Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne muscular dystrophy. Insmed intends to start Phase I clinical trials for INS1201 in the first half of 2025 after the FDA approved its investigational new drug (IND) application in December 2024.
What is Insmed INS1201 Gene Therapy?
INS1201 targets the central nervous system directly and is administered intrathecally, in contrast to Elevidys, which needs systemic intravenous injection. The safety and effectiveness issues with systemic gene treatments are intended to be addressed by this focused delivery strategy.
Intrathecal administration dramatically lowers the necessary dose by ten to fifty times, preventing systemic toxicity and off-target effects by concentrating the medication where it is needed. Despite obstacles like spinal cord injury, nerve damage, and the risk for infections, this might provide patients with a safer and possibly more successful option, especially in light of safety issues associated with the large viral vector dosages utilized in intravenous therapy.
Learn More: Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
