Michael Hocquemiller, PhD, and Samantha Parker of Lysogene talk about the latest developments in the company’s gene therapy, LYS-SAF302, to treat Sanfilippo syndrome type A.
Sanfilippo syndrome type A (MPS IIIA) is a progressive, life-threatening, and rare inherited metabolic disorder affecting children from a young age. This condition is caused by mutations in the SGSH gene that results in the accumulation of heparan sulfate in lysosomes. MPS IIIA mainly affects the central nervous system where it causes severe and progressive degeneration.
LYS-SAF302 is a gene therapy that delivers a functional copy of the human SGSH gene directly to brain cells using the adeno-associated virus carrier, AAVrh.10. The results of the ongoing phase 1/2 study were presented at this year’s virtual WORLDSymposium.
The study is currently on clinical hold to assess abnormal variations in white matter that have been observed in MRI findings. However, Ms. Parker notes the program is complete as 19 patients in the study are being dosed with gene therapy and that data continues to be collected.
To learn more about this and other gene therapy trials, click here.