Posted by Friends of FSH Research on Nov 20, 2021
Progress report by Charles Marusak
miRecule has conducted an extensive bioinformatic analysis of the expression of disease genes in FSHD patients enabling us to design RNA therapies that can block DUX4, and additional genes that cause inflammation and pain advancing FSHD. In our previous project update we reported on creating a library of RNA therapies for the treatment of FSHD, and early testing of this library.
We have also developed research tools that enable high-throughput screening of DUX4 inhibiting compounds in muscle cells. Once fully validated this assay can be used to screen our hundreds of anti-DUX4 ASOs and then our ASO-antibody conjugates. We plan to release this assay to the research community in the coming years to enable others in the field to use it.
Following our in vitro screening, in the next phase of our development we will test our lead candidates in mouse models of FSHD demonstrated proof of concept they can treat the disease.
We are very fortunate to have been able to receive early funding from the Friends of FSH Research in the development of our research program, and the initial data developed with this grant has enabled us to win an large $3.7 M SBIR grant from the NINDS to further develop the antibody side of our antibody-ASO conjugates for the treatment of FSHD.
This support as well as from other parts of the community has allowed us to flesh out our therapeutic and dramatically increase its likelihood of success. In the next year we hope to have data of our ASO-antibody conjugates in mice, demonstrating their clinical potential.
See original grant Antisense Oligonucleotide-Antibody Conjugates to Treat FSHD