Some families may have heard recently that two of REGENXBIO’s gene therapy programs have been placed ‘on hold’ and may be feeling worried. This short update is meant to reassure you, that the current press release in the news regarding REGENXBIO’s gene therapy program does not involve in any way their Duchenne Muscular Dystrophy (DMD) gene therapy program.
According to a new press release, the Food & Drug Agency (FDA) in the USA has put a hold on two of REGENXBIO’s gene therapy programs (RGX-111 and RGX-121) due to the death of a child enrolled in their clinical trial. The child who passed away had a very rare lysosomal storage disorder, called mucopolysaccharidosis, or MPS. The child did not have Duchenne muscular dystrophy. An ongoing investigation is in process to determine the reasons for this adverse outcome. So far, there is no indication that the gene therapy directly caused the adverse event (fatality).
We want to clearly reassure the Duchenne community that the recent news from the company update does not affect the Duchenne gene therapy program (RGX-202). RGX-202 for Duchenne is a completely different therapy, using a different viral vector, a different gene construct, and a different delivery approach.
In the ongoing Duchenne clinical trial (RGX-202), no serious safety concerns have been reported, and the study continues as planned, with enrollment in the confirmatory trial moving forward. Patient safety remains their top priority.
Duchenne families enrolled in trials remain closely monitored and supported, and we will continue to share updates if anything changes. Currently, there is no change to the Duchenne program, and families do not need to take any action.
For more information on the update from REGENXBIO, see the press release below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: