Financial boost to tackle autism with precision medicine

Autism spectrum disorders (ASD) reflect a critical gap in medical care, often involving challenges in social communication, with limited treatments available for core symptoms. A key barrier to developing effective therapies is the incomplete understanding of ASD's underlying biological causes.

Established in early 2022, Translation-X is exploring a novel therapeutic approach by targeting selective mRNA translation. Its research has identified dysregulated mRNA translation as a core issue in a sub group of ASDs, with a particular focus on Map-kinase interacting kinases (MNK) as a drug target. By inhibiting MNK, Translation-X aims to restore normal mRNA translation and neural function, offering a promising treatment that directly addresses the root pathology of a genetically defined sub-group of autism.

Accordingly, 1 in 100 children globally are affected by ASD, presenting a significant unmet need for targeted treatments. Translation-X addresses this need by developing brain-penetrant MNK inhibitors to target core symptoms. Focusing initially on rare genetic conditions, the biotech startup is developing a phenotyping approach for a mechanistically defined larger target population. These treatments could benefit from orphan drug designation, providing both a strategic market entry and a foundation for expansion.

The startup has won the Venture Kick finale, securing CHF 150,000 to further develop its lead drug candidates, focusing on refining MNK inhibitors for treating core symptoms of ASD. The funds will also support business development activities, helping the company prepare for future investments and partnerships.

Behind Translation-X is a team comprising Özgür Genç (senior scientist and project leader), Prof. Peter Scheiffele (scientific advisor), and Guido Koch (medicinal chemistry advisor). Additionally, faculty members Prof. Martin Smiesko and Prof. Markus Lill from the Pharmazentrum contribute computational chemistry expertise to the project.

"Venture Kick helped us better understand the business challenges of the startup world. Throughout the three stages, we benefited from its structured approach, which helped us focus on essential business tasks like defining the business model and preparing for investment readiness," highlighted Özgür Genç. "It also introduced us to key experts and networks, opening valuable opportunities for us. The funds were effectively used to support the development of Translation-X."

(Press release/RAN)
Image: Peter Scheiffele, Özgür Genç, and Guido Koch