A new initiative for rare diseases called the New Modalities Ecosystem’, launched in 2018 by the Finnish pharmaceutical company Orion, and supported by Business Finland, aims to exploit Finnish expertise to deliver new treatments and technologies for rare diseases. The project aims to unite academia with industry; bringing together academics, businesses and drug developers to help improve understanding of disease pathology and contribute to the development of better treatments.

It is initially targeting rare diseases that are part of the Finnish disease heritage, i.e. genetic diseases that are significantly more common in Finland than they are in any other population. This includes a form of ataxia known as mitochondrial recessive ataxia syndrome (MIRAS). MIRAS is a mitochondrial disease caused by mutations of the POLG1 gene. It includes sensory ataxia neuropathy dysarthria and opthalmoplegia (SANDO) and spinocerebellar ataxia with epilepsy (SCAE) and is a common cause of autosomal recessive juvenile, or adult-onset, ataxia in Scandinavia.

Researchers at the University of Helsinki have been investigating the disease mechanisms of MIRAS and have already made valuable new insights. They will expand upon their findings as part of the New Modalities Ecosystem and, in conjunction with Orion, apply their results for the benefit of patients.

Dr Amir Snapir, Orion’s rare diseases development director says: “Breaking down the walls between pharma companies and academia – as well as technology companies, who will be providing, for example, diagnostics or measurement tests – will be of enormous benefit to drug development programmes for rare diseases, which need to find a way to maximise benefit to the patients but at the same time control costs of development.”

View the original article from Health Europa here.