LX1004 Earns FDAs Orphan Drug Designation

Release Date: 16-Sep-2021

US FDA has granted both rare pediatric disease designations and orphan drug designation to LX1004 for CLN2 disease which is also known as late infantile Batten disease. Lexeo Therapeutics has completed a Phase 1 clinical trial of LX1004 and expects to advance, in 2022, to an essential study seeking to prove the therapy’s efficacy.


LX1004 is an adeno-associated virus (AAV)-mediated gene therapy, which is designed to deliver a healthy copy of CLN2, the gene that gives instructions to make an enzyme called tripeptidyl peptidase 1 (TPP1), to the central nervous system, that is comprised of the brain and spinal cord. When CLN2 is mutated, as in late infantile Batten disease, a lack of TTP1 makes cells andmdash; mainly neurons, or nerve cells - die. This results in symptoms such as the loss of motor skills and cognitive ability, and seizures.

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