Increased investment spurs advances in gene therapy

The success of approved products for rare illnesses has opened the door for gene-based treatments of more prevalent diseases, finds Frost & Sullivan.

Analysis from Frost & Sullivan, Advances in Gene Therapy, finds gene therapy can be effective for several emerging disease groups, including ocular diseases, diabetes, infectious illnesses and many of cancer types. Gene therapy for rare genetic diseases has been the most fruitful in terms of clinical trials. The success of these niche therapeutics has paved the way for similar applications for additional widespread diseases.

Until now, adverse side effects, as well as high costs, had made regulatory bodies and patients skeptical about gene therapy, while health insurance companies had been reluctant to provide reimbursements.

However, an increasing number of collaborations among research and pharmaceutical companies to understand the potential applicability of gene therapy for prevalent diseases, such as cancer, will help develop novel and approved modes of treatment.

"With the rise in clinically proven remedies, it is likely insurance companies will begin to acknowledge the commercial advantage of gene therapy," said TechVision Senior Research Analyst Madhumitha Rangesa.

Improvements in vector technology and delivery systems will further enhance efficacy and clinical outcomes. Currently, the ocular diseases space is witnessing a rapid influx of companies equipped with the appropriate vector delivery systems capable of carrying small gene loads to specific target sites in the human body.

The use of combination therapies is another novel idea that will boost the therapeutic effects of gene technology. Competing industries, like cancer immunotherapy, are already shifting towards combination therapies within their own industry, with conventional or other burgeoning therapeutic fields.

Once the gene therapy industry bounces back with noteworthy products, governments around the world will step in to accelerate innovation. In the U.S., the growing emphasis on precision medicine capable of catering to a specific subset of people based closely on their genetic makeup is widening the scope for gene-based therapeutics.

"North America has the highest number of clinical trials in gene therapy largely due to the extensive focus on innovation and availability of funding," noted Rangesa. "In terms of market commercialization, Europe has overtaken North America with two products approved for commercialization within Europe and one approved for commercialization globally; however Asia is slowing catching up."

The global interest in the technology will attract investments and support the evolution of gene-based remedies from production and clinical trials to commercialization.