Gene therapies have been garnering attention due to the high costs attached to recently approved therapies and the potential for these therapies to positively transform the treatment landscape of various diseases. 

Because these therapies are expected to bolster treatment options in the future, it is worth looking at the clinical trials happening in various neurology indications to understand trends in this area. 

An analysis of 63 planned, ongoing, and completed trials listed in GlobalData’s Pharma Intelligence Center revealed that 84% of these trials are in early-stage development (Phase I, Phase I/II, and Phase II). 

Figure 1 and 2 show the percentage split of these trials based on trial phase and trial status. Big pharmaceutical and small biotech companies are collectively responsible for sponsoring 79% of these trials. This dominance is expected to continue as different types of strategic deals between companies help strengthen their gene therapy portfolios and commercial operations.

A total of 54% of these trials are being conducted in the US, followed by 32% in the five major European markets (5EU: France, Germany, Italy, Spain, and the UK), and 5% each in China and Japan. It was also observed that clinical trials evaluating viral-based vectors had a longer duration compared to non-viral vectors; currently, viral vectors dominate the pipeline of gene therapy products that are in development for neurology indications. The top three neurology indications with the most clinical trials were found to be Parkinson’s disease, pain, and amyotrophic lateral sclerosis (ALS). 

Figure 1: Percentage of clinical trials by trial phase for neurology indications

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Source: GlobalData            ©GlobalData


Figure 2: Percentage of clinical trials by trial status for neurology indications


Source: GlobalData     ©GlobalData

As more gene therapy-based clinical trials are expected to be conducted in the future, GlobalData believes that industry participants and academic institutes with a focus on gene therapy programmes and platforms are likely to become attractive partners for collaborations with bigger companies or possible buy-outs.  

Upcoming reports

GlobalData (2019). Gene Therapies in Neurology – Hot Topic Report, to be published.