FSHD Global Research Foundation is investing in the future of FSHD treatment
FSHD Global Research Foundation (Sydney, Australia) is excited to announce its investment in US-based Armatus Bio (Columbus, Ohio), a leader in precision-targeting of microRNA treatments for neuromuscular disorders.
“Alongside our investments in small molecules, AI-enabled clinical trial technologies and next-generation therapeutics, Armatus Bio adds an exciting precision RNAi platform targeting DUX4. By supporting multiple scientific approaches, we increase the likelihood of delivering meaningful treatment options for the entire FSHD community. FSHD Global’s investment strategy is deliberately diversified because we know there is unlikely to be a single solution for every person living with FSHD,” says Emma Weatherley, CEO of FSHD Global.
The ARM-201 therapy is a vectorized microRNA engineered with a second-generation myotropic capsid; it has been designed to effectively, safely, and durably silence toxic DUX4 expression that is the primary driver of FSHD muscular dystrophy.
A robust series of preclinical studies have strongly supported a positive profile for ARM-201, with clear improvements in FSHD-linked biomarkers, as well as neuromotor and behaviour improvements. Large animal studies, and now clinical experience with their SLB-101 capsid (POLARIS-101TM, licensedfrom Solid Bio), have shown highly effective biodistribution to muscles with no major safety signals.
ARM-201 has completed preclinical studies and successful pre-IND engagement with the U.S. Food and Drug Administration (FDA) and has received both Orphan Drug and Rare Paediatric Disease designations. They are now scaling for clinical studies.
“We are deeply grateful to FSHD Global for their belief in our precision approach. This strategic investment provides vital support as we advance from the lab into clinical studies—helping us evaluate how this therapy could transform care for people living with FSHD who currently have no options,” says Rachel Salzman, DVM, Chief Executive Officer, Armatus Bio.
“Organisations like FSHD Global are essential in bridging the gap between early discovery and clinical trials in areas of significant unmet need like FSHD. Beyond catalytic funding, their leadership builds the community awareness and trust needed to accelerate innovative therapies and bring real hope to the FSHD community,” says Salzman.
“One of the greatest privileges of our work is seeing research we’ve supported evolve into therapies with the potential to change lives. FSHD Global was proud to fund pioneering research led by Professor Scott Harper that helped lay the foundations for Armatus Bio’s exciting program. Today, we’re proud to continue that journey by supporting ARM-201 as it advances toward clinical trials. It’s a powerful reminder that sustained investment in research can help turn scientific discovery into hope for families living with FSHD,” says Weatherley.
FSHD Global: https://fshdglobal.org/
Armatus Bio: https://armatusbio.com/