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["@sio"].
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["@company2024"].
SIO-101 is an AAV-based gene therapy designed to deliver a functional copy of the [GBA1](/genes/gba1) gene to treat [Parkinson's disease](/diseases/parkinsons-disease) in patients with [GBA1](/genes/gba1) mutations or sporadic disease[@gba2021].
Mechanism of Action:
Delivers functional [GBA1](/genes/gba1) gene via AAV vector
Increases glucocerebrosidase (GCase) activity in the [brain](/brain-regions/cortex)
Reduces [alpha-synuclein](/proteins/alpha-synuclein) aggregation in [dopaminergic neurons](/cell-types/substantia-nigra-dopamine-neurons)
Addresses [lysosomal dysfunction](/mechanisms/lysosomal-dysfunction) — a central feature of [Parkinson's disease](/diseases/parkinsons-disease) pathogenesis
Approach: AAV-delivered therapeutic (gene replacement or silencing)
Status: Discovery stage
[LRRK2](/genes/lrrk2) mutations cause increased kinase activity leading to mitochondrial dysfunction, defective autophagy, and altered synaptic function in [dopaminergic neurons](/cell-types/substantia-nigra-dopamine-neurons).
Alzheimer's Disease Program
SIO-201 is an undisclosed [Alzheimer's disease](/diseases/alzheimers-disease) program:
Target: Novel therapeutic target
Approach: AAV gene therapy
Status: Discovery
Technology Platform
AAV Gene Therapy
Sio uses adeno-associated virus (AAV) vectors for gene delivery to the central nervous system:
Sio Gene Therapies has undergone significant transformation:
2014: Founded as Axovant Sciences
2015: IPO on NASDAQ
2017: Intepirdine failed in Phase 3 — halted
2020: Renamed to Sio Gene Therapies
2021: Pivot to gene therapy pipeline
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.
Partnerships
| Partner | Focus Area | Status | |---------|------------|--------| | Oxford Biomedica | AAV vector manufacturing | Active | | Academic collaborators | Research programs | Multiple | | Patient foundations | Clinical development | Active |
Competitive Landscape
Sio competes with other gene therapy companies targeting neurodegenerative diseases: