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

Video

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

“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.
Recent Videos
Manali Kamdar, MD, on Liso-Cel's Ongoing Benefit in the Treatment Lanscape for LBCL
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
© 2024 MJH Life Sciences

All rights reserved.