Sio Gene Therapies Inc. (formerly Axovant Sciences) is a clinical-stage biotechnology company headquartered in Cambridge, Massachusetts, focused on developing gene therapies for neurodegenerative diseases. The company leverages AAV vector technology to deliver therapeutic genes to the central nervous system[1].
Sio's pipeline includes programs targeting Parkinson's disease, Alzheimer's disease, and other neurological disorders. The company has a strategic partnership with Oxford Biomedica for vector manufacturing and has in-house clinical development capabilities[2].
Sio's gene therapy pipeline targets neurodegenerative diseases using AAV-delivered therapeutic genes:
| Program | Target/Mechanism | Indication | Phase | Status |
|---|---|---|---|---|
| SIO-101 | AAV-GBA1 gene therapy | Parkinson's Disease | Preclinical | Research |
| SIO-102 | AAV-LRRK2 targeting | Parkinson's Disease | Discovery | Research |
| SIO-201 | Undisclosed | Alzheimer's Disease | Discovery | Research |
| SIO-301 | Undisclosed | CNS disorder | Discovery | Research |
SIO-101 is an AAV-based gene therapy designed to deliver a functional copy of the GBA1 gene to treat Parkinson's disease in patients with GBA1 mutations or sporadic disease[3].
Mechanism of Action:
Rationale:
Development Status:
SIO-102 is a discovery-stage program targeting LRRK2 kinase in Parkinson's disease:
LRRK2 mutations cause increased kinase activity leading to mitochondrial dysfunction, defective autophagy, and altered synaptic function in dopaminergic neurons.
SIO-201 is an undisclosed Alzheimer's disease program:
Sio uses adeno-associated virus (AAV) vectors for gene delivery to the central nervous system:
| Component | Description |
|---|---|
| Serotype | CNS-optimized capsid (AAV9, AAVrh10) |
| Promoter | Neuron-specific expression (Synapsin, CamKIIa) |
| Delivery | Intraparenchymal injection or intrathecal |
| Manufacturing | Oxford Biomedica partnership |
Sio Gene Therapies has undergone significant transformation:
The transition from small molecule approaches to AAV-based gene therapy reflects the company's strategy to address the underlying genetic and molecular causes of neurodegenerative diseases.
| Partner | Focus Area | Status |
|---|---|---|
| Oxford Biomedica | AAV vector manufacturing | Active |
| Academic collaborators | Research programs | Multiple |
| Patient foundations | Clinical development | Active |
Sio competes with other gene therapy companies targeting neurodegenerative diseases: