LEXEO Therapeutics receives rare paediatric disease designation and orphan drug designation for LX2006 for the treatment of Friedreich’s ataxia

Lexeo Therapeutics

30 June 2021 - Phase I/II clinical trial in patients with cardiomyopathy associated with Friedreich’s ataxia expected to initiate in 2021.

LEXEO Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation and orphan drug designation to LX2006 for the treatment of Friedreich’s ataxia. 

LX2006 is an IV administered, adeno-associated virusmediated gene therapy encoding the human frataxin gene.

Read LEXIO Therapeutics press release

Michael Wonder

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Michael Wonder