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Lysogene Shifts Sanfilippo Focus To Subgroup Following Pivotal Trial Flop

As Cash Runway Dries Up

 
• By Ayisha Sharma

The French firm’s late-stage trial of its gene therapy candidate for Sanfilippo syndrome missed several endpoints but promising data from a cohort of young infants could help pave a way forward - if it can get more cash.    

Broken piggy bank representing the use of cash savings in times of crisis, running out of money.
Lysogene Needs More Funding Immediately • Source: Shutterstock

Lysogene SA’s LYS-SAF302 for Sanfilippo syndrome has missed the mark in a Phase II/III trial but the firm is pinning its hopes on positive signals in young infants, which it believes could offer a path ahead if it can secure its financial future ahead of a cash crunch early next year.

The open-label, single-arm AAVance trial studied the gene therapy in 20 infants with Sanfilippo syndrome (also known as mucopolysaccharidosis Type IIIA) at seven trial sites across the US and

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