027

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37 company

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THE U.S. GENE THERAPY MARKET SIZE WAS VALUED AT USD 1.8 BILLION IN 2021 AND IS EXPECTED TO REACH USD 16.01 BILLION BY 2027, GROWING AT A CAGR OF 43.44%.

U.S. Gene Therapy Market Report Includes Size, Share, & Trends Analysis By

  • Vector Type: Retroviral and Others
  • Product Type: Car T Therapy and Non-Car T Therapy
  • Indication: Oncology and Others
  • Gene Transfer: Ex-Vivo and In-Vivo
  • End-User: Hospitals, Cancer Care Centers, and Academic & Research Centers

Industry Analysis Report, Regional Outlook, Growth Potential, Price Trends, Competitive Market Share & Forecast, 2022–2027

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U.S. GENE THERAPY MARKET SIZE & FORECAST

REPORT ATTRIBUTEDETAILS
Market Size (Revenue)USD 16,013.28 Million (2027)
CAGR43.44% (2022-2027)
Base Year2021
Forecast Year2022-2027
Market Segments (Vector Type)Retroviral and Others
Market Segments (Product)Car T Therapy and Non-Car T Therapy
Market Segments (Indication)Oncology and Others
Market Segments (Gene Transfer)Ex-Vivo and In-Vivo
End UserHospitals, Cancer Care Centers, and Academic & Research Centers
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MARKET INSIGHTS

The U.S. gene therapy market is projected to grow at a CAGR of 43% from 2022 to 2027 and is expected to reach USD 16.01 billion by 2027 from USD 1.8 billion in 2021. The gene therapy market in the U.S. is one of the fastest-growing segments of the regenerative medicine market. This growth can be attributable to the growing prevalence of various chronic diseases like cancer, cartilage-related problems, wounds, diabetic foot ulcers, genetic disorders, and other rare diseases across the United States. Further, the U.S. gene therapy market share is witnessing a steady growth due to increased funding availability from various public and private institutes. In addition, there is increased support from the U.S. regulatory bodies for product approvals and providing fast-track designations to the products, encouraging vendors to manufacture the products faster.

Gene therapy is a process in which the genes inside the body are altered to treat or stop the disease. Research and development activities in the U.S. gene therapy market are witnessing significant growth with support from both public and private entities. In 2020, the U.S. FDA saw a yearly doubling of new gene therapy applications. By 2025, the US is expected to approve 10–20 gene therapies yearly. In addition, the US government is creating awareness about gene therapies among the population and bringing various strategies to facilitate affordability and payments for these treatments. However, the demand for innovative medicines and therapies increased across the US. The investment in gene therapy has also increased drastically in recent years.

KEY INSIGHTS

  • Vendors' and government organizations' high emphasis on R&D and augmented investment have led to a strong portfolio of U.S. gene therapy pipeline products. Many vendors are planning to tap the prevailing potential of the U.S. gene therapy market by developing and commercializing innovative products.
  • In the past several years, Gene therapies have continued their rising trend in the biopharmaceutical industry. Biopharma companies are investing in these therapies, and the FDA has encouraged gene therapy development.
  • The Alliance for Regenerative Medicine has worked with the Centers for Medicare & Medicaid Services to ensure appropriate reimbursement for CAR-T and other innovative therapies. This favorable situation will boost the number of patients opting for treatments over conventional treatments in the U.S. gene therapy market.

MARKET TRENDS & DRIVERS

  • The U.S. Food and Drug Administration (FDA), the National Institutes of Health (NIH), 10 pharmaceutical companies, and 5 non-profit organizations are working together to accelerate the development of gene therapy for 30 million Americans with rare diseases.
  • Currently, the gene therapy market in the U.S. is witnessing many M&As. Vendors, especially the global players, are increasingly focused on pursuing inorganic growth strategies such as acquisitions and investments in smaller biotech firms and emerging companies to expand their presence, enhance their product portfolio, and improve expertise in the market.
  • Price trends for gene therapy products were analyzed for the past few years and given due consideration to derive the projections for the forthcoming six years using a set of forecasting techniques. As the gene therapy pipeline approaches the market, the acknowledgment and awareness of outcome-based payment systems are potentially effective mechanisms for reducing long-term data uncertainty. The research shows that stakeholders are motivated to consider and adopt innovative payment solutions, which will continue.
  • Over the last three decades, gene therapy in the U.S. has made tremendous progress, offering significant reductions from pure laboratory experiments to FDA-approved products to patients who previously had no treatment options for serious illnesses. Therefore, this ongoing trend in gene therapy is expected to boost the market growth in the U.S.
  • The demand for innovative medicines and therapies is increasing across the United States. The investment in the gene therapy market has also increased drastically in recent years. Recently, many government organizations and private firms have funded many biotech start-ups and research institutes that invest in the R&D of gene therapy products.

MARKET SEGMENTATION

Gene transfer via retroviral vectors is central to the development of gene therapy. Recently, the broader field of gene transfer and gene therapy via retroviral vectors has been revived with the development of a new class of lentiviral-derived retroviral vectors. The retroviral US gene therapy market is expected to reach USD 8,861.75 million by 2027.

Advanced therapies such as CAR T-cell therapy are being introduced, prompting major countries to invest in the infrastructure and improve these therapies for patient populations. The U.S. gene therapy market is growing at a double-digit CAGR, which is expected to help the oncology segment as many cell and gene therapies are commercially available. Most of the recently launched drugs for cancer are fetching healthy revenues for manufacturers as they are more expensive than most cell and gene therapies.

With the significant increase in the development of in-vivo gene therapy in recent years, the biotechnology and pharmaceutical industries have invested heavily in commercializing these potentially curative therapies to treat various diseases. This is an exciting time for gene therapy, with AAV playing a leading role.

COMPETITIVE LANDSCAPE

Gilead Sciences, Novartis, F. Hoffmann-La Roche, Amgen, bluebird bio, and Bristol Myers Squibb are the leading players with significant U.S. gene therapy market share. Many regional and local businesses threaten key players with innovative, cost-effective products and technologies. This shows that the market offers tremendous growth opportunities for existing and emerging players. Major players focus on licensing, strategic acquisitions, and collaboration agreements with emerging players to enter the gene therapy market and quickly access commercially launched products. The gene therapy market in the U.S. is highly consolidated, with Novartis and Gilead Sciences accounting for significant shares. Amgen and Spark Therapeutics are other vendors with notable shares in the U.S. gene therapy market.

Frequently Asked Questions

What is the expected U.S. Gene Therapy Market size by 2027?

The U.S. gene therapy market is expected to cross USD 16.0133 billion by 2027. This growth can be attributable to the growing prevalence of various chronic diseases like cancer, cartilage-related problems, wounds, diabetic foot ulcers, genetic disorders, and other rare diseases across the United States.

What are the latest trends in the U.S. gene therapy market?

Product launches & regulatory approvals and, regulatory support & special designations for gene therapies are the latest trends in the market.

Who are the key market players in the U.S. gene therapy market?

Key players include Amgen, bluebird bio, Bristol Myers Squibb, Gilead Sciences, Novartis, and F. Hoffmann-La Roche.

Which vector segments hold the major U.S. gene therapy market share?

Retroviral holds the largest share in the U.S. gene therapy market. Gene transfer via retroviral vectors is central to the development of gene therapy. Retroviral vectors are the most used type of gene delivery vectors for clinical gene therapy in the U.S. The significant advantages of retroviral vectors are the ability to stably integrate into the target cell genome and ease of manipulation to insert the therapeutic gene.

What initiatives are vendors taking in the gene therapy market in the U.S.?

High emphasis on R&D and augmented investment are some of the initiatives taken by vendors in the U.S. gene therapy market. Investing in R&D activities to develop novel therapies is key for the long-term sustainability of companies operating in the U.S. gene therapy market. Therefore, many players, including key and emerging companies and smaller vendors, are investing in many R&D activities.

U.S. gene therapy market size was valued at USD 1,838.49 million in 2021 and is expected to reach USD 16,013.28 million by 2027, growing at a CAGR of 43.44% during 2022-2027

The following factors are likely to contribute to the growth of the U.S. gene therapy market –

  • Increasing Market Access for Gene Therapies
  • Introduction of Universal Car-T Therapy
  • Increased Funding for R&D Activities of Gene Therapies
  • Product Launches & Regulatory Approvals

Base Year: 2021

Forecast Year: 2022-2027

The study considers a detailed scenario of the present U.S. gene therapy market and its market dynamics for 2022−2027. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Vendors

  • Amgen
    • Business Overview
    • Product Offerings
    • Key Strategies
    • Key Strengths
    • Key Opportunities
  • bluebird bio
  • Bristol Myers Squibb
  • Gilead Sciences
  • Novartis
  • F. Hoffmann-La Roche

Other Prominent Vendors

  • 4DMT
    • Business Overview
  • Abeona Therapeutics
  • AnGes
  • AskBio
  • Astellas Gene Therapies
  • Autolus Therapeutics
  • Candel Therapeutics
  • Castle Creek Biosciences
  • Cellectis
  • Evox Therapeutics
  • Freeline Therapeutics
  • Gene Biotherapeutics
  • Generation Bio
  • GenSight Biologics
  • Helixmith
  • Janssen Pharmaceuticals
  • Kolon Tissue Gene
  • Krystal Biotech
  • MeiraGTx
  • Orchard Therapeutics
  • Pfizer
  • Poseida Therapeutics
  • Passage Bio
  • RegenxBio
  • Sana Biotechnology
  • Sarepta Therapeutics
  • StrideBio
  • Solid Biosciences
  • VBL Therapeutics
  • Voyager Therapeutics
  • Uniqure

Market Segmentation by Vector Type

  • Retroviral
  • Others

Market Segmentation by Product Type

  • Car T Therapy
  • Non-Car T Therapy

Market Segmentation by Indication

  • Oncology
  • Others

Market Segmentation by Gene Transfer

  • Ex-Vivo
  • In-Vivo

Market Segmentation by End User

  • Hospitals
  • Cancer Care Centers
  • Academic & Research Centers

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 (%)

1 RESEARCH METHODOLOGY

2 RESEARCH OBJECTIVES

3 RESEARCH PROCESS

4 SCOPE & COVERAGE

4.1 MARKET DEFINITION

4.1.1 INCLUSIONS

4.1.2 EXCLUSIONS

4.1.3 MARKET ESTIMATION CAVEATS

4.2 BASE YEAR

4.3 SCOPE OF THE STUDY

4.3.1 MARKET SEGMENTATION BY VECTOR

4.3.2 MARKET SEGMENTATION BY PRODUCT

4.3.3 MARKET SEGMENTATION BY INDICATION

4.3.4 MARKET SEGMENTATION BY GENE TRANSFER

4.3.5 MARKET SEGMENTATION BY END-USERS

5 REPORT ASSUMPTIONS & CAVEATS

5.1 KEY CAVEATS

5.2 CURRENCY CONVERSION

5.3 MARKET DERIVATION

6 MARKET AT A GLANCE

7 INTRODUCTION

7.1 BACKGROUND

7.2 GENE THERAPY: AN OVERVIEW

7.2.1 EVOLUTION OF GENE THERAPY

8 REIMBURSEMENT FOR GENE THERAPY IN US

8.1 OVERVIEW

9 REGULATORY SCENARIO FOR GENE THERAPY PRODUCTS

9.1 OVERVIEW

9.2 CLINICAL TRIAL PROCESS FOR GENE THERAPY

10 COMMERCIAL GENE THERAPY PRODUCTS IN US

10.1 OVERVIEW

10.1.1 ABECMA (IDECABTAGENE VICLEUCEL)

10.1.2 BREYANZI (LISOCABTAGENE MARALEUCEL)

10.1.3 TECARTUS (BREXUCABTAGENE AUTOLEUCEL)

10.1.4 ZOLGENSMA (ONASEMNOGENE ABEPARVOVEC)

10.1.5 LUXTURNA (VORETIGENE NEPARVOVEC)

10.1.6 YESCARTA (AXICABTAGENE CILOLEUCEL)

10.1.7 KYMRIAH (TISAGENLECLEUCEL-T)

10.1.8 IMLYGIC (TALIMOGENE LAHERPAREPVEC)

11 GENE THERAPY PRODUCTS IN PIPELINE

11.1 OVERVIEW

12 MARKET OPPORTUNITIES & TRENDS

12.1 INCREASING MARKET ACCESS FOR GENE THERAPIES

12.2 INTRODUCTION OF UNIVERSAL CAR-T THERAPY

12.3 INCREASE IN STRATEGIC ACQUISITIONS

12.4 INCREASED FUNDING FOR R&D ACTIVITIES OF GENE THERAPIES

13 MARKET GROWTH ENABLERS

13.1 INCREASE IN CMOS TO MANUFACTURE GENE THERAPIES

13.2 INCREASING TARGET PATIENT POPULATION

13.3 PRODUCT LAUNCHES & REGULATORY APPROVALS

13.4 REGULATORY SUPPORT & SPECIAL DESIGNATIONS FOR GENE THERAPIES

14 MARKET RESTRAINTS

14.1 HIGH COST OF GENE THERAPY PRODUCTS

14.2 LIMITATIONS OF GENE THERAPY PRODUCTS

14.3 AVAILABILITY OF ALTERNATIVE THERAPEUTICS & WITHDRAWAL OF PRODUCTS

15 MARKET LANDSCAPE

15.1 MARKET OVERVIEW

15.2 MARKET SIZE & FORECAST

15.2.1 INSIGHTS BY VECTOR TYPE

15.2.2 INSIGHTS BY PRODUCT TYPE

15.2.3 INSIGHTS BY INDICATION TYPE

15.2.4 INSIGHTS BY GENE TRANSFER TYPE

15.2.5 INSIGHTS BY END-USERS

15.3 FIVE FORCES ANALYSIS

15.3.1 THREAT OF NEW ENTRANTS

15.3.2 BARGAINING POWER OF SUPPLIERS

15.3.3 BARGAINING POWER OF BUYERS

15.3.4 THREAT OF SUBSTITUTES

15.3.5 COMPETITIVE RIVALRY

16 VECTOR TYPE

16.1 MARKET SNAPSHOT & GROWTH ENGINE

16.2 MARKET OVERVIEW

16.3 RETROVIRAL

16.3.1 MARKET OVERVIEW

16.3.2 MARKET SIZE & FORECAST

16.4 OTHER VECTORS

16.4.1 MARKET OVERVIEW

16.4.2 MARKET SIZE & FORECAST

17 PRODUCT TYPE

17.1 MARKET SNAPSHOT & GROWTH ENGINE

17.2 MARKET OVERVIEW

17.3 CAR-T THERAPY

17.3.1 MARKET OVERVIEW

17.3.2 MARKET SIZE & FORECAST

17.4 NON-CAR-T THERAPY

17.4.1 MARKET OVERVIEW

17.4.2 MARKET SIZE & FORECAST

18 INDICATION

18.1 MARKET SNAPSHOT & GROWTH ENGINE

18.2 MARKET OVERVIEW

18.3 ONCOLOGY

18.3.1 MARKET OVERVIEW

18.3.2 MARKET SIZE & FORECAST

18.4 OTHERS

18.4.1 MARKET OVERVIEW

18.4.2 MARKET SIZE & FORECAST

19 GENE TRANSFER

19.1 MARKET SNAPSHOT & GROWTH ENGINE

19.2 MARKET OVERVIEW

19.3 EX-VIVO

19.3.1 MARKET OVERVIEW

19.3.2 MARKET SIZE & FORECAST

19.4 IN-VIVO

19.4.1 MARKET OVERVIEW

19.4.2 MARKET SIZE & FORECAST

20 END-USERS

20.1 MARKET SNAPSHOT & GROWTH ENGINE

20.2 MARKET OVERVIEW

20.3 HOSPITALS

20.3.1 MARKET OVERVIEW

20.3.2 MARKET SIZE & FORECAST

20.4 CANCER CARE CENTERS

20.4.1 MARKET OVERVIEW

20.4.2 MARKET SIZE & FORECAST

20.5 ACADEMIC & RESEARCH CENTERS

20.5.1 MARKET OVERVIEW

20.5.2 MARKET SIZE & FORECAST

21 COMPETITIVE LANDSCAPE

21.1 COMPETITION OVERVIEW

21.2 MARKET SHARE ANALYSIS

21.2.1 AMGEN

21.2.2 BLUEBIRD BIO

21.2.3 BRISTOL MYERS SQUIBB

21.2.4 GILEAD SCIENCES

21.2.5 NOVARTIS

21.2.6 F. HOFFMANN-LA ROCHE

22 KEY COMPANY PROFILES

22.1 AMGEN

22.1.1 BUSINESS OVERVIEW

22.1.2 PRODUCT OFFERINGS

22.1.3 KEY STRATEGIES

22.1.4 KEY STRENGTHS

22.1.5 KEY OPPORTUNITIES

22.2 BLUEBIRD BIO

22.2.1 BUSINESS OVERVIEW

22.2.2 PRODUCT OFFERINGS

22.2.3 KEY STRATEGIES

22.2.4 KEY STRENGTHS

22.2.5 KEY OPPORTUNITIES

22.3 BRISTOL MYERS SQUIBB

22.3.1 BUSINESS OVERVIEW

22.3.2 PRODUCT OFFERINGS

22.3.3 KEY STRATEGIES

22.3.4 KEY STRENGTHS

22.3.5 KEY OPPORTUNITIES

22.4 GILEAD SCIENCES

22.4.1 BUSINESS OVERVIEW

22.4.2 PRODUCT OFFERINGS

22.4.3 KEY STRATEGIES

22.4.4 KEY STRENGTHS

22.4.5 KEY OPPORTUNITIES

22.5 NOVARTIS

22.5.1 BUSINESS OVERVIEW

22.5.2 PRODUCT OFFERINGS

22.5.3 KEY STRATEGIES

22.5.4 KEY STRENGTHS

22.5.5 KEY OPPORTUNITIES

22.6 F. HOFFMANN-LA ROCHE

22.6.1 BUSINESS OVERVIEW

22.6.2 PRODUCT OFFERINGS

22.6.3 KEY STRATEGIES

22.6.4 KEY STRENGTHS

22.6.5 KEY OPPORTUNITIES

23 GENE THERAPY INVESTIGATIONAL COMPANIES

23.1 4DMT

23.1.1 BUSINESS OVERVIEW

23.2 ABEONA THERAPEUTICS

23.2.1 BUSINESS OVERVIEW

23.3 ANGES

23.3.1 BUSINESS OVERVIEW

23.4 ASKBIO

23.4.1 BUSINESS OVERVIEW

23.5 ASTELLAS GENE THERAPIES

23.5.1 BUSINESS OVERVIEW

23.6 AUTOLUS THERAPEUTICS

23.6.1 BUSINESS OVERVIEW

23.7 CANDEL THERAPEUTICS

23.7.1 BUSINESS OVERVIEW

23.8 CASTLE CREEK BIOSCIENCES

23.8.1 BUSINESS OVERVIEW

23.9 CELLECTIS

23.9.1 BUSINESS OVERVIEW

23.10 EVOX THERAPEUTICS

23.10.1 BUSINESS OVERVIEW

23.11 FREELINE THERAPEUTICS

23.11.1 BUSINESS OVERVIEW

23.12 GENE BIOTHERAPEUTICS

23.12.1 BUSINESS OVERVIEW

23.13 GENERATION BIO

23.13.1 BUSINESS OVERVIEW

23.14 GENSIGHT BIOLOGICS

23.14.1 BUSINESS OVERVIEW

23.15 HELIXMITH

23.15.1 BUSINESS OVERVIEW

23.16 JANSSEN PHARMACEUTICALS

23.16.1 BUSINESS OVERVIEW

23.17 KOLON TISSUE GENE

23.17.1 BUSINESS OVERVIEW

23.18 KRYSTAL BIOTECH

23.18.1 BUSINESS OVERVIEW

23.19 MEIRAGTX

23.19.1 BUSINESS OVERVIEW

23.20 ORCHARD THERAPEUTICS

23.20.1 BUSINESS OVERVIEW

23.21 PFIZER

23.21.1 BUSINESS OVERVIEW

23.22 POSEIDA THERAPEUTICS

23.22.1 BUSINESS OVERVIEW

23.23 PASSAGE BIO

23.23.1 BUSINESS OVERVIEW

23.24 REGENXBIO

23.24.1 BUSINESS OVERVIEW

23.25 SANA BIOTECHNOLOGY

23.25.1 BUSINESS OVERVIEW

23.26 SAREPTA THERAPEUTICS

23.26.1 BUSINESS OVERVIEW

23.27 STRIDEBIO

23.27.1 BUSINESS OVERVIEW

23.28 SOLID BIOSCIENCES

23.28.1 BUSINESS OVERVIEW

23.29 VBL THERAPEUTICS

23.29.1 BUSINESS OVERVIEW

23.30 VOYAGER THERAPEUTICS

23.30.1 BUSINESS OVERVIEW

23.31 UNIQURE

23.31.1 BUSINESS OVERVIEW

24 REPORT SUMMARY

24.1 KEY TAKEAWAYS

24.2 STRATEGIC RECOMMENDATIONS

25 QUANTITATIVE SUMMARY

25.1 MARKET BY VECTOR TYPE

25.2 MARKET BY PRODUCT TYPE

25.3 MARKET BY INDICATION

25.4 MARKET BY GENE TRANSFER

25.5 MARKET BY END-USER

26 APPENDIX

26.1 ABBREVIATIONS

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Frequently Asked Questions

What is the expected U.S. Gene Therapy Market size by 2027?

The U.S. gene therapy market is expected to cross USD 16.0133 billion by 2027. This growth can be attributable to the growing prevalence of various chronic diseases like cancer, cartilage-related problems, wounds, diabetic foot ulcers, genetic disorders, and other rare diseases across the United States.

What are the latest trends in the U.S. gene therapy market?

Product launches & regulatory approvals and, regulatory support & special designations for gene therapies are the latest trends in the market.

Who are the key market players in the U.S. gene therapy market?

Key players include Amgen, bluebird bio, Bristol Myers Squibb, Gilead Sciences, Novartis, and F. Hoffmann-La Roche.

Which vector segments hold the major U.S. gene therapy market share?

Retroviral holds the largest share in the U.S. gene therapy market. Gene transfer via retroviral vectors is central to the development of gene therapy. Retroviral vectors are the most used type of gene delivery vectors for clinical gene therapy in the U.S. The significant advantages of retroviral vectors are the ability to stably integrate into the target cell genome and ease of manipulation to insert the therapeutic gene.

What initiatives are vendors taking in the gene therapy market in the U.S.?

High emphasis on R&D and augmented investment are some of the initiatives taken by vendors in the U.S. gene therapy market. Investing in R&D activities to develop novel therapies is key for the long-term sustainability of companies operating in the U.S. gene therapy market. Therefore, many players, including key and emerging companies and smaller vendors, are investing in many R&D activities.