Gene Therapy Market Size, Share & Trends Analysis Report By Indication (Acute Lymphoblastic Leukemia, Large B-cell Lymphoma), By Vector Type, By Region, And Segment Forecasts, 2020 - 2027

Industry Insights

The global gene therapy market size was estimated at USD 1.2 billion in 2019 and is projected to register a CAGR 16.6% over the forecast period. Gene therapy is mainly driven by the potential exhibited by CAR T-cell usage which has gained significant attention from commercial and non-commercial sponsors as a new business model. Gene therapy accounts for a significant share in the pharmaceutical industry’s pipeline, and this is anticipated to continue since products in the R&D phase would become commercially available over the next few years. The notable approvals of Yescarta and Kymriah in 2017 have prompted these unprecedented developments in the market for gene therapy.

Furthermore, rapid and significant progress in the molecular and cellular biology arena, driven by technological advancements in genomics and gene-editing tools, has contributed to an increasing number of approved gene therapies as well as an expanding pipeline. According to the Alliance for Regenerative Medicine (ARM), by the end of the second quarter of 2019, there were more than 700 clinical trials ongoing globally.

With recent product approvals, the cell and gene therapy sector is witnessing increasing competition for gene therapy buyouts, resulting in hefty premiums in the marketplace. For instance, in December 2019, Roche acquired Spark Therapeutics, a company that develops and delivers gene therapies.

Despite the significant health gains offered by gene therapy, these usually comprise high upfront costs, which further pose sustainability and budget challenges to the healthcare systems. Although affordability and budget impacts are not considered as key components of the value of new therapy treatments, they are important elements for access-related decision making.

Owing to the presence of the small size of the target patient populations for several products, coupled with their applicability as one-off treatments, the prices of products need to be set at a level that makes the product economically viable for the developer. The gene therapy market has witnessed the withdrawal of several products due to their non-financial viability in the past years. Thus, value to patients and healthcare systems needs to be balanced with the financial sustainability for both, developers and payers.

Indication Insights

The number of gene therapy applications has increased in recent years, including the treatment of various rare and incurable diseases such as cancer and inherited genetic diseases. With an increase in the number of molecules being tested across various clinical trials, the trials intended for the development of cancer therapies are leading the count.

For instance, Novartis is focusing on extending its approval of Kymriah, a gene therapy designed for the treatment of hematological cancer, in other established and emerging countries. This is anticipated to drive the acute lymphoblastic leukemia (ALL) and large B-cell lymphoma segments, which are estimated to register a double-digit growth rate over the forecast period.

Furthermore, Kite Pharma developed a team for providing training with regard to logistical and biological intricacies of the drug to hospitals and clinics that will be using Yescarta for Large B-cell Lymphoma. The team offers certifications to centers to ensure the safe and effective implementation of Yescarta. These factors are expected to significantly impact the revenue growth for the large B-cell lymphoma segment.

Vector Type Insights

Owing to the high accuracy in delivering the gene to the region of interest, the demand and usage for adeno-associated viruses (AAV) as a platform for gene therapy delivery continue to increase within research settings in clinical trials. Companies such as Spark Therapeutics, AGTC, Biogen, Sangamo Biosciences, Dimension Therapeutics, and AveXis are engaged in gene therapy R&D using AAV viral vectors.

Lentivirus is anticipated to register the highest CAGR over the forecast period since key developers are increasingly interested in this vector type, post-approval of Zynteglo in 2019 and Kymriah in 2017. Furthermore, the presence of companies that offer an exclusive LentiVector platform to key gene therapy developers is driving the segment growth.

Several biopharmaceutical companies such as Novartis, Orchard Therapeutics, Sanofi, GSK, and ImmuneDesign have collaborated with Oxford Biomedica to use its LentiVector platform, which can carry payloads of up to 9 kilobase for the development of new gene therapy molecules. A relatively smaller number of approved products, coupled with low marketing activities for non-viral vectors, has slowed down the revenue growth for non-viral vectors.

Regional Insights

Increase in financing for cell and gene therapy companies across European countries such as U.K. is anticipated to drive the Europe market at a lucrative growth rate. Moreover, European companies are engaged in extending product distribution across the globe through securing approval from multiple regulatory bodies.

For instance, post-EU approval of BlueBird Bio’s Zynteglo (formerly LentiGlobin), a gene therapy for transfusion-dependent β-thalassemia in June 2019, the company is planning to file for FDA approval. Moreover, Orchard Therapeutics’ OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy, is anticipated to enter the market in North America by 2020. These factors are anticipated to positively impact the revenue share in both, the Europe and North America markets.

China is recognized as the global focal point for new therapy development. In the current scenario, the number of ongoing gene therapy clinical trials in China has surpassed the number of trials in U.S. On the other hand, in May 2019, a Japanese government panel approved Kymriah, which allowed Novartis to sell its product in the country, thereby driving the market’s revenue generation in Japan. The Asia Pacific market for gene therapy is hence estimated to witness a double-digit growth rate over the forecast period.

Gene Therapy Market Share Insights

The increasing interest of public and private investors in gene therapy is anticipated to accelerate the entry of new players in the market. The entry of well-established pharmaceutical companies in gene therapy is anticipated to result in companies adopting strategies such as mergers and acquisitions.

Over the past few years, the market has witnessed notable merger and acquisitions. For instance, Celgene Corporation acquired Juno Therapeutics and Novartis acquired AveXis. In addition, key players are engaged in collaborating with other market players for product development. Partnerships between companies in this market have driven the upfront values by 55.0% from 2017 to 2018.

Current major players in the market are Novartis AG; Spark Therapeutics LLC; Bluebird Bio; Gilead Sciences Inc.; Celgene Corporation; GlaxoSmithKline; Sangamo Biosciences; Shire Plc; Voyager Therapeutics; Dimension Therapeutics; Chiesi Farmaceutici S.p.A; Bristol Myer’s Squibb; UniQure NV; Human Stem Cell Institute; and Celgene Corporation. These players focus on extending product distribution and expanding market operations to boost their revenue share in the market.

Report Scope

Segments Covered in the Report

This report forecasts revenue growth global, regional, and country levels, and provides an analysis of latest industry trends in each of the sub-segments from 2016 to 2027. For the purpose of this study, Grand View Research has segmented the global gene therapy market report on the basis of indication, vector type, and region:

• Indication Outlook (Revenue, USD Million, 2016 - 2027)

  • Acute Lymphoblastic Leukemia (ALL)

  • Inherited Retinal Disease

  • Large B-cell Lymphoma

  • ADA-SCID

  • Melanoma (Lesions)

  • Beta-Thalassemia Major/SCD

  • Head & Neck Squamous Cell Carcinoma

  • Peripheral Arterial Disease

  • Spinal Muscular Atrophy (SMA) 

  • Others

• Vector Type Outlook (Revenue, USD Million, 2016 - 2027)

  • Lentivirus

  • AAV

  • Retrovirus & Gammaretrovirus

  • Modified Herpes Simplex Virus

  • Adenovirus

  • Non-viral Plasmid Vector

• Regional Outlook (Revenue, USD Million, 2016 - 2027)

  • North America

    • U.S.

    • Canada

  • Europe

  • Asia Pacific

    • Japan

    • China

    • Russia

    • Australia