Benzinga | Jun 25, 2021 07:32AM EDT

Dyne Therapetuics Highlights Presentation Of Preclinical Data From Facioscapulohumeral Musclar Dystrophy Program During FSHD Society Int'l. Research Congress

- FORCE(tm) platform enables targeted muscle delivery with lead FSHD program candidate demonstrating potent suppression of DUX4 biomarkers in patient cell line -

WALTHAM, Mass., June 25, 2021 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is presenting today preclinical data from its facioscapulohumeral muscular dystrophy (FSHD) program during the 28th Annual FSHD Society International Research Congress. Data from in vitro studies in an FSHD patient cell line being presented highlight that Dyne's proprietary FORCE(tm) platform enabled targeted muscle delivery with its lead FSHD candidate demonstrating potent suppression of DUX4 transcriptome markers.

FSHD is caused by aberrant activation of the double homeobox 4 (DUX4) transcription factor in muscle cells, leading to skeletal muscle loss, progressive muscle weakness and wasting. Dyne's approach is designed to address the genetic basis of the disease by reducing DUX4 mRNA expression. The company's lead FSHD candidate consists of a transferrin 1 receptor-binding fragment antibody (Fab) conjugated to a phosphorodiamidate morpholino oligomer (PMO) targeting DUX4 mRNA.

"It is critically important that we continue to advance the science and new therapeutic candidates for FSHD, a highly debilitating, progressive genetic disease with no approved treatments," said Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. "We are excited to share these data, which build upon our prior in vitro studies and support our approach to targeting the genetic basis of FSHD. These results demonstrate that our lead FSHD candidate achieved potent suppression of key DUX4 biomarkers, reinforcing the potential of the FORCE platform to enable targeted delivery to muscle tissue. We look forward to advancing our FSHD program toward the clinic as one of the three IND submissions we have planned between the fourth quarter of 2021 and the fourth quarter of 2022."

The in vitro proof of concept studies were conducted using an immortalized FSHD patient cell line. Three well-known DUX4 transcriptome markers MBD3L2, TRIM43, and ZSCAN4 were tracked and a significant increase in expression was observed in these markers once cells were differentiated to myotubes. Dyne's lead FSHD candidate was subsequently evaluated in vitro and showed potent suppression of these DUX4 transcriptome markers with IC50 in the low nanomolar range and also demonstrated superior reduction of these same markers compared to naked (unconjugated) PMO.

Today's presentation entitled, "FORCE(tm) platform enables muscle-targeted delivery of antisense oligonucleotide and silencing of DUX4 activity in an FSHD cell line" will be available in the Scientific Publications & Presentations section of Dyne's website following the meeting at https://www.dyne-tx.com/our-forcetm-publications/.