Professor, genetics, Federal University of Rio Grande do Sul and chief, medical genetics service, Clinic Hospital of Porto Alegre, Rio Grande do Sul, Brazil

Roberto Giugliani, MD, PhD

“Patients with the severe form of MPS have progressive cognitive decline. Along the first few years of life, they start to slow down in the neurological development. They do not progress. And at the end, they start to some regress in this development. This is something that occurs in about 75% of patients with MPS that have this severe form. So, there is a need for the majority of patients with MPS to have a therapy to address the central nervous system deterioration.”

The gene therapy RGX-121 (REGENXBIO) was well-tolerated with promising efficacy in treating mucopolysaccharidosis type 2 (MPS II; Hunter syndrome), according to updated data from the phase 1/2/3 CAMPSIITE trial (NCT03566043).

These data were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2022 Annual Symposium, in Freiburg, Germany, taking place August 30 – September 2, 2022, by Roberto Giugliani, MD, PhD, professor, genetics, Federal University of Rio Grande do Sul and chief, medical genetics service,Clinic Hospital of Porto Alegre, Rio Grande do Sul, Brazil.

 spoke with Giugliani to learn more about the potential advantages of RGX-121 gene therapy in MPSII. He discussed unmet needs in this patient population.

Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from a phase 1/2 study. Presented at: SSIEM 2022 Annual Symposium, Freiburg, Germany, August 30 – September 2, 2022.

Videos

The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII. 

Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2 

“The results are quite interesting. The lower dose and the normal doses were able to significantly decrease the levels of the glycosaminoglycans in the central nervous system... We saw a very significant reduction of this just a few weeks after infusion. With the higher dose, the results are still more significant, we were able to see over 80% reductions in some cases.”

RGX-121, REGENXBIO’s gene therapy intended for the treatment of mucopolysaccharidosis type 2 (MPS II; Hunter syndrome) was well-tolerated with promising efficacy, according to updated data from the phase 1/2/3 CAMPSIITE trial (NCT03566043).

These data were presented by Roberto Giugliani, MD, PhD, professor, genetics, Federal University of Rio Grande do Sul and chief, medical genetics service,Clinic Hospital of Porto Alegre, Rio Grande do Sul, Brazil, at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2022 Annual Symposium, in Freiburg, Germany, taking place August 30 – September 2, 2022.

 discussed the updated data from CAMPSIITE with Giugliani. He highlighted the significant reductions in glycosaminoglycans in the cerebral spinal fluid seen in the trial and the positive safety profile of the intra cisternal magna administration of the therapy.

The professor of genetics at Federal University of Rio Grande do Sul discussed updated data from the CAMPSIITE trial. 

Roberto Giugliani, MD, PhD

Articles

Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2

Roberto Giugliani, MD, PhD, on Reducing GAGs in MPS Type 2 With Gene Therapy