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ssAAV9.P546.IGHMBP2
- Development stage
- Unknown
- Lead developer
- Nationwide Children's Hospital
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
ssAAV9.P546.IGHMBP2 (ACTX-401) is an adeno-associated virus serotype 9 (AAV9)-mediated gene replacement therapy designed to deliver a functional copy of the immunoglobulin mu-binding protein 2 (IGHMBP2) gene. Developed by Nationwide Children's Hospital in collaboration with Alcyone Therapeutics, the therapy utilizes a truncated methyl-CpG-binding protein 2 (MeCP2) promoter (P546) to drive moderate, physiological expression of the IGHMBP2 transgene in neurons and glial cells. It is administered via a single intrathecal injection into the cerebrospinal fluid to target the central nervous system. The therapy is currently in Phase I/IIa clinical development for the treatment of IGHMBP2-related diseases, including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot-Marie-Tooth disease type 2S (CMT2S).
- Other names
- AAV9-IGHMBP2AAV-9-IGHMBP2AAV 9-IGHMBP2AAV9.IGHMBP2AAV-9.IGHMBP2AAV 9.IGHMBP2AAV9.P546.IGHMBP2AAV-9.P546.IGHMBP2AAV 9.P546.IGHMBP2
02
Targets
IGHMBP2 (Immunoglobulin mu-binding protein 2)
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