Section 2/3 research of gene remedy for younger kids with MPS IIIA yields combined outcomes

November 21, 2022

2 min learn

Lysogene has introduced topline outcomes from its part 2/3 research evaluating LYS-SAF302, an investigational gene remedy for the therapy of mucopolysaccharidosis sort IIIA, or Sanfilippo syndrome.

In line with an organization launch, six sufferers aged youthful than 30 months who have been enrolled within the open-label, single-arm multicenter AAVance research had statistically vital enchancment in cognitive development, as assessed by Bayley Scales of Toddler and Toddler Growth (third version), in contrast with pure historical past at 24 months post-dosing.

hand, tongs, DNA animation
Topline outcomes from a part 2/3 research of a novel gene remedy for mucopolysaccharidosis sort IIIA demonstrated enchancment in cognitive improvement for sufferers aged youthful than 30 months however not older than 30 months. Supply: Adobe Inventory

This cohort additionally achieved secondary efficacy standards, together with % of individuals with stabilized or improved cognitive, language or motor developmental age at 24 months relative to baseline, and evolution of Vineland Adaptive Conduct Scales (second version) scores in contrast with pure historical past information.

Nevertheless, the corporate acknowledged that the research didn’t meet its major efficacy consequence in the primary cohort of 12 sufferers aged 30 months and older at enrollment in contrast with the pure historical past cohort. As well as, key secondary efficacy endpoints weren’t met.

“Whereas we’re disillusioned with the leads to the primary cohort of the sufferers enrolled at 30 months or older, these can seemingly be defined by the speedy development of the illness and the current studying from different scientific research that gene remedy therapy of neurodegenerative ailments ought to be initiated on the earliest attainable age with a purpose to present a therapeutic profit earlier than the onset of irreversible neuronal injury,” Karen Aiach, founder, chairman and CEO of Lysogene, mentioned within the launch.

In line with the discharge, the security and tolerability of LYS-SAF302 was in line with what has been beforehand communicated, particularly that white matter abnormalities have been noticed on the MRIs close to injection websites in all handled sufferers.

As well as, lesions have stabilized or diminished in measurement in most sufferers and no clinically vital signs have been noticed that may be instantly attributed to white matter abnormalities. Different hostile results noticed have been in line with the pure historical past of illness.

Lysogene additionally acknowledged within the launch that it’s going to full full evaluation and analysis of information from the primary research cohort and work with investigators on future presentation and publication of the outcomes.

“I stay up for persevering with to work with Lysogene to investigate the part 2/3 information and decide probably the most applicable path to bringing gene remedy with LYS-SAF302 to these sufferers who’ve the best potential to profit from it,” Chester B. Whitley, MD, PhD, principal investigator of Lysosomal Illness Community and professor of pediatrics and experimental and scientific pharmacology on the College of Minnesota, mentioned within the launch.


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