A mid-sized pharmaceutical company was considering the option of expanding into developing gene therapies for CNS diseases. Alacrita was asked to help them evaluate and prioritize the technical feasibility of correcting various CNS and musculoskeletal diseases with gene therapy modalities and map the landscape of potential partners and competitors developing attractive assets for the prioritized indications.
First we developed a list of technical considerations including gene size, mutation characteristics (i.e., loss or gain of function), inheritance, number of mutations, availability of strategies to correct mutations, etc. These technical considerations were then judged for each disease and gene therapy technology, and assigned a score of high, medium, and low. This enabled us to derive a comparative technical fit score. Each indication was then ranked accordingly and an asset landscape was provided to guide potential partnering considerations for the indications with the greatest technical feasibility of correction using a gene therapy approach.
We can help you gauge and rank the opportunities for your R&D programs or technology platforms to deliver value, by framing the commercial potential and charting the path to market.