From roadmap to research
Our Cure Roadmap recommendations are being put into motion in established contract research labs (CROs) and in pop-up lab spaces where families/foundations are in control.
Perlara is now operating a decentralized lab at S.T.E.A.M. Lab (aka SFBL), a biotech incubator and workspace in San Francisco where we currently have fibroblast and yeast capabilities.
Two of the Cure Roadmaps we produced on behalf of entrepreneurial families/foundations are now in the execution phase, meaning the research recommendations we made are being put to the test in the laboratory.
The PLOD1-kEDS Cure Roadmap called for a set of proof-of-concept experiments to establish the feasibility of an exon-skipping ASO approach and an enzyme replacement approach using patient-derived fibroblasts. Last week, a shipment of several patient-derived PLOD1 fibroblasts were shipped to SFBL from Coriell. We received the cells and started the process of expanding them and cryopreserving vials of cells for future experiments, and safekeeping.
At the same time, the Cure KCNH1 Foundation for whom we produced the KCNH1 Cure Roadmap, embarked on their allele-agnostic ASO project with a San Diego-based CRO called iXCells. Last month, the family behind Cure KCNH1 visited iXCells to get a tour of the lab and meet the team:
As these projects advance in the lab, we’ll provide updates on results. And as other Cure Roadmap projects transition into research operations mode, we’ll share updates on their progress as well.
The floodgates are open: as BioPharma companies retreat from the ultra-rare disease space, families and foundations are rushing in to fill the gaps. Perlara stands ready to partner with entrepreneurial families/foundations who don’t take no for an answer.
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