Gene Therapy Biotechs Treating Sanfilippo Syndrome Hit Cash Crunch

 
• By Ayisha Sharma
  
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Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.

Gene Therapies Are Risky Business For Both Developers And Investors • Source: Shutterstock

Two biotechs hoping to develop gene therapies for the rare disorder Sanfilippo syndrome have hit funding roadblocks that have pushed them down two different paths. Lysogene SA is banking on new clinical data to loosen the purse strings of investors, while Abeona Therapeutics has offloaded its candidate to Ultragenyx Pharmaceutical Inc. to free up resources for other disease therapies.

Touting preliminary data from a late-stage Sanfilippo syndrome study of its lead candidate LYS-SAF302, Lysogene EO Karen Aiach said, “These...

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