As the US Food and Drug Administration approval of Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) has generated excitement for gene therapies in general, it is worth looking into how strong the pipeline is for the central nervous system (CNS) space where such therapies are being developed.

What’s in the global pipeline?

According to GlobalData’s Pharma Intelligence Center, 14 CNS indications are the subjects of gene therapy-based pipeline products in the eight major markets (the US, France, Germany, Italy, Spain, the UK, Japan and China).

Parkinson’s disease is the top CNS indication, followed by metachromatic leukodystrophy (MLD) and Batten disease. Among the 29 products that were analysed, 72% are in Phase II development, followed by 21% in Phase I development. The remaining products that are in late-stage development include Novartis’ Zolgensma, which is in pre-registration stage in Europe and Japan for spinal muscular atrophy (SMA), and Orchard Therapeutics’ OTL-200, which is being developed for MLD and is currently in Phase III.

Adeno-associated virus-based vectors are the most dominant delivery platforms among the pipeline products for CNS indications. From a developer perspective, 81% of the developers of gene therapies in CNS are pharma companies, and the remaining are represented by academic institutes or medical centres.

From a geography perspective, approximately 67% of these developers are headquartered in the US, followed by 10% of developers each in China and the UK, and 5% each in Japan, Netherlands and South Korea.

Given that the understanding of gene therapy mechanisms is expected to expand as more of these therapies enter the market, GlobalData believes that companies, academic institutes and medical centres with a focus on gene therapy programmes and platforms will quickly become attractive partners for collaborations with bigger companies or possible buy-outs.

Upcoming related reports
GlobalData (2019). Gene Therapies in Neurology, to be published