Research & Development
VICO Therapeutics receives orphan-drug designation from FDA for VO659
29 July 2021 -

Netherlands' biotech company VICO Therapeutics reported on Thursday the receipt of the orphan-drug designation for its investigational VO659 for the treatment of Huntington's disease (HD) from the US Food and Drug Administration (FDA).

Huntington's disease (HD) is a rare inherited neurodegenerative disorder that has a progressive and fatal course characterized by movement disorders, cognitive impairment, dementia and psychiatric manifestations including depression and psychosis, added the company.

This follows VO659's the month old orphan-drug designation from the USD FDA for the treatment of spinocerebellar ataxia (SCA), said the company. The new designation completes the quartet of orphan designations for VO659 in HD and SCA in both the US and EU.

The company revealed the lead antisense oligonucleotide (AON) product, VO659, is reportedly designed to suppress mutant proteins and slow or halt disease progression. A major strength of its approach is the broad applicability to different polyQ diseases and the preferential reduction of mutant relative to normal polyQ proteins.

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