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ELJ-101 is an adeno-associated virus (AAV) gene therapy candidate being developed by Elaaj Bio for the treatment of CDKL5 Deficiency Disorder (CDD). CDD is a rare, X-linked genetic disorder caused by mutations in the *CDKL5* gene, which leads to the absence or dysfunction of the CDKL5 protein, a kinase essential for normal brain development and function. ELJ-101 is designed to deliver a functional copy of the *CDKL5* gene to cells within the central nervous system, thereby restoring protein expression and potentially addressing the underlying cause of the severe seizures and developmental impairments associated with the condition. The program is currently in the IND-enabling stage of development.
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