Dates: March 17-21, 2026
Location: Copenhagen, Denmark
Organizer: Kenes Group
Overview
flowchart TD
events_adpd_2026_gene_therapy["AD/PD 2026 Gene Therapy"]
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events_adpd_2026_gen_0["AAV-Based Gene Delivery"]
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events_adpd_2026_gen_1["Vectors and Serotypes"]
events_adpd_2026_gene_therapy -->|"includes"| events_adpd_2026_gen_1
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events_adpd_2026_gen_2["Clinical Programs"]
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events_adpd_2026_gen_3["Gene Silencing Technologies"]
events_adpd_2026_gene_therapy -->|"includes"| events_adpd_2026_gen_3
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events_adpd_2026_gen_4["Antisense Oligonucleotides ASOs"]
events_adpd_2026_gene_therapy -->|"includes"| events_adpd_2026_gen_4
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events_adpd_2026_gen_5["siRNA Approaches"]
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... Dates: March 17-21, 2026
Location: Copenhagen, Denmark
Organizer: Kenes Group
Overview
Mermaid diagram (expand to render)
Gene therapy emerged as a rapidly evolving area at AD/PD 2026, with multiple programs advancing through clinical development. The conference showcased advances in viral vector delivery, gene silencing technologies, and novel approaches targeting both monogenic and polygenic forms of neurodegenerative diseases["@mendell2023"][@kalia2023].
AAV-Based Gene Delivery
Vectors and Serotypes Advances in AAV vector development were a major focus[@song2024]:
Novel Serotypes : Next-generation AAV variants with improved CNS tropismCross-Blood-Brain-Barrier : Vectors with enhanced BBB penetrationTargeting Specific Cell Types : Astrocyte and neuron-specific promotersManufacturing Advances : Scalable production methods for clinical supplyClinical Programs
Parkinson's Disease
AAV2-GAD - Neurologix : Glutamic acid decarboxylase gene therapy for motor symptomsVY-AADC02 - Voyager : Optimized aromatic L-amino acid decarboxylase deliveryAAV2-AADC - Meir Pharma : Ongoing clinical validationAlzheimer's Disease
BDNF Delivery : Brain-derived neurotrophic factor gene therapyAPOE4 Modulation : Gene therapy approaches to modify APOE4 riskAnti-Amyloid Gene Therapy : Novel approaches to continuous amyloid reductionGene Silencing Technologies
Antisense Oligonucleotides (ASOs) ASO technology continues to advance:
SNCA Targeting : ASOs for alpha-synuclein reduction in PDGBA Carriers : ASOs for glucocerebrosidase modulation in GBA-associated PDMAPT Targeting : Tau-targeting ASOs in ADStereotactic Delivery : Direct brain delivery approachessiRNA Approaches
Viral-Mediated RNAi : AAV-delivered short hairpin RNAsAllele-Specific Silencing : Targeting mutant alleles in genetic formsNon-Viral Delivery : Lipid nanoparticle approaches for siRNA deliveryCRISPR and Genome Editing
Base Editing Base editing technologies were highlighted as next-generation approaches[@guyon2023]:
Prime Editing : More precise genetic modificationsAllele-Specific Editing : Targeting disease-causing mutationsEpigenetic Modulation : CRISPR approaches to gene expression regulationIn Vivo Gene Editing
AAV-CRISPR : Direct delivery of CRISPR components to CNSNon-Viral Vectors : Novel delivery methods for genome editingSafety Considerations : Off-target effects and immunogenicityNeurotrophic Factor Delivery
GDNF Family
GDNF - Virin : Glial cell line-derived neurotrophic factor gene therapyNeurturin : AAV-delivered neurturin for PDBDNF Variants : Engineered brain-derived neurotrophic factorsCombination Approaches
Multi-Gene Therapy : Simultaneous delivery of multiple therapeutic genesGene Therapy + Small Molecule : Combined delivery and pharmacological approaches
Parkinson's Disease
LRRK2
Kinase Domain Mutants : Gene therapy for specific LRRK2 mutationsGene Replacement : Wild-type LRRK2 delivery for loss-of-function variantsGBA
Enzyme Enhancement : Gene therapy for glucocerebrosidaseSubstrate Reduction : Combined approaches for GBA-associated PDSNCA
Alpha-Synuclein Reduction : Multiple approaches to lower SNCA expressionAggregation Modifiers : Genes affecting alpha-synuclein aggregationAlzheimer's Disease
APP and Amyloid
APP Modulation : Gene therapy approaches to amyloid productionBACE Targeting : Genetic approaches to beta-secretase modulationAPOE
APOE4 Conversion : Gene therapy to modify APOE4 to APOE3 APOE2 Delivery : Protective APOE2 for APOE4 carriersClinical Trial Updates
Active Programs
Multiple Phase 1/2 trials showing safety and preliminary efficacy
Long-term follow-up data from early gene therapy trials
Biomarker-based patient selection for genetic subpopulations
Regulatory Pathways
Accelerated Approval : Gene therapy regulatory pathways for neurodegenerative diseasesCombination Therapy Guidance : Regulatory frameworks for gene therapy combinationsManufacturing Standards : CMC requirements for gene therapy productsFuture Directions
Next-Generation Vectors
Self-complementary AAV vectors for enhanced expression
Dual-promoter systems for cell-type specificity
Regulatable expression systems
Delivery Routes
Intranasal delivery for non-invasive CNS targeting
Systemic delivery with targeted CNS uptake
CSF administration for widespread CNS distribution
Key Takeaways
AAV technology maturing — Multiple clinical programs showing safety signalsGenetic stratification increasing — More trials targeting specific genetic subtypesDelivery advances — Novel vectors with improved brain penetrationGene silencing progressing — ASO and siRNA approaches entering CNS trialsCombination strategies — Gene therapy + pharmacological combinations emerging
See Also
[AD/PD 2026 Conference](/events/adpd-2026)](/events)
[AD/PD 2026 Company Presentations](/events/adpd-2026-company-presentations)](/events)
[AD/PD 2026 Clinical Trial Updates](/events/adpd-2026-clinical-trials)](/events)
[AD/PD 2026 Emerging Therapeutic Targets](/events/adpd-2026-emerging-therapeutic-targets)](/events)
[Events Index](/events/conference-index)
References
Unknown, AD/PD 2026 Conference Website (2026)
[Mendell JR, et al., Gene therapy for neurological disorders. Nat Rev Neurol. 2023 (2023)](https://doi.org/10.1038/s41582-023-00789-5)
[Kalia LV, et al., Gene therapy in Parkinson's disease. Nat Rev Neurol. 2023 (2023)](https://doi.org/10.1038/s41582-023-00645-9)
[Song J, et al., AAV vectors in CNS gene therapy. Mol Ther. 2024 (2024)](https://doi.org/10.1016/j.ymthe.2024.01.015)
[Guyon A, et al., CRISPR approaches to neurodegeneration. Nat Rev Drug Discov. 2023 (2023)](https://doi.org/10.1038/s41573-023-00712-5) Show full description