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

“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.

CGTLive 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.

REFERENCE

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.