Re: Little Lulu's Progress - recent write up on Lysogene
"Lysogene – a low risk high reward opportunity" April 10, 2019 By EVEN WANG
"Lysogene’s strategy to treating MPSIIIA is to replace the SGSH gene by using AAVrh.10 licensed from Regenxbio. This vector is directly injected into both brain hemispheres by neurosurgery."
"Lysogene’s main competition is Abeona an American company. They are partnering with Dr. Kevin Flanigan at Nationwide Children’s hospital. They use the AAV9 to deliver the SGSH gene. Their phase 1 study showed significant reduction in heparan sulfate but did not show striking clinical response."