AAIC 2026: Gene Therapy Developments

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event519 wordssynced 2026-04-02

Conference: AAIC 2026 | Dates: July 12-15, 2026 | Location: Excel London, UK

Overview

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Gene therapy represents a transformative approach to treating Alzheimer's disease, with the potential to deliver disease-modifying payloads directly to the brain. AAIC 2026 features the latest advances in viral vector delivery, gene editing, and RNA-targeted approaches for neurodegenerative diseases["@sengupta2024"].

Key Topics

Viral Vector Delivery Systems

AAV Vectors

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Conference: AAIC 2026 | Dates: July 12-15, 2026 | Location: Excel London, UK

Overview

Mermaid diagram (expand to render)

Key Topics

Viral Vector Delivery Systems

AAV Vectors

Serotype selection: CNS-tropic AAV capsids
AAV9 and AAVrh.10: Most common in CNS applications
Novel capsids: Engineered variants with enhanced brain targeting
Delivery routes: Intrathecal, intravenous, and direct brain injection

Delivery Optimization

Blood-brain barrier crossing: Strategies to overcome BBB
Promoter selection: Cell-type specific expression
Dose optimization: Balancing efficacy and toxicity
Repeat dosing challenges: Immune response considerations

Gene Therapy Targets

Amyloid-Targeting Genes

APP gene silencing: siRNA and antisense approaches
BACE1 knock-down: Reducing amyloid precursor cleavage
Anti-amyloid antibody genes: In vivo antibody production

Tau-Targeting Genes

MAPT gene modulation: Reducing tau expression
Tau kinase inhibitors: Gene-based delivery
Anti-tau antibody genes: Continuous antibody production

Neuroprotective Genes

BDNF delivery: Brain-derived neurotrophic factor
GDNF delivery: Glial cell line-derived neurotrophic factor
Nrf2 gene therapy: Antioxidant response activation
CLU (Clusterin): Enhancing amyloid clearance

Gene Editing Approaches

CRISPR-Cas Systems

Gene knock-out: Targeting risk genes (e.g., APOE4)
Gene knock-in: Protective allele introduction
Base editing: Precise single-nucleotide modifications
Prime editing: Broader sequence modifications

Delivery for Gene Editing

AAV-Cas9 delivery: In vivo editing systems
Lipid nanoparticle delivery: Non-viral alternatives
Ex vivo editing: Cell-based approaches

RNA-Targeted Therapies

Antisense Oligonucleotides (ASOs)

APP-targeting ASOs: Reducing amyloid production
MAPT-targeting ASOs: Reducing tau expression
SOD1-targeting ASOs: For ALS/AD overlap

siRNA Delivery

Viral-vector delivered siRNA: Long-term silencing
Non-viral nanocarriers: Safety advantages
Target cell selection: Specific neuronal populations

Clinical Development

Ongoing Clinical Trials

AAV-Mediated Gene Delivery

AAV-BDNF trials: Phase 1/2 results expected
AAV-GDNF trials: Parkinsonism studies with AD relevance
AAV-anti-amyloid trials: Novel antibody gene delivery

ASO Programs

APP-targeting ASOs: Safety and efficacy data
Tau-targeting ASOs: Initial dose-escalation results
Combination approaches: Multiple target strategies

Regulatory Considerations

Long-term follow-up: Duration requirements
Pediatric considerations: Early intervention potential
Somatic vs. germline editing: Ethical frameworks

Notable Sessions

Symposia

"Gene Therapy for Alzheimer's Disease: Where Are We?"
"AAV Vectors for CNS Disorders: Engineering the Perfect Capsid"
"RNA-Targeted Approaches in Neurodegeneration"

Workshops

"Designing Gene Therapy Clinical Trials for AD"
"Biomarkers for Gene Therapy Response"
"Manufacturing Challenges for AAV Production"

[Gene Therapy for Neurodegeneration](/investment/gene-therapy-neurodegeneration)
AAV Vector Biology
CRISPR Gene Editing
BDNF Signaling Pathway
RNA Interference Mechanisms

References

[Sengupta R, et al., Gene therapy for Alzheimer's disease: Progress and challenges. Nat Rev Drug Discov. 2024 (2024)](https://doi.org/10.1038/s41573-024-00919-0)

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

[Kantor O, et al., RNA-targeted therapeutics for neurodegenerative disease. Nat Rev Neurol. 2024 (2024)](https://doi.org/10.1038/s41582-024-00867-4)

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AAIC 2026: Gene Therapy Developments

Overview

Key Topics

Viral Vector Delivery Systems

AAV Vectors

Overview

Key Topics

Viral Vector Delivery Systems

AAV Vectors

Delivery Optimization

Gene Therapy Targets

Amyloid-Targeting Genes

Tau-Targeting Genes

Neuroprotective Genes

Gene Editing Approaches

CRISPR-Cas Systems

Delivery for Gene Editing

RNA-Targeted Therapies

Antisense Oligonucleotides (ASOs)

siRNA Delivery

Clinical Development

Ongoing Clinical Trials

AAV-Mediated Gene Delivery

ASO Programs

Regulatory Considerations

Notable Sessions

Symposia

Workshops

Related NeuroWiki Content

See Also

References