Why gene therapy is set to transform medicine

Posted Jan 14, 2020 by Tim Sandle
Advances in gene therapy offer the possibility to transform medicine. These types of medicines were given their first market approvals in 2017, and since then sector growth has accelerated.
China has now halted human gene-editing using the Crispr techonology  after widespread outrage
China has now halted human gene-editing using the Crispr techonology, after widespread outrage
Gene therapies are in the in-demand medicine, due to their potential to treat life-threatening diseases, including illnesses classified as ‘rare genetic diseases’. Examples include treating sickle cell anemia and anti-tumor immunotherapy. As well as being demand, the market is also lucrative, expected to grow to $11.96 billion by 2025.
The growth with gene therapies is represented by the range of gene therapy products in the clinical pipeline, where Frost and Sullivan estimate some 400 cell and gene therapy products in development. The last year alone has seen a 27 percent increase in the number of clinical trials involving gene therapy products. This is also reflected with the number of emergent start-up companies entering the gene therapy space.
Most start-up companies elect to outsource the downstream manufacturing stages, due to the relatively low costs involved, as off-set against the costs involved with constructing specialist facilities.
Big Pharma is also investing in the field. For example, Pfizer has injected $500 million into one of its plants in North Carolina and Fujifilm Diosynth has made a similarly large investment to boost production capability.
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The growth has been fuelled by support from governments and regulatory agency. Here the U.S. 21st Century Cures Act as been a key driver, supported by the U.S. Food and Drug Administration (FDA).
There are, however, some limitations to be overcome.
One limitation with progressing gene therapy products is with costs and pricing structures, which is resulting in many therapies being unstainable. This is also limiting the transition to scale, given the complexities tied to growing, sourcing and transporting cells. Costs are high partly as a result of the development and partly because many gene therapies offer curative solutions, making them an in-demand and expensive medicine.
A solution to the costs factors is, according to Vered Caplan (Orgensis Inc) the development of point-of-care automation, including the application of closed systems for processing cell therapies. These systems require the use of fewer personnel and permit real-time quality control testing to take place.
A second limitation is with the availability of rare materials of sufficient quality, such as plasmid DNA and tranfection agents), which need to be manufactured under the same standards applied to mainstream pharmaceuticals (that is, Good Manufacturing Practices.
With new technology emerging to improve product yield and to drive down costs, coupled with increased investments, the expansion of gene therapy products looks set to be one of the big drivers within healthcare over the next five years.