The recent FDA approvals of the first two gene therapies marked a groundbreaking new era for the medical world. Both therapies fall within oncology: Novartis’s Kymriah, approved in August 2017, aims to treat pediatric patients with acute lymphoblastic leukemia (ALL), while Gilead/Kite Pharma’s Yescarta, approved in October 2017, is targeting adults with relapsed or refractory large B-cell lymphoma.

The CNS market is now expected to follow suit, with the first CNS gene therapies entering the market in the near future. Although curative therapies still do not exist for the vast majority of CNS diseases, gene therapies offer a promising new approach to the treatment of various neurological disorders, either by inhibiting disease-causing proteins or by enabling the expression of missing proteins. The blood-brain barrier (BBB) has presented a unique physiological barrier for these indications. The BBB plays a central role in regulating the chemical micro-environment of the brain by preventing diffusion of most small molecules circulating in the blood. A second challenge is also presented by the fact that some neurology indications, such as Huntington’s disease, affect very large brain areas, and therefore the transgene would need to be distributed to the whole brain.

Over the last decade, however, recombinant adeno-associated virus (rAAV) has emerged as an excellent tool for gene delivery to the CNS. AAV is a small harmless virus that can transduce both dividing and non-dividing cells, a useful feature for delivering transgenes to non-dividing neuronal cells. There are two main methods of gene delivery to the CNS using AAV vectors: either a brain-directed local therapy, or a less invasive systemic therapy. As identified by GlobalData’s Clinical Trials Database, the table below shows that nearly half of the gene therapies in development for CNS disorders are utilizing AAV—if the preclinical successes can now be translated in the clinical arena, this field will continue to generate optimism and perhaps even an end to many devastating CNS diseases.


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