Gene therapy has been on the rise the first human gene therapy trial was conducted three decades ago. Over the past five years, gene therapy has garnered over $600 million in venture capital funds. Many countries are focusing on projects in the field, particularly the UK, which predicts that by 2035 the region may have a 15% stake in global cell and gene therapy activities, $10 billion in revenue and 18,000 jobs. As of June last year, 59 cell and gene therapy trials were active in the UK, with 10 trials for in vivo gene therapies.

Since gene therapy involves long-term therapeutic effects, regulatory issues may pose a challenge, for regulators will have to make long-term decisions based on limited data. Also, once approved by federal agencies, gene therapy products will always be costly to produce and supply, which will require creative payment models for insurers and patients. However, experts in the industry hope that the cost of gene therapies will eventually reduced to an annual costs comparable to a regime of a monoclonal antibody.

Globally, there is a lack of capacity for clinical-grade vectors, resulting in small- and medium-sized businesses having to compete to get their products manufactured. But experts predict that there may be an oversupply of gene therapy products in the market within the next few years that will bring the prices down, providing greater opportunities for smaller businesses to produce their therapeutics.

A critical component to progressing gene therapy is technology advancements. Currently, a batch of viral vector that is large enough to treat up to six patients can cost up to $2 million from a contract manufacturer, which means that for a clinical trial, millions of dollars may have to be spent just to source the viral vector. Similar to the evolution of monoclonal antibodies, scalable platform processes are extremely important to the advancement of gene therapies, such as large bioreactors for adherent cells or a customized process that allows cells to be cultured in suspension.
Source: Chemistry World