Early Phase Trial of REGENXBIO's Gene Therapy for Duchenne Muscular Dystrophy Begins

 January 24, 2023

                                       A Phase I/II trial for the gene therapy RGX-202 by REGENXBIO is starting for Duchenne muscular dystrophy. The new microdystrophin protein that is produced by RGX-202 has the functional components of the C-Terminal (CT) domain present in naturally occurring dystrophin.

The NAV AAV8 vector, which has been utilised in a number of clinical studies, and a muscle-specific promoter are employed in the design of RGX-202 for the targeted delivery and expression of genes across skeletal and cardiac muscle (Spc5-12).

The RGX-202 programme is a crucial component of our "5x'25" plan, which calls for having five AAV Therapeutics either on the market or in late-stage development by 2025, according to Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. As we advance a very distinctive product candidate with the potential to enhance muscular strength and motor function in boys with Duchenne, we look forward to continuing to engage closely with the Duchenne community.

A uncommon hereditary condition known as Duchenne muscular dystrophy (DMD) is brought on by mutations in the gene that makes the protein dystrophin, which is essential for the structure and operation of muscle cells. A worldwide prevalence of one in 3,500 to one in 5,000 men is impacted by the illness, which mostly affects males.

There are now over 60 studies for gene therapy for DMD, which is a strongly fought field. Biological obstacles still seem to exist despite positive laboratory experiments. One of the most important factors in this method is the selection of the vector.

A one-time intravenous (IV) injection of RGX-202 is being tested in patients with Duchenne in the multicenter, open-label AFFINITY DUCHENNE study by REGENXBIO.

Six ambulatory juvenile Duchenne patients (aged four to eleven) are anticipated to participate in two cohorts receiving doses of 1 1014 genome copies (GC)/kg body weight (n=3) and 2 1014 GC/kg body weight (n=3) during the trial's dosage assessment phase.

A comprehensive, short-term prophylactic immunosuppression regimen to proactively mitigate potential complement-mediated immunologic responses is included in the trial design, along with inclusion criteria based on the status of dystrophin gene mutations, including DMD gene mutations in exons 18 and above. Safety measures informed by the Duchenne community and engagement with key opinion leaders are also included.

The business is also looking for participants for their observational screening research, the AFFINITY BEYOND trial, whose main goal is to gauge the prevalence of AAV8 antibodies in DMD kids under the age of 12. Potential participants for the AFFINITY DUCHENNE trial and upcoming RGX-202 studies may be found using data from this study.

Aravindhan Veerapandiyan, MD, a principal investigator in the study and Director of the Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care Center, and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children's Hospital, said, "Duchenne muscular dystrophy is a devastating disease and there are still unmet therapeutic needs." The progressive nature of Duchenne may be impacted by gene treatments like RGX-202.

Adeno-associated virus (AAV) gene delivery platform owned exclusively by REGENXBIO, the NAV Technology Platform includes exclusive rights to more than 100 unique AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. The NAV Technology Platform is being used by REGENXBIO and its third-party NAV Technology Platform Licensees to create a large pipeline of prospects, including late-stage and commercial projects in several therapeutic areas.

Under the terms of a Creative Commons licence, this article has been taken from INSIDE PRECISION MEDICINE. Go here to read the original article.