LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia

Shots:

• The US FDA has granted RPD and ODD to LX2006 for the treatment of FA. The company plans to start a P-I/II clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021
• Additionally, if BLA for LX2006 is approved, a company may be eligible to receive PRV that may be sold or transferred
• The company focuses to advance clinical programs to commercialization & maintain an ongoing research collaboration with Weill Cornell to foster the company’s pre-clinical pipeline. LX2006 (IV) is an AAV-mediated gene therapy encoding the human frataxin gene

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