Joint statement on Modalis' MDL-101 program for LAMA2

Cure CMD, Voor Sara, and Modalis Therapeutics have released a joint statement on MDL-101 — an investigational, one-time gene therapy for LAMA2 muscular dystrophy. Instead of replacing the faulty LAMA2 gene, the therapy uses CRISPR-GNDM technology to switch on the related LAMA1 gene and restore the missing laminin, without changing the DNA sequence.

A Phase 1/2 trial is in preparation, and a submission to the U.S. FDA is still pending — no enrollment date has been set. The first study will be small (around ten or fewer children) at a single U.S. site that can also enroll international patients. Eligibility criteria are still being defined.

For Bulgarian families: trial sites reach participants through the patient registry. If your child has LAMA2, registering with CMDIR is the surest way to hear directly about eligibility and enrollment.