The conception of gene therapy in the 1960s and the development of viral vectors for gene therapy have paved a way to treat a variety of heritable and acquired diseases.
Viral vectors are modified viruses developed using sophisticated DNA recombination techniques. This alteration makes the viruses safe for the transfer of genes of interest to target cells or tissues for therapeutic purposes. Various types of viruses (e.g., adenoviruses, retroviruses, poxviruses, adeno-associated viruses and herpes simplex viruses) are currently being investigated for their potential in gene therapy.
The approvals of gene therapies, such as Kymriah, Yescarta, Luxturna, Strimvelis and Imlygic, by the U.S. FDA and EMA over the past 3 years have boosted the research on viral vector–based gene therapies globally. More than 500 potential gene therapies for cancer, genetic disorders and infectious diseases are currently under development, with approximately 30 products in the most advanced stages of development.
Leveraging a Cure
Cancer is a major public health concern globally, and innovative technologies are being leveraged to find a cure. Viral vectors are being explored for applications in cancer therapies; around two-thirds of the research in gene therapy is currently directed towards oncology. Kymriah, the first-in-class lentiviral vector-based CAR-T cell gene therapy marketed by Novartis, was approved by the FDA in 2017 for acute lymphoblastic leukemia (ALL) and large B cell lymphoma. This has attracted considerable attention to the further development of CAR-T cell therapies globally. Gene therapy companies, such as Juno Therapeutics (U.S.), bluebird bio (U.S.), Ziopharm Oncology (U.S.), Bellicum Pharmaceuticals (U.S.) and Cellectis SA (France), have already invested in CAR-T cell research. Rare diseases are another major topic of interest in gene therapy, as 350 million patients are diagnosed worldwide, and there is a lack of available, effective treatments. The growing burden of these diseases is a major driver for investment in viral vector-based gene therapies.
Viral vectors are preferred for gene transfers owing to their high transfection efficiency and stable expression. Additionally, they enable successful and effective gene delivery unhindered by various extracellular and intracellular barriers. The popularity of viral vectors in gene transfer is evident in the registration of around 2,600 clinical trials on vector-mediated gene therapy through 2017.1 Additionally, there is a surge in investments for the development of gene therapies. The Alliance for Regenerative Medicine (ARM), a U.S.-based non-profit organization in the field of advanced therapy, reported a 164% increase in funding for gene and gene-modified cell therapy in 2017 over 2016 and a global investment of USD 4.5 billion in 2017.2
Investigating Viruses for Gene Transfer
Lentiviruses, adenoviruses, adeno-associated viruses (AAVs) and viruses such as vaccinia virus, poxvirus, herpes simplex virus, measles virus, cytomegalovirus, flavivirus, rhabdoviruses, Newcastle disease virus, picornaviruses and alphavirus are being investigated for gene transfer. Lentivirus –– a type of retrovirus –– has been successfully used in the approved CAR-T cell therapies Kymriah and Yescarta. AAVs have gained popularity recently, owing to their suitability for most approaches to cell-based gene therapies. AAV vector clinical trials target monogenic diseases, such as Sanfilippo syndrome, Factor IX deficiency and Duchenne muscular dystrophy (DMD); ocular diseases, such as retinitis pigmentosa and age-related macular degeneration (AMD); and neurodegenerative diseases. Along with gene therapy applications, some viral vectors, such as vaccinia virus, CMV, Sendai virus and adenovirus, are being developed to immunize patients against HIV-1, influenza, hepatitis, tuberculosis and malaria.
The booming gene therapy market has created opportunities for the engineering and manufacture of viral vectors. The viral vector manufacturing market is highly competitive, with a few established players, mid-tier companies and startup firms. Prominent players in the market include Brammer Bio (U.S.), Lonza (Switzerland), Merck (Germany), Oxford BioMedica (UK), CGT Catapult (UK), Spark Therapeutics (U.S.), Kaneka Eurogentec (Japan), FUJIFILM Diosynth Biotechnologies (U.S.), Novasep (France), Cobra Biologics (UK), FinVector (Finland), REGENXBIO (U.S.), uniQure (Netherlands) and MassBiologics (US). Collaborations, partnerships and agreements with pharmaceutical and biopharmaceutical companies and research institutes have proven to be the major growth strategy adopted by viral vector manufacturing companies in this market over the past three years. Other key growth strategies include expansion of production sites in high-demand markets, such as the United States and the UK, and acquisitions of companies to fulfill the growing requirements of viral vectors and to broaden product pipelines..
According to a recent research study published by MarketsandMarkets, the viral vector manufacturing market is projected to reach USD 815.8 million by 2023 from USD 327.8 million in 2018, at a compound annual growth rate (CAGR) of 20.0% during the forecast period (2018–2023).3
While the market is driven by the rising prevalence of cancer and genetic disorders, the availability of funding for gene therapy development, the effectiveness of viral vectors in gene therapy delivery and ongoing research into viral vector–based gene and cell therapies, the high costs of gene therapies (currently ranging between USD 500,000 and USD 1.5 million) and short shelf-life of viral vectors is currently a matter of significant concern to viral vector manufacturers.
References:
- Samantha L., Anais K. Amaya, Ian E. Alexander, Michael Edelstein, Mohammad R. Abedi. “Gene therapy clinical trials worldwide to 2017: An update.” The Journal of Gene Medicine. 20: e3015 (2018).
- Quarterly Data Report: Q2 2018. Rep. Alliance for Regenerative Medicine. 8 Aug 2018. Web.
- Viral Vector Manufacturing Market by Type (Retrovirus, Gammaretrovirus, AAV), Disease (Cancer, Infectious Disease, Genetic Disorders), Application (Gene Therapy, Vaccinology), End User (Biotech companies, Research Institutes) - Global Forecast to 2023. Rep. Markets and Markets. Aug. 2018. Web. https://www.marketsandmarkets.com/Market-Reports/viral-vector-manufacturing-market-89341986.html
