RARE Daily

Vanda and OliPass Partner to Develop Antisense Oligonucleotide Therapeutics

September 30, 2022

Vanda Pharmaceuticals has entered into a research and development collaboration with South Korean biotech OliPass Corporation to jointly develop a set of antisense oligonucleotide molecules based on OliPass’ proprietary modified peptide nucleic acids.

The partnership leverages the respective strengths of Vanda and OliPass to support the development of ASO-based precision medicine therapeutics and potentially create value opportunities for both companies.

Using Olipass’s Olipass Peptide Nucleic Acids (OPNA) technology, the collaboration will focus on editing and modifying gene expression using ASOs in disease states where the expression of genes is either altered or the sequence of the expressed genes can be altered for therapeutic benefit.

“Olipass’ delivery platform holds the promise of efficient delivery of our ASO’s in pursuit of the development of precision therapeutics,” said Mihael Polymeropoulos, president, CEO and chairman of Vanda Pharmaceuticals.

OPNAs are selectively modified to enhance both stability and cell permeability of peptide nucleic acids while maintaining very high binding affinities to target nucleic acids. Therapeutic efficacy for OPNAs has been observed with dosages as low as 10 ng/kg in animal models, showing effects at dosages many orders of magnitude lower than previously achievable with existing ASO technologies. Furthermore, OPNAs have the capacity to bind to pre-mRNA in the nucleus and enable targeting pathogenic variants and conditions that were previously untreatable.

Vanda has already identified two ASO targets that have been validated in cell lines that model two undisclosed disease targets, one rare orphan and the other applicable to a broad set of immuno-oncological conditions. Vanda’s partnership with OliPass to enhance the existing ASOs with OPNA chemistry is the next step to take these preclinical findings to in vivo and clinical testing.

Mihael Polymeropoulos, president, CEO and chairman of Vanda Pharmaceuticals.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.