Sio Gene Therapies

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SiO Gene Therapies

Overview

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SiO Gene Therapies

Overview

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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"].

Funding

IPO: 2020 (NASDAQ: SIOX)
Funding: Series A-C

Pipeline Overview

Sio's gene therapy pipeline targets neurodegenerative diseases using AAV-delivered therapeutic genes:

| Program | Target/Mechanism | Indication | Phase | Status |
|---------|-----------------|------------|-------|--------|
| SIO-101 | AAV-[GBA1](/genes/gba1) gene therapy | [Parkinson's Disease](/diseases/parkinsons-disease) | Preclinical | Research |
| SIO-102 | AAV-[LRRK2](/genes/lrrk2) targeting | [Parkinson's Disease](/diseases/parkinsons-disease) | Discovery | Research |
| SIO-201 | Undisclosed | [Alzheimer's Disease](/diseases/alzheimers-disease) | Discovery | Research |
| SIO-301 | Undisclosed | CNS disorder | Discovery | Research |

Parkinson's Disease Programs

SIO-101 (GBA1 Gene Therapy)

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

Rationale:

[GBA1](/genes/gba1) mutations increase PD risk 5-20x
GCase activity is reduced in sporadic PD [Parkinson's disease](/diseases/parkinsons-disease)
[Lysosomal dysfunction](/mechanisms/lysosomal-dysfunction) is central to alpha-synuclein accumulation
Gene therapy provides long-term benefit over small molecule approaches

Development Status:

Preclinical development stage
IND-enabling studies underway
Vector manufacturing partnership with Oxford Biomedica

SIO-102 (LRRK2 Targeting)

SIO-102 is a discovery-stage program targeting [LRRK2](/genes/lrrk2) kinase in [Parkinson's disease](/diseases/parkinsons-disease):

Target: [LRRK2](/genes/lrrk2) mutations (G2019S, R1441C/H/G)
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:

| Component | Description |
|-----------|-------------|
| Serotype | CNS-optimized capsid (AAV9, AAVrh10) |
| Promoter | Neuron-specific expression (Synapsin, CamKIIa) |
| Delivery | Intraparenchymal injection or intrathecal |
| Manufacturing | Oxford Biomedica partnership |

Clinical Capabilities

In-house clinical development team
Regulatory expertise in gene therapy
Global trial network for neurodegeneration
Patient access and advocacy programs

History

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:

[Voyager Therapeutics](/companies/voyager-therapeutics) — AAV-based PD programs
[Prevail Therapeutics](/companies/prevail-therapeutics) (Eli Lilly) — GBA1 gene therapy
[Neuromyx](/companies/neuromyx) — AAV capsid engineering
[Spark Therapeutics](/companies/spark-therapeutics) (Roche) — gene therapy platform
[Meiragtx](/companies/meiragtx) — CNS gene therapy

References

[Sio Gene Therapies Corporate Website](https://www.siogt.com)[@sio].

[Company Pipeline — Investor Presentation 2024](https://investor.siogt.com)[@company2024].

[GBA1 Gene Therapy for Parkinson's Disease](https://pubmed.ncbi.nlm.nih.gov/32895569/). Mol Ther, 2021[@gba2021].

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Sio Gene Therapies

SiO Gene Therapies

Overview

SiO Gene Therapies

Overview

Funding

Pipeline Overview

Parkinson's Disease Programs

SIO-101 (GBA1 Gene Therapy)

SIO-102 (LRRK2 Targeting)

Alzheimer's Disease Program

Technology Platform

AAV Gene Therapy

Clinical Capabilities

History

Partnerships

Competitive Landscape

See Also

References