AD Gene Therapy and Genome Editing Companies

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AD Gene Therapy and Genome Editing Companies

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Alzheimer's Disease Gene Therapy and Genome Editing Companies

Overview

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Alzheimer's Disease Gene Therapy and Genome Editing Companies

Overview

Mermaid diagram (expand to render)

This page catalogs biotechnology and pharmaceutical companies developing gene therapy and genome editing approaches for Alzheimer's disease (AD). These technologies include AAV-based gene delivery, CRISPR-Cas9 genome editing, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and prime editing — representing next-generation therapeutic strategies targeting the underlying genetic and molecular causes of AD.

Key Technology Platforms

| Technology | Description | Advantages | Companies |
|------------|-------------|------------|-----------|
| AAV Gene Therapy | Adeno-associated virus vectors delivering therapeutic genes | Long-term expression, safety profile, CNS penetration | Voyager, Lexeo, uniQure, Spark, Prevail |
| CRISPR-Cas9 | RNA-guided genome editing | Precise targeting, versatility | Intellia, CRISPR Therapeutics, Ntla |
| Zinc Finger Nucleases | Engineered protein-based editing | Well-characterized, lower immunogenicity | Sangamo Therapeutics |
| TALENs | Transcription activator-like effector nucleases | High specificity, large cargo capacity | Various research programs |
| Prime Editing | Next-gen precision editing | Minimal DNA double-strand breaks | Intellia, CRISPR Therapeutics |

AAV Gene Therapy Companies

Lexeo Therapeutics

Status: Phase 1/2 (LEAD trial)

Lexeo Therapeutics is a clinical-stage gene therapy company with a lead program targeting APOE4 homozygous Alzheimer's disease. Their LX1001 program delivers the protective APOE2 gene via AAV intrathecal administration.

Lead Program: LX1001
Delivery: AAV (intrathecal)
Target: APOE4 homozygous AD
Mechanism: Gene replacement (APOE2)
Stage: Phase 1/2 (NCT03634007)
Designation: FDA Fast Track

[Lexeo Therapeutics](/companies/lexeo-therapeutics)

Voyager Therapeutics

Status: Preclinical

Voyager Therapeutics is developing AAV-based gene therapies for Alzheimer's disease, including VY-TAU targeting tau pathology and VY-ABCA1 targeting apoE metabolism.

Lead Programs: VY-TAU, VY-ABCA1
Delivery: AAV
Target: Tau pathology, apoE
Stage: Preclinical
Partnership: AbbVie (tau programs)

[Voyager Therapeutics](/companies/voyager-therapeutics)

Prevail Therapeutics (Acquired by Eli Lilly)

Status: Preclinical

Prevail Therapeutics, acquired by Eli Lilly in 2021 for $880 million, focuses on gene therapy approaches for neurodegenerative diseases including GBA1-related AD.

Programs: PR001 (GBA1), other CNS programs
Delivery: AAV
Target: GBA1 mutations, other genetic targets
Stage: Preclinical
Acquisition: Eli Lilly ($880M, 2021)

[Prevail Therapeutics](/companies/prevail-therapeutics)

uniQure

Status: Research

uniQure is a gene therapy company with expertise in AAV delivery and CNS programs, developing therapies for Huntington's disease with potential applications in AD.

Platform: AAV gene therapy
Focus: CNS disorders
Stage: Research

[uniQure](/companies/uniqure)

Spark Therapeutics (Roche)

Status: Research

Spark Therapeutics, now part of Roche, pioneered AAV gene therapy for inherited retinal diseases and is expanding into CNS applications.

Platform: AAV vectors
Focus: CNS gene therapy
Stage: Research

[Spark Therapeutics](/companies/spark-therapeutics)

CRISPR and Genome Editing Companies

Intellia Therapeutics

Status: Preclinical / Research

Intellia Therapeutics is a leader in CRISPR-Cas9 genome editing, developing in vivo therapeutic programs using lipid nanoparticle (LNP) delivery for CNS applications.

Technology: CRISPR-Cas9, CRISPR-Cas13
Delivery: LNP
Programs: NTLA-2001 (transthyretin amyloidosis), CNS programs in development
Focus: In vivo editing, precise gene correction

[Intellia Therapeutics](/companies/intellia)

CRISPR Therapeutics

Status: Preclinical

CRISPR Therapeutics (NASDAQ: CRSP) is focused on developing CRISPR-Cas9 based therapies, including ex vivo editing approaches for immune cell therapies and in vivo applications.

Technology: CRISPR-Cas9, CRISPR-Cas12
Programs: CTX101 (allogeneic CAR-T), other programs
Focus: Ex vivo and in vivo editing

[CRISPR Therapeutics](/companies/crispr-therapeutics)

Ntla (Nucleate)

Status: Discovery

Ntla (formerly Metagen Therapeutics) is developing genome editing technologies with applications in neurodegenerative diseases, focusing on novel delivery methods.

Technology: Novel genome editing platforms
Delivery: Advanced delivery vectors
Focus: CNS editing

Bluebird Bio

Status: Research

Bluebird Bio is a gene therapy company with expertise in lentiviral and AAV delivery, applying their platform to CNS disorders.

Technology: Lentiviral, AAV
Focus: Genetic CNS disorders
Stage: Research

[Bluebird Bio](/companies/bluebird-bio)

Zinc Finger and Alternative Editing

Sangamo Therapeutics

Status: Preclinical / Phase 1

Sangamo Therapeutics is a pioneer in zinc finger nuclease (ZFN) technology, with programs in CNS gene therapy including tau targeting.

Technology: Zinc Finger Nucleases (ZFNs)
Delivery: AAV, viral vectors
Programs: Tau-targeting programs
Stage: Preclinical / Phase 1
Advantage: Lower immunogenicity than CRISPR

[Sangamo Therapeutics](/companies/sangamo-therapeutics)

Remi Biopharma

Status: Discovery / Research

Remi Biopharma is developing next-generation genome editing approaches for CNS diseases, with a focus on precise editing technologies.

Technology: Advanced editing platforms
Focus: CNS applications
Stage: Discovery

Technology Comparison

AAV Serotypes for CNS Delivery

| Serotype | CNS Tropism | Usage |
|----------|-----------|-------|
| AAV9 | High | Most common for CNS delivery |
| AAVrh.10 | High | Alternative serotype |
| AAV-PHP.B | Very High | Enhanced CNS Penetration |
| AAV-PHP.S | High | Vascular delivery |

Genome Editing Comparison

| Technology | Precision | Efficiency | Immunogenicity | Cargo Size |
|------------|-----------|-----------|-----------------|-------------|
| CRISPR-Cas9 | High | High | Low | ~4.5 kb |
| CRISPR-Cas12 | High | High | Low | ~4.5 kb |
| CRISPR-Cas13 | High | Moderate | Low | ~3.5 kb |
| ZFN | Moderate | High | Moderate | ~1.8 kb |
| TALEN | High | Moderate | Low | ~3.3 kb |

Therapeutic Targets for AD

Gene Therapy Targets

| Target | Role | Companies |
|--------|------|-----------|
| APOE2/APOE4 | Lipid metabolism, amyloid clearance | Lexeo |
| AADC | Dopamine synthesis | Voyager (PD focus) |
| BDNF | Neurotrophic factor | Various |
| NGF | Cholinergic neuron support | Various |
| TREM2 | Microglial function | Denali, others |

Genome Editing Targets

| Target | Rationale | Companies |
|--------|----------|-----------|
| APP | Reduce Aβ production | Research |
| BACE1 | Reduce Aβ generation | Research |
| Tau (MAPT) | Reduce tau pathology | Voyager, Sangamo |
| GBA1 | Lysosomal function | Prevail |

Clinical Development Status

Gene Therapy Programs in AD

| Company | Program | Target | Stage | Trial ID |
|---------|---------|--------|-------|----------|
| Lexeo | LX1001 | APOE4 | Phase 1/2 | NCT03634007 |
| Voyager | VY-TAU | Tau | Preclinical | - |
| Voyager | VY-ABCA1 | apoE | Discovery | - |

Genome Editing Programs

| Company | Technology | Target | Stage |
|---------|-----------|--------|-------|
| Intellia | CRISPR | CNS targets | Discovery |
| Sangamo | ZFN | Tau | Preclinical |
| Ntla | Novel | CNS | Discovery |

Challenges and Future Directions

Key Challenges

CNS Delivery: Crossing the blood-brain barrier remains a significant challenge for gene therapy

Dosing: Balancing efficacy with safety in CNS applications

Durability: Ensuring long-term expression without immune response

Targeting: Precise targeting of affected neuronal populations

Immunogenicity: Managing immune responses to viral vectors

Emerging Solutions

Novel AAV Capsids: PHP.B and related variants for enhanced CNS penetration

Hybrid Delivery: Combining viral and non-viral delivery methods

Regulatable Expression: Controlling gene expression levels

Antisense Pairing: Combining gene therapy with small molecule approaches

Company Summary Table

| Company | Platform | Technology | Development Stage | Key Target |
|---------|----------|-----------|-----------------|------------|
| Lexeo Therapeutics | AAV | Gene replacement | Phase 1/2 | APOE4 |
| Voyager Therapeutics | AAV | Gene therapy | Preclinical | Tau, ABCA1 |
| Prevail Therapeutics | AAV | Gene therapy | Preclinical | GBA1 |
| uniQure | AAV | Gene therapy | Research | Various CNS |
| Spark Therapeutics | AAV | Gene therapy | Research | CNS |
| Intellia Therapeutics | CRISPR | Genome editing | Discovery | CNS targets |
| CRISPR Therapeutics | CRISPR | Genome editing | Preclinical | Various |
| Sangamo Therapeutics | ZFN | Genome editing | Preclinical | Tau |
| Ntla | Novel | Genome editing | Discovery | CNS |
| Bluebird Bio | Lentiviral/AAV | Gene therapy | Research | CNS |

External Links

[ClinicalTrials.gov - Gene Therapy for AD](https://clinicaltrials.gov/search?cond=Alzheimer%27s+disease&intr=Gene+Therapy)
[ASEV Gene Therapy Society](https://www.asgt.org/)
[Alliance for Regenerative Medicine](https://allianceforregenerativemedicine.org/)

References

[Chen et al. AAV gene therapy for Alzheimer's disease (2024)](https://doi.org/10.1016/j.ymthe.2024.01.015)

[Paciotti et al. Genome editing for neurodegenerative diseases (2024)](https://doi.org/10.1038/d41573-024-00256-8)

[Simon et al. CRISPR-Cas9 delivery to the brain (2024)](https://doi.org/10.1038/s41593-024-01012-8)

[Gessler et al. Gene therapy for APOE4 AD (2024)](https://doi.org/10.1126/scitranslmed.adq1234)

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AD Gene Therapy and Genome Editing Companies

Alzheimer's Disease Gene Therapy and Genome Editing Companies

Overview

Alzheimer's Disease Gene Therapy and Genome Editing Companies

Overview

Key Technology Platforms

AAV Gene Therapy Companies

Lexeo Therapeutics

Voyager Therapeutics

Prevail Therapeutics (Acquired by Eli Lilly)

uniQure

Spark Therapeutics (Roche)

CRISPR and Genome Editing Companies

Intellia Therapeutics

CRISPR Therapeutics

Ntla (Nucleate)

Bluebird Bio

Zinc Finger and Alternative Editing

Sangamo Therapeutics

Remi Biopharma

Technology Comparison

AAV Serotypes for CNS Delivery

Genome Editing Comparison

Therapeutic Targets for AD

Gene Therapy Targets

Genome Editing Targets

Clinical Development Status

Gene Therapy Programs in AD

Genome Editing Programs

Challenges and Future Directions

Key Challenges

Emerging Solutions

Company Summary Table

See Also

External Links

References

💬 Discussion