Israel-based Skip Therapeutics and Sheba Medical Center in Ramat Gan have joined forces on the development of RNA-based treatments, specifically antisense oligonucleotide (ASO) therapies, for rare genetic diseases.

As part of the agreement, Sheba will provide Skip with its de-identified patient mutation repository, and Skip Therapeutics will conduct a bioinformatic assessment to determine the feasibility of using ASOs.

The Israeli company leverages its proprietary bioinformatic engine to pinpoint the most promising ASO-based molecular strategies tailored to specific patient sub-populations based on their unique mutations.

Skip Therapeutics chief scientific officer Ariel Feiglin said: “Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at relatively low costs to restore protein function of mutated genes.

“This presents exciting possibilities of drug development for small patient cohorts and eventually even for single patients.

“To effectively realise these possibilities, it is necessary to incorporate computational tools in the developmental process, and we believe that our computational approach will enable an effective and rapid development of ASO-based treatments.”

The move is expected to bypass the mutations, restore protein function, and establish effective therapeutic approaches for rare diseases.

Currently, Skip Therapeutics is identifying targets and conducting in-vitro functionality studies in collaboration with various hospitals and academic institutes worldwide.

Sheba Medical Center haematology laboratory director Dr Dan Dominissini said: “Skip Therapeutics' novel computational engine will enable us not only to determine the most appropriate therapeutic design for a given genetic disease but also to directly identify treatable patient populations, thereby maximising efficiency and minimising costs.

“We look forward to propelling gene therapy forward as a result of this exciting collaboration.”