Original Post: October 1, 2024
Updated: October 30, 2024
Years of basic pre-clinical research seed funded by Cure CMD accumulated substantial evidence that LAMA-111, a "sister" protein to the LAMA-211 protein, may have therapeutic benefits for LAMA2-RD affected individuals.
Modalis Therapeutics, a biotech company developing precision genetic medicines, approached Cure CMD in 2018, interested in applying their nascent technology to upregulate the LAMA1 gene to compensate for the LAMA2 loss of function. The company requested our support to bring the voices of the LAMA2 community and our relationships with key opinion leaders in the LAMA2-RD research and clinical field.
That collaboration reached a milestone with the good news that the FDA granted Modalis Therapeutics' MDL-101 program for treating LAMA2-CMD, a Rare Pediatric Disease Designation. This determination was followed a few weeks later by the FDA's Orphan Drug Designation.
We celebrate these achievements while continuing to support this potential therapy’s road to the clinical trial stage. On a cautionary note, there is no clear timeline for completing all of the pre-clinical toxicology studies, clinical trial protocol, trial site selection, and numerous regulatory approvals to move this program forward. However, the LAMA2 community will be informed when relevant steps are made and your engagement is necessary.
Visit the FDA website to learn more about the process of designating an orphan product. After receiving FDA’s Rare Pediatric Disease and Orphan Drug Designations, Modalis CEO, Haru Morita commented, "These designations from the FDA are proof that our efforts are rational and achieving results, and we hope to further accelerate the development of this world's first product using the cutting-edge technology."
Learn more about Modalis' preliminary planning of the MDL-101 clinical trial and the FY2024 2Q Financial Results in a public presentation made in August 2024.
Are you registered in the Congenital Muscle Disease International Registry? This is the best way to ensure you are notified of opportunities to participate in future clinical studies or trials, and to contribute your data to research by completing surveys.
If you've already registered, please log into your profile to ensure everything is accurate and up to date, that you have completed all surveys, and submitted your genetic report for curation. Please do not create a new profile! Get in touch if you are unable to use the platform's password reset for assistance.