1. Scrip
  2. >>Business

Gene Therapy Biotechs Treating Sanfilippo Syndrome Hit Cash Crunch

 
• By Ayisha Sharma

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 promising data will support our

More from Business

More from Scrip

Novo Nordisk’s CEO Forced Out After Falling Behind In GLP-1 Battle

 
• By Andrew McConaghie

Company surprises investors with Jørgensen's exit but insists its strategy will not change.

In Brief: US FDA Delay Sends Biohaven Shares Down

 
• By Andrew McConaghie

The US regulator’s decision has taken Biohaven and investors by surprise, with the reason for a ‘major amendment’ status not clear.

Executives On The Move: Pathos Procures CEO From AstraZeneca, New CEO For TME Pharma, And More

Recent moves in the industry include changes at the top at Akamis Bio, Innospera Pharma & Ispen, plus Astellas Korea appoints an ex-Novartis Korea executive.