Clarity in cell line development

Gene therapy in 2019: did it deliver?

Gene therapy was a hot topic in 2019, with many notable advances in the development of new medicines that could provide life-saving treatment for previously incurable diseases. There are many complex technical challenges involved in producing gene therapies, including the need for stable producer cell lines for viral vectors, resulting in close ties between gene therapy and cell line development communities. In this blog we look back at 2019, exploring the progress in gene therapy to date, as well as looking forward to what the future might hold for the sector.

Gene therapy is here to stay
Since the first gene therapy clinical trials took place in the 1990s the technology has advanced greatly and has been applied to a wide range of diseases from blindness to cancer. This year there have been further notable advancements in the development of new gene therapies to treat serious and life-altering diseases. Bluebird Bio’s (Zyntegio) gene therapy for thalassemia and sickle cell disease eliminated serious pain crises and the need for red blood cell transfusions in a small study of patients[1]. In May 2019, the US Food and Drug Administration (FDA) approved Zolgensma®, a gene therapy by AveXis, a Novartis company, for the treatment of paediatric patients with spinal muscular atrophy (SMA)[2]. Another gene therapy to treat Duchenne muscular dystrophy successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease without triggering immune responses known to hinder previous therapeutic approaches[3](results reported in mice and dogs). In another study, researchers reported promising results in a Phase I gene therapy clinical trial for Tay-Sachs, an inherited metabolic disorder causing early childhood death[4].

There have also been notable developments in technologies for the delivery of gene therapies. For example, Johns Hopkins Medicine researchers have developed a method to deliver sight-saving gene therapies to the retina in experiments using a small needle to inject harmless, genetically engineered viruses into the space between the white of the eye and the eye’s vascular layer[5]. The new injection is less invasive, safer and more accessible than current techniques and could provide a new, more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration. Another innovative gene therapy delivery method is the use of nanocapsules packed with gene-editing tools[6]. This method could overcome many of the problems associated with typical methods for delivering gene therapies to specific tissues in the body, which can be complicated and cause troubling side effects.

Acquisitions by big pharma are on the rise
2019 saw several major acquisitions of gene therapy companies by Big Pharma. In June 2019, Biogen announced that it had completed its acquisition of Nightstar Therapeutics, a clinical-stage gene therapy company which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. In December 2019, Roche acquired Spark Therapeutics, Ltd, a company committed to discovering, developing and delivering gene therapies for genetic diseases, including blindness, haemophilia, lysosomal storage disorders and neurodegenerative diseases[7]. In the same month, Astellas Pharma Inc announced their acquisition of Audentes Therapeutics Inc, a leading AAV-based genetic medicines company focused on developing and commercialising innovative products for serious rare neuromuscular diseases[8].

Skilled people are in demand
The future for the cell and gene therapy sector looks bright, with high growth predicted and many innovative new technologies in the pipeline. A report published in November 2019 by the Cell and Gene Therapy Catapult (CGT Catapult) has shown that the UK is set to more than double its cell and gene therapy employment by 2024, as more therapies move towards commercialisation[9]. In the last seven years, the industry has expanded from 500 jobs in 2012 to over 3,000 in 2019 and is predicted to reach over 6,000 jobs by 20246. To put the year into perspective, at the end of Q3 2019: there were 106 companies in Europe developing gene therapies; there were 5 near-term approvals in Europe; and there were 57 Phase 2 clinical trials and 20 Phase 3 trials for gene therapy products in Europe, mostly treatments for rare cancers and haematology, demonstrating an increase in the scale in companies in the field.

Production capacity needs to catch up
However, the industry faces a major challenge: the process development production capacity for gene therapy products is a major limiting factor which is causing a backlog for new gene therapies and placing huge pressure on companies to find innovative ways to bring their products to market quicker. For instance, Thermo Fisher acquired Brammer Bio in order to help the company address the long-term challenge of producing viral vectors at required scale[10]. Similarly, Novartis is building a new $90 million cell and gene therapy factory in northern Switzerland to begin commercial production of its cell therapy Kymriah for cancer in 2020[11], allowing the drug maker to make its Kymriah treatment for European patients without first having to fly their immune cells across the Atlantic Ocean.

More complex conditions are next
Where will gene therapy go next? With the growing range of tools and technologies available, it is becoming easier for scientists to develop gene therapies for parts of the body that have traditionally been difficult to target, such as the heart, lungs or the pancreas. Gene therapy is expanding beyond the realm of well-understood diseases with a single mutation cause to more complex conditions that were previously unreachable. Possible targets for future gene therapies include epilepsy, Parkinson’s and chronic pain, although such research is still in very early stages and presents unique challenges such as crossing the Blood Brain Barrier. We’re excited to see what 2020 holds for gene therapy.

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