GFPD Board Member, Corin Chapman and Catherine Argyriou, postdoctoral fellow, Braverman Lab, McGill University recently presented at the World Orphan Drug Congress in Boston. Corin and Catherine shared information about the GFPD and the advancements made toward treatments for peroxisomal disorders including retinal gene therapy and small molecule therapies.
Catherine shared, “We are planning IND-enabling studies for retinal gene therapy and planning what a clinical trial could look like with potential endpoints. Our current investors are interested in staying on for the next round, however, we will need an industry partner or additional investment to make this all possible.
If gene therapy isn’t for you, Diosmetin, a drug approved by the EMEA for use in pregnant women, has been shown to improve peroxisome function in select mutations in PEX 1, PEX 6 and PEX 26. Diosmetin has recently been reformulated to improve bioavailability in the mouse. Recently, a company was formed around this asset, but due to a subsequent change in strategy, Diosmetin remains available, unencumbered.”
If you are interested in learning more about the opportunities to partner with the GFPD and push ahead potential treatments for individuals with peroxisomal disorders, please contact Catherine Argyriou at email@example.com or Corin Chapman at firstname.lastname@example.org