Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

NCTID	NCT03634007 (View at clinicaltrials.gov)
Description	This clinical trial is an open label, dose-ranging study designed to evaluate gene therapy to treat patients who are APOE4 homozygotes with clinical diagnosis varying from mild cognitive impairment due to Alzheimer's, mild dementia due to Alzheimer's disease, and moderate dementia due to Alzheimer's disease. (Show More)
Development Status	Active
Indication	Alzheimer Disease, Early Onset Alzheimer Disease
Disease Ontology Term	DOID:10652
Compound Name	LX1001
Compound Description	AAVrh.10hAPOE2-HA
Sponsor	Lexeo Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	15
Results Posted	Not Available

Target Gene/Variant	APOE2
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type	none
Dose 1	1.4E10 gc/mL CSF
Dose 2	4.4E10 gc/mL CSF
Dose 3	1.4E11 gc/mL CSF
Dose 4	1.4E14 gc (fixed dose)
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track
Recent Updates	Lexeo is pursuing partnership opportunities for continued development