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Regenxbio Presents Positive Interim Data From Phase I/II Trial Of RGX-121 For Treatment Of MPS II

Regenxbio Inc. (RGNX) said that RGX-121, a one-time gene therapy for Mucopolysaccharidosis Type II, continues to be well-tolerated with no drug-related serious adverse events, as per phase I/II trial results.

The company said it will present the additional positive interim data from the ongoing phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, at the American Society of Gene and Cell Therapy 24th Annual Meeting.

The company noted that the biomarker data from those patients continues to demonstrate that the I2S enzyme is active in the CNS and importantly, continued cognitive development has been observed in the majority of patients who have been followed for more than 6 months.

In addition, emerging evidence of systemic enzyme expression and activity has been shown in urine and plasma, including in patients who are naive to enzyme replacement therapy, the current standard of care.

"It is encouraging to see continued efficacy signals up to two years after administration of RGX-121 in our Phase I/II trial..." said Steve Pakola, chief Medical Officer of Regenxbio.

The company stated that it has begun dosing patients in Cohort 3, and it looks forward to providing further program updates later this year.

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