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Drug intelligence / Profile preview
ssAAV9.IGHMBP2
- Development stage
- Unknown
- Lead developer
- Nationwide Children's Hospital
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
ssAAV9.IGHMBP2 (also known as ACTX-401) is an investigational single-stranded adeno-associated virus serotype 9 (AAV9) gene replacement therapy designed to treat immunoglobulin mu-binding protein 2 (IGHMBP2)-related disorders, including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot-Marie-Tooth disease type 2S (CMT2S). Developed by Nationwide Children's Hospital and licensed to Alcyone Therapeutics, the therapy delivers a functional copy of the human IGHMBP2 gene to restore expression of the IGHMBP2 protein, an ATP-dependent helicase essential for motor neuron survival and translation homeostasis. It is administered as a single intrathecal injection into the cerebrospinal fluid and is currently being evaluated in a Phase I/II clinical trial.
- Other names
- AAV9-IGHMBP2AAV-9-IGHMBP2AAV 9-IGHMBP2AAV9.IGHMBP2AAV-9.IGHMBP2AAV 9.IGHMBP2ssAAV9-IGHMBP2ssAAV-9-IGHMBP2ssAAV 9-IGHMBP2
02
Targets
IGHMBP2 (Immunoglobulin mu-binding protein 2)
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