Dyne Therapeutics Presents New Preclinical Data from its Myotonic Dystrophy Type 1 Program During American Society of Gene & Cell Therapy Annual Meeting Demonstrating Sustained Knockdown of Toxic Human Nuclear DMPK RNA - Seite 2

Dyne expanded its analysis in the hTfR1/DMSXL model to evaluate the administration of a single, low 10 mg/kg dose of its lead DM1 candidate after 4 weeks. These new data show sustained DMPK knockdown at 4 weeks: 51 percent in the diaphragm, 46 percent in both the heart and tibialis anterior, and 42 percent in the gastrocnemius. Dyne’s candidate was well tolerated in the hTfR1/DMSXL studies.

Additionally, Dyne is reporting during ASGCT new in vitro findings from DM1 patient cells with approximately 380 and 2,600 CTG repeats, where its candidate showed a robust, dose-dependent reduction in DMPK RNA, nuclear foci and correction of splicing defects as measured by BIN1 exon 11 inclusion. The results in the cell line with approximately 2,600 CTG repeats are particularly notable given the severity of DM1 disease represented.

“At Dyne we are focused on delivering disease-modification for patients, and the DMPK knockdown we are observing in our hTfR1/DMSXL model is consistent with the range that genetic studies suggest can be clinically meaningful,” said Joshua Brumm, president and chief executive officer of Dyne. “These latest findings further strengthen the dataset we’ve already assembled, showing reduction in nuclear foci and splicing correction in patient cells, as well as splicing correction and reversal of myotonia in the well-validated HSA^LR in vivo model. We believe we are well positioned as we continue to advance our DM1 program toward the clinic.”

Data from Dyne’s DM1 program are being featured during the following presentations at ASGCT today and will be made available in the Scientific Publications & Presentations section of Dyne’s website following the meeting:

Presentation: Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
Scientific Symposium: Hot Topics and Remaining Challenges in RNAi and Oligonucleotide Therapy for 2021
Time: 10:26 a.m. ET

Oral Presentation: The FORCE Platform Achieves Robust Knock Down of Toxic Human Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in Newly Developed hTfR1/DMSXL Mouse Model (Abstract #247)
Session: Oligonucleotide Therapeutics
Time: 1:15 p.m. ET

DM1 Program Webcast

Dyne will host a live webcast event today at 4:00 p.m. ET to review the company’s DM1 program and preclinical data, and the importance of targeting the genetic basis of the disease. Joining management on the webcast will be Charles Thornton, M.D., the Saunders Distinguished Professor of Neuromuscular Research at the University of Rochester. Dr. Thornton has been engaged in bench and clinical research on myotonic dystrophy for 30 years.