Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

NCTID	NCT04211714 (View at clinicaltrials.gov)
Description	This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders. (Show More)
Development Status	Active
Indication	Telomere Shortening, Bone Marrow Failure
Disease Ontology Term	DOID:0061213
Compound Name	EXG34217
Sponsor	Elixirgen Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	12 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	ZSCAN4
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	SeV18/TS15ΔF
Editor Type	none
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
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