What Could RNA Therapies Do for the Treatment of CNS Disorders?

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Evelyn chatted with Thomas de Vlaam to find out what his company, Amylon, is working on and how it hopes to change the field fighting CNS disorders. 

De Vlaam came up with AT-010, a drug candidate for the treatment of Katwijk‘s disease, during his time at ProQR. When it became clear that he had something huge on his hands, he led the charge as CEO to form Amylon. The company, based in Leiden and formed only last month, focuses on CNS disorders using an ultra-genetics approach. CNS research and companies are often met with skepticism, and De Vlaam didn’t expect the enthusiasm and excitement following the spin-out but he found it “exhilarating”.

Despite Alzheimer’s being one of the obvious targets for a biotech fighting CNS disorders, Amylon chose Katwijk‘s disease – an inherited orphan disease that causes cerebral hemorrhages. De Vlaam explained that he was born and bred in an orphan disorders company so was motivated to fight the unmet need of a disorder that “[would] otherwise never [have] seen a glimmer of hope for a potential therapy because it is overshadowed by a disease like Alzheimer’s.”

Amylon uses RNA, a field boosted by the news of Alnylam‘s positive Phase III results. De Vlaam commented: “It has been a long time coming… It’s the best sort of motivation that a company like Amylon can have.” His sense of the field’s momentum convinced him that it was the right time to spin out.

De Vlaam is not surprised that RNA has become a popular approach: “Genetic disorders can often be boiled down to a single protein malfunction, and working backwards from protein malfunction… to gene therapy… there was a logical result that ultimately people started looking at RNA.” But, it wasn’t always plain sailing with early barriers like immunogenicity preventing the use of RNA. Now, clever chemistry has overcome these, opening the door to their use once again.

Amyloid plaques indicate that a protein has gone wrong and is not functioning properly, and they are a characteristic of a number of CNS disorders. De Vlaam told Evelyn that RNA therapeutics allows you to step in before these plaques form: “With genetic disorders, the mutation predetermines that the proteins will start aggregating. Being able to interfere with the production of these proteins provides with the opportunity to prevent the toxic process from ever taking place”.

By taking on a rare disease with relatively few patients, Amylon has limited space to recoup its initial investment. De Vlaam explained that Amylon plans to do this by applying its findings to other CNS disorder that share characteristics.

Despite recent disappointments in the field, including Axovant’s Alzheimer’s drug failure on top of Eli Lilly’s, De Vlaam‘s take-home message was positive. He revealed that there is a companionship between companies taking on CNS disorders: “There is far more to learn than to lose from each other… We are received in a field where everyone is trying to get these drugs to patients and find solutions for disorders that have never had hope…”

Image – jm1366 / shutterstock.com

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