Gene Therapy: Global Market Trends & Notable Developments

By Priyanka Bhendale, assistant manager – Healthcare Market Research, Markets and Markets

With technological improvements, a better understanding of genetics, and regulatory approvals, the gene therapy market is fast evolving, providing new hope to patients with previously untreatable genetic disorders.

What does the next decade look like for this field of science in bringing solutions, health, and continued hope to patients?

According to our published market research, the gene therapy market is estimated to reach $9.0 billion in 2023 and is projected to reach $23.9 billion by 2028 with a CAGR of 21.4% during the forecast period (2023-2028). This growth is driven by factors such as the increasing number of regulatory approvals for gene therapy products, growing investments and funding for gene therapy research initiatives, and growing technological advancements, among other factors.

In this article, we will examine some of the anticipated developments, market dynamics, and trends in the gene therapy market at the global, regional, and segment levels.

Macro Trends In The Gene Therapy Market

Looking ahead, the gene therapy market is poised for continued growth, fueled by scientific advancements, expanding indications, and increasing acceptance among healthcare providers and patients. Several key trends shaping the landscape of the gene therapy market include the following.

Advancements in Technologies: Continued innovations in gene editing tools, such as CRISPR-Cas9, TALEN, ZFN, and base editing, are expected to enhance the precision and efficiency of gene therapy, opening up new possibilities for treatment of previously untreatable genetic conditions. For instance, in 2021, the FDA approved the first trial of CRISPR-based gene therapy for the treatment of sickle cell disease. This was a landmark event, and it is expected to pave the way for the approval of other CRISPR-based gene therapies in the near future. Further, AI can aid in the development of a novel medicine at several stages of the R&D value chain, such as target discovery, payload design optimization, translational and clinical development, and end-to-end (E2E) digitization.

Personalized Medicine: Gene therapy has the potential to usher in an era of personalized medicine, where treatments are tailored to an individual's genetic makeup. Advances in genomic sequencing and analysis will enable clinicians to identify patients who are most likely to benefit from gene therapy. In 2023, the FDA approved 26 personalized medications, including 20 new molecular entities and six newly approved cell/gene-based therapies. Additionally, in 2023, the FDA expanded 19 indications for existing personalized therapies.

Regulatory Approvals: An increasing number of gene therapies are receiving regulatory approvals, streamlining their path to commercialization and patient access. According to an analysis of gene therapy clinical pipelines by CVS Health Corporation, from a study of current pipeline products in Phase 3 clinical trials, the number of approvals is expected to rise significantly in the near future. In 2024, up to 21 cell therapy launches and approximately 31 gene therapy launches (including more than 29 adeno-associated virus (AAV) therapies) are expected.

Expansion of Indications: As research continues, gene therapy is expected to expand into new therapeutic areas beyond rare genetic disorders. This includes applications in oncology, neurology, cardiology, and infectious diseases.

Which Segment Of Gene Therapies Has The Largest Market Share?

For the purposes of our market research analysis, we segmented the gene therapy market into:

• gene silencing,
• cell replacement,
• gene augmentation, and
• other therapies.

Gene therapy products employing gene silencing mechanisms of action such as SPINRAZA (nusinersen) from Biogen, EXONDYS 51 (eteplirsen) from Sarepta Therapeutics, and ONPATTRO (patisiran) from Alnylam Pharmaceuticals, etc., account for the largest share of the market, owing to factors such as the advantages offered by gene silencing therapies, such as flexibility, versatility, epigenetic modifications, and precise and specific regulation of gene expressions. These therapies are based on the concept of shutting down the activity of genes, either temporarily or permanently, to treat a wide range of genetic and acquired disorders. Gene silencing can be achieved through several mechanisms, including RNA interference and oligonucleotides, which can be adaptable to a wide range of genetic targets, making it suitable for various diseases, including cancer, neurodegenerative disorders, and viral infections.

Viral Vector-based Versus Non-Viral Vector-based Gene Therapies

The range of viral vectors, such as retroviral vectors, adeno-associated viral vectors, and other viral vectors, such as adenoviral vectors and herpes simplex virus vectors, encompass delivery mechanisms designed for both temporary and long-lasting expression. The gene therapy market for viral-vector-based therapies is driven by the advantages offered by viral vectors, such as high efficiency in gene delivery in the target cells, long-lasting gene expression, and high specificity, among other advantages. Retroviral vectors are the major subtype contributing to the growth of this segment. This is mainly due to the use of these vectors for treating cancer, along with the presence of a large number of marketed gene therapies employing retroviral vectors. Some of the examples of viral vector-based gene therapies include LUXTURNA (voretigene neparvovec-rzyl), YESCARTA (axicabtagene ciloleucel), TECARTU.S. (brexucabtagene autoleucel), and ZOLGENSMA (onasemnogene abeparvovec-xioi). Further, with the recent success in commercializing AAV gene therapies, the use of AAV in clinical trials has increased. Therefore, the growth rate of the AAV vector segment in gene therapy is higher compared to the retroviral segment.

Gene Therapy By Therapeutic Area

The therapeutic areas of neurology, oncology, genetic disorders, and rare diseases are expected to see the most significant growth within the gene therapy market. Neurology, including diseases such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), is the largest therapeutic area in the market due to the increasing focus on targeted therapies against neurological conditions and the growing number of regulatory approvals for these therapies. For instance, in June 2023, Sarepta Therapeutics received U.S. FDA approval for ELEVIDYS, a gene therapy for the treatment of DMD.

In the realm of gene therapy development, oncology continues to be the primary focus area. However, the focus is gradually shifting toward other diseases. In the first quarter of 2023, there were 52 trials initiated for gene therapies. The proportion of gene therapy trials for non-oncology indications reached 27% in Q1 2023, up from 15% in Q4 2022.

Commercial Gene Therapies Majorly Employ The In Vivo Delivery Method

Based on delivery methods, the gene therapy market is segmented into in vivo and ex vivo delivery methods. The in vivo delivery method accounted for the largest share of the market in 2022. Currently, many marketed gene therapies employ the in vivo delivery method. Viral vectors (such as herpes simplex virus and adeno-associated virus) and non-viral vectors (such as plasmids, oligonucleotides, and interference RNA) are widely used for in vivo-based gene therapies. These therapies are meant for patients with cancer, neuromuscular diseases, and peripheral artery disease, among others. Commercial in vivo-based gene therapies include ZOLGENSMA (Novartis AG), EXONDYS 51 (Sarepta Therapeutics, Inc.), HEMGENIX (CSL Behring LLC), and LUXTURNA (Spark Therapeutics, a part of F. Hoffman La Roche).

The Effectiveness Of Intravenous Gene Therapy Products Supports The Market Demand

Gene therapies are administered intravenously and through other routes of administration, including subretinal, intrathecal, intralesional, topical, and intratumoral. The intravenous segment accounts for the largest market share owing to factors such as the effectiveness of intravenous administration and the high penetration of intravenous gene therapy. The growing demand for the intravenous route of administration is due to the fact that it is one of the fastest ways to deliver therapeutic products directly into the bloodstream. It has also been observed that certain viral vectors used in gene therapy can only be delivered through the intravenous route. A few of the intravenously administered products recently approved and to be commercialized between 2023 and 2024 include ROCTAVIAN (valoctocogene roxaparvovec-rvox), ELEVIDYS (delandistrogene moxeparvovec-rokl), SKYSONA (elivaldogene autotemcel), and ZYNTEGLO (betibeglogene autotemcel).

The Players Operating In Gene Therapy Market

Novartis (Switzerland), Biogen (U.S.), Bristol-Myers Squibb (U.S.), Pfizer (U.S.), Gilead Sciences, (U.S.), and F. Hoffmann-La Roche (Switzerland) are the prominent players operating in the gene therapy market. Some of the recent strategic initiatives in the gene therapy market are:

• In January 2024, NAYA Biosciences Inc. (U.S.) entered into a binding letter of intent to acquire Florida Biotechnologies, Inc. (U.S.) a gene therapy company focusing on mitochondrial disease treatment.
• In February 2023, Gilead Sciences acquired Tmunity Therapeutics (U.S.) to enhance its capabilities associated with CAR-T therapies and related technologies.
• In January 2023, Sarepta Therapeutics (U.S.) collaborated with Catalent (U.S.) for the manufacturing of its recently approved gene therapy, ELEVIDYS, against DMD.
• In May 2022, Biogen and Scribe Therapeutics (U.S.) entered a partnership to develop a new neurological disease target in gene therapy using the latter’s CRISPR technology.
• In March 2022, Novartis and Voyager Therapeutics (U.S.) entered into a licensing agreement to develop next-generation AAV technologies.

The surge of interest from pharmaceutical companies, venture capitalists, and governmental agencies has resulted in a considerable increase in funding for gene therapy research and development. Series A and seed financings for advanced molecular companies (gene, cell, and RNA therapy companies) in 2023 reached 67 transactions at an aggregate value of $2,977.8 million, representing a nearly 37% increase compared to the value of 2022 (61 transactions worth $2,180.7 million). (Source: American Society of Gene & Cell Therapy (ASGCT). Additionally, startups are also venturing into this modality. Some of the notable examples of startups that have raised financing between $50 and $200 million include:

• CARGO Therapeutics (U.S.) is involved in the development of next-generation CAR T cell therapy using STASH/GAS technology to address resistance and relapse with current CAR-T therapies. In March 2023, CARGO Therapeutics raised $200 million in oversubscribed, upsized Series A financing to advance its pipeline of next-generation CAR T cell therapies.
• Aera Therapeutics (U.S.), focusing on protein nanoparticles for delivery of gene therapy and gene editing systems, raised $193 million from combined Series A and B financings led by ARCH Venture Partners, GV and Lux Capital in February 2023.
• In March 2023, Switch Therapeutics (U.S.) raised Series A funding of $50 million to advance first-of-its-kind RNAi technology.

Conclusion

With ongoing research and technological advancements, gene therapy is set to redefine the future of medicine, bringing personalized, targeted treatments within reach for patients around the globe. The market for gene therapies has witnessed substantial growth, driven by a growing number of regulatory approvals for these therapies, increasing R&D investments by biopharma companies, technological advancements, and an increasing demand for targeted approaches to therapies. Another factor includes a growing number of product approvals for therapies against serious neurological conditions. The gene therapy market specifically is highly consolidated, with a few players dominating the market.

About The Author:

Priyanka Bhendale, assistant manager in healthcare market research at Markets And Markets, has more than nine years of experience in healthcare market research and consulting across the pharmaceutical, biotechnology, and medical device verticals. She has expertise in market assessment, market sizing and forecasting, end user perception analysis, opportunity analysis, and competitive intelligence studies. With her expertise in qualitative and quantitative research and analytical skills, she also has worked on growth engagements across bioprocessing technologies, cell culture technologies, contract research, and manufacturing.