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Globe Biotech SMN1 gene therapy
- Development stage
- Preclinical
- Lead developer
- GEMMA Biotherapeutics
- Modality
- Gene Therapies
- Administration
- Intracisterna Magna
01
Overview
Globe Biotech SMN1 gene therapy is an investigational gene therapy candidate being developed for the treatment of spinal muscular atrophy (SMA). The therapy is designed to address the underlying genetic cause of SMA by delivering a functional copy of the survival motor neuron 1 (SMN1) gene or otherwise restoring its function. SMA is a rare, progressive neuromuscular disorder characterized by the loss of motor neurons in the spinal cord and brainstem, leading to muscle weakness and atrophy due to a deficiency in the survival motor neuron (SMN) protein. This program is part of Globe Biotech's emerging pipeline in advanced therapeutics, though specific details regarding the delivery vector (e.g., AAV) and clinical phase are currently limited.
- Other names
- SMN1 gene therapySMN-1 gene therapySMN 1 gene therapy
02
Targets
Adeno-associated virus serotype 9 capsid–receptor interface (AAV9-receptor interface)SMN (Survival motor neuron protein)
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