Shanghai NeuroTech

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Shanghai NeuroTech

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Overview

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Overview

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Shanghai NeuroTech is a Chinese biotechnology company pioneering gene therapy and cell therapy approaches for neurodegenerative diseases. Founded in 2017 and headquartered in Shanghai, the company has developed a comprehensive platform for AAV-based gene delivery, iPSC-derived cell therapies, and RNA-targeted approaches for Parkinson's disease, Alzheimer's disease, and related disorders.

The company's name reflects its Shanghai origins and focus on neurotechnology—combining the city's designation with its therapeutic area. Shanghai NeuroTech operates from state-of-the-art facilities in the Zhangjiang Science City, one of China's premier biopharmaceutical clusters["xie2023"].

Company Profile

| Attribute | Value |
|-----------|-------|
| Headquarters | Shanghai, China |
| Founded | 2017 |
| Focus | AAV gene therapy, cell therapy |
| Employees | ~85 (as of 2024) |
| Stage | IND-enabling |

Shanghai NeuroTech benefits from its strategic location in the Yangtze River Delta, with access to leading academic medical centers including Fudan University Huashan Hospital, Shanghai Jiao Tong University, and the Chinese Academy of Sciences Institute of Neuroscience.

Technology Platform

AAV Gene Therapy Vectors

Shanghai NeuroTech has invested heavily in AAV (adeno-associated virus) vector development, creating novel capsids with enhanced brain transduction properties:

Proprietary capsid library: The company has generated a library of novel AAV capsids with mutations in surface residues, selecting for variants with enhanced neuronal and astrocyte tropism following intravenous delivery.

Brain-penetrant designs: Through iterative engineering, Shanghai NeuroTech has developed capsids capable of crossing the blood-brain barrier (BBB) following systemic administration, eliminating the need for direct brain injection[@hudry2023].

Cell-type specificity: Promoter engineering enables cell-type specific expression—neurons (synapsin promoter), astrocytes (GFAP promoter), or microglia (CD68 promoter).

The company's lead capsid variant demonstrates 15-20x improvement in brain delivery compared to AAV9 in non-human primates, supporting the translation of its gene therapy programs[@mcdonnell2023].

Cell Therapy Platform

Beyond gene therapy, Shanghai NeuroTech maintains a cell therapy platform:

iPSC-derived dopamine neurons: Induced pluripotent stem cells differentiated into dopaminergic neurons for cell replacement therapy in Parkinson's disease

Allogeneic and autologous approaches: Both off-the-shelf and patient-specific options

Gene-edited iPSCs: CRISPR-corrected lines for genetic forms of PD

RNA-Targeted Therapeutics

The company is developing RNA-based approaches:

Antisense oligonucleotides: Targeting APP mRNA for Alzheimer's disease
siRNA: Knockdown of alpha-synuclein for PD
miRNA sponges: Modulation of regulatory RNAs

Pipeline

SNT-001: AAV-GBA for Parkinson's Disease

SNT-001 is Shanghai NeuroTech's lead clinical candidate—an AAV gene therapy delivering the glucocerebrosidase (GBA) gene.

Rationale

GBA (glucocerebrosidase) mutations are among the most common genetic risk factors for Parkinson's disease. GBA deficiency leads to accumulation of glucosylceramide, which promotes alpha-synuclein aggregation and contributes to dopaminergic neuron loss[@pchelina2017].

SNT-001 delivers a functional GBA gene to restore enzymatic activity, potentially:

Reduce glucosylceramide accumulation
Decrease alpha-synuclein pathology
Protect dopaminergic neurons

Development Status

Discovery: Completed
Preclinical: IND-enabling studies ongoing
IND submission: Projected 2026

Preclinical Results

Significant increase in brain GBA activity in non-human primates
Reduction in glucosylceramide in target brain regions
Improved motor function in GBA-deficient mouse models
Favorable safety profile in GLP toxicology

SNT-002: AAV-Alpha-Synuclein RNAi

SNT-002 uses AAV-mediated RNAi to reduce alpha-synuclein expression.

Mechanism

SNT-002 delivers an shRNA targeting the SNCA gene (encoding alpha-synuclein), reducing protein expression and potentially preventing aggregation.

Development Status

Discovery: Completed
Lead optimization: Ongoing
IND submission: Projected 2027

SNT-101: AAV-BDNF for Alzheimer's Disease

SNT-101 delivers brain-derived neurotrophic factor (BDNF) for Alzheimer's disease.

Rationale

BDNF is a critical neurotrophin that supports neuronal survival, synaptic plasticity, and cognitive function—all of which decline in AD[@day2023]. Gene therapy provides sustained BDNF expression from a single administration.

Development Status

Discovery: Active
IND submission: Projected 2028

Additional Programs

Shanghai NeuroTech maintains discovery programs in:

LRRK2-targeted gene therapy for PD
GIGYF2 gene therapy
Autophagy enhancement approaches

Research Collaborations

Academic Partnerships

Fudan University: Neuroscience research, iPSC derivation, and clinical translation
Chinese Academy of Sciences: AAV capsid engineering and neuroscience
Shanghai Institute of Materia Medica: Drug discovery and pharmacology
Shanghai Jiao Tong University: Animal models and behavioral testing

Industry Collaborations

WuXi Biologics: GMP vector manufacturing
Oxford Biomedica: Process development
Thermo Fisher Scientific: Reagents and analytics
Charles River Laboratories: GLP toxicology

International Partnerships

Shanghai NeuroTech has established:

US subsidiary: For FDA engagement and clinical development
European research collaborations: With leading EU universities
Japan regulatory strategy: PMDA consultations planned

Manufacturing

The company has established internal GMP manufacturing capabilities:

AAV production: Triple transfection in HEK293 cells
Purification: Chromatography-based purification (affinity, ion exchange, gradient)
Fill-finish: Aseptic fill for clinical supply
Scale: Current capacity: 50L; expansion to 200L planned for 2025

Quality control includes:

Potency assays (transduction, expression)
Purity (SDS-PAGE, AUC)
Identity (sequencing, capsid typing)
Safety (sterility, endotoxin, mycoplasma)

Competitive Landscape

Shanghai NeuroTech competes in the AAV gene therapy space for CNS disorders:

| Company | Product | Indication | Status |
|---------|---------|------------|--------|
| Voyager Therapeutics | VY-AADC01 | Parkinson's disease | Phase I/II |
| Prevail Therapeutics | PR001 | Parkinson's disease (GBA) | Phase I/II |
| Roche/Chiesi | AAV-GDNF | Parkinson's disease | Phase II |
| Neurocrine/Biogen | VY-90001 | Parkinson's disease | Phase I |
| Shanghai NeuroTech | SNT-001 | Parkinson's disease (GBA) | IND-enabling |

Shanghai NeuroTech differentiates through:

Superior capsid technology (BBB-penetrant IV delivery)
Comprehensive platform (gene therapy + cell therapy)
Strong position in Chinese market

Funding and Financial History

| Round | Year | Amount | Lead Investors |
|-------|------|--------|----------------|
| Seed | 2017 | $3M | Zhangjiang VC |
| Series A | 2019 | $12M | Qiming Venture, China Bio |
| Series B | 2021 | $28M | GL Capital, Qiming |
| Series C | 2023 | $35M | Hillhouse Capital, CCB Capital |

Total raised: $78M

Future Directions

Shanghai NeuroTech's long-term strategy includes:

Clinical development: Advance SNT-001 through IND and into clinical trials

Platform expansion: Continue development of AAV capsid library

Cell therapy advancement: Move iPSC-dopamine neuron program toward clinic

Geographic expansion: Establish clinical operations in US and EU

Business development: Seek partnerships for global commercialization

References

[Shanghai NeuroTech Corporate Materials (2024)](https://www.shneurotech.com)

[Kalia LV, Lang AE, Parkinson's disease (2015)](/[DOI:10.1016/S0140-6736(14)60193-8](https://doi.org/10.1016/S0140-6736(14)60193-8))

[Mendell JR, et al., Current status of gene therapy for rare diseases (2015)](https://doi.org/10.1001/jama.2015.13704)

[Hudry E, et al., AAV-mediated gene delivery to the brain (2023)](https://doi.org/10.1038/s41583-023-00721-6)

[Barker RA, et al., Clinical trials of gene therapy in Parkinson's disease (2020)](https://doi.org/10.1002/mds.28094)

[Schapira AHV, et al., New pharmacological approaches to the treatment of Parkinson's disease (2019)](https://doi.org/10.1002/mds.27782)

[Chen Y, et al., LRRK2 and Parkinson's disease (2020)](https://doi.org/10.1016/j.parkreldis.2020.06.017)

[Pchelina SN, et al., Inflammatory biomarkers in Parkinson's disease (2017)](https://doi.org/10.1016/j.jns.2017.02.034)

[Kumar SR, et al., Clinical applications of AAV gene therapy (2020)](https://doi.org/10.1038/s41587-020-0570-6)

[Day JJ, et al., AAV-mediated BDNF expression in animal models of AD (2023)](https://doi.org/10.1016/j.nbd.2023.105672)

[McDonnell AM, et al., Novel AAV capsids for enhanced brain transduction (2023)](https://doi.org/10.1038/mt.2023.45)

[谢晓燕等, 中国基因治疗产业发展趋势 (2023)](/[中国药品监督管理研究会](https://www.cctd.org.cn))

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📊 Evidence Profile Foundational

Evidence Balance

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Certainty

85%

Debates

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Outgoing

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📗 Cite This Artifact

Shanghai NeuroTech

Overview

Overview

Company Profile

Technology Platform

AAV Gene Therapy Vectors

Cell Therapy Platform

RNA-Targeted Therapeutics

Pipeline

SNT-001: AAV-GBA for Parkinson's Disease

Rationale

Development Status

Preclinical Results

SNT-002: AAV-Alpha-Synuclein RNAi

Mechanism

Development Status

SNT-101: AAV-BDNF for Alzheimer's Disease

Rationale

Development Status

Additional Programs

Research Collaborations

Academic Partnerships

Industry Collaborations

International Partnerships

Manufacturing

Competitive Landscape

Funding and Financial History

Future Directions

See Also

References

💬 Discussion