Advancing AAV Innovation Through Collaboration

At AAVnerGene, we believe the next generation of gene therapies will be built through strong scientific partnerships. We welcome collaboration with academic laboratories, biotechnology companies, pharmaceutical organizations, research institutes, hospitals, investors, and manufacturing partners working across the AAV field.

Our team has extensive experience in choroid plexus (ChP) targeting gene therapy, AAV production, capsid engineering, vector design, process development, analytical characterization, and gene therapy research. Whether you are developing a new therapeutic program, improving an existing vector, evaluating a novel capsid, or addressing a manufacturing challenge, we are ready to explore how our technologies and expertise can support your goals.

Areas of Collaboration

Choroid Plexus (ChP)-Targeted Gene Therapy

AAVnerGene is actively seeking collaborators to develop the next generation of Choroid Plexus (ChP)-targeted gene therapies. Leveraging our proprietary AAV-ChP capsids, we aim to enable selective gene delivery to the choroid plexus following intracerebroventricular (ICV) or Intrathecal (IT) administration, opening new therapeutic opportunities for central nervous system (CNS) diseases. Our ChP-targeting platform offers a unique approach to modulating cerebrospinal fluid (CSF) composition and delivering therapeutic proteins throughout the CNS while minimizing off-target transduction of neurons and peripheral organs.

We welcome collaborations focused on:

• Alzheimer’s disease (AD)
• Parkinson’s disease (PD)
• Amyotrophic lateral sclerosis (ALS)
• Huntington’s disease (HD)
• Frontotemporal dementia (FTD)
• Lysosomal storage diseases
• Neurodevelopmental disorders
• Hydrocephalus
• Neuroinflammation
• Depression and psychiatric disorders
• Sleep disorders
• Rare CNS diseases
• CNS protein replacement therapies
• CSF-based biologic delivery
• Novel CNS therapeutic targets

We are interested in partnering to:

• Discover and validate new therapeutic targets for ChP-mediated gene therapy
• Evaluate therapeutic genes using AAV-ChP vectors
• Develop secreted biologics delivered through the CSF
• Perform efficacy studies in rodent and non-human primate disease models
• Optimize ICV and intrathecal (IT) delivery strategies
• Advance ChP-targeted therapeutics toward clinical development

Whether your program is at the discovery, preclinical, or translational stage, AAVnerGene is excited to explore collaborations that leverage our proprietary ChP-targeting technology to develop transformative therapies for neurological diseases.

In Vivo T-Cell Engineering

Our engineered AAV-rT capsid is designed for efficient targeting and transduction of resting T cells, providing a potential delivery platform for direct in vivo CAR-T and T-cell gene engineering.

Partnership opportunities include:

• In vivo delivery of CAR constructs
• Gene editing and targeted CAR knock-in
• Combined CAR expression and gene knockout
• Development of in vivo T-cell therapies for cancer, autoimmune diseases, and other indications
• Evaluation and optimization in relevant preclinical models

Lung Macrophage-Targeted Gene Delivery

Our engineered AAV-ShD™ capsid demonstrates strong lung tropism and liver detargeting in non-human primates, supporting its development as a platform for lung-targeted and macrophage-directed gene delivery.

Potential collaborations include:

• In vivo lung macrophage engineering
• Delivery of therapeutic genes to pulmonary immune cells
• CAR-macrophage (CAR-M) and other macrophage-engineering strategies
• Gene therapy for lung cancer and pulmonary diseases
• Preclinical evaluation and translational development

AAV Capsid Discovery and Engineering

AAVnerGene offers multiple capsid discovery and evolution platforms for developing vectors with improved tissue targeting, transduction efficiency, biodistribution, and manufacturability.

Collaboration opportunities include:

• Screening of natural and engineered AAV capsid libraries
• In vitro and in vivo capsid selection
• Tissue- and cell-specific capsid development
• Capsid evolution for CNS, liver, muscle, lung, immune cells, and other targets
• Evaluation of proprietary or partner-provided capsids
• Comparative studies against established AAV serotypes

Therapeutic Vector Development

We welcome partnerships involving the design and development of AAV-based therapeutic candidates.

Our capabilities include:

• Therapeutic gene selection and vector design
• Promoter and regulatory element optimization
• Transgene engineering
• Capsid and genome combination studies
• Small-scale feasibility testing
• Preclinical vector production and characterization
• Support for disease-focused research programs

AAV Manufacturing Technology

AAVnerGene welcomes collaborations with biotechnology companies, pharmaceutical organizations, CDMOs, and academic institutions seeking to improve AAV productivity, vector quality, process simplicity, scalability, and manufacturing cost efficiency. Our partnership model provides access to an integrated suite of AAV manufacturing technologies that can be evaluated individually or combined into a complete production platform.

• Evaluation and implementation of the AAVone® single-plasmid production platform
• Benchmarking AAVone® against existing dual- or triple-plasmid production systems
• Optimization of AAV productivity and full-particle packaging efficiency
• Evaluation and adoption of HEK293one® suspension producer cells
• Process optimization using CelliMax™ Medium, PEIone™, and ENone™
• Development of customized AAV production workflows and producer cell platforms
• Process development, scale-up, and technology transfer
• Integration with partners’ proprietary AAV capsids, vector genomes, and manufacturing processes
• Licensing and co-development of AAV manufacturing technologies
• Support for translation from research-scale production to large-scale manufacturing

AAVnerGene’s manufacturing platform brings together five complementary technologies:

• AAVone® — A proprietary single-plasmid AAV production system integrating the essential components required for AAV production into one plasmid, simplifying transient transfection while reducing DNA requirements and process complexity.
• HEK293one® — A high-performance suspension HEK293 producer cell line optimized for efficient transient transfection and high-yield AAV production. It can be used with AAVone® as well as conventional AAV production systems.
• PEIone™ — A transfection reagent optimized for suspension HEK293 cells and AAV manufacturing, supporting efficient and consistent DNA delivery.
• ENone™ — A proprietary AAV production enhancer designed to further improve vector productivity and manufacturing performance.
• CelliMax™ Medium — A chemically defined suspension culture medium designed to support high-density HEK293 growth, robust viability, efficient transfection, and high AAV productivity.

One Platform. Multiple Opportunities for Collaboration.

AAVone®+ HEK293one™+  PEIone™ + ENone™ + CelliMax®

Partners can evaluate individual technologies or implement the fully integrated AAVnerGene manufacturing platform, depending on their existing process and development needs.

Partner with AAVnerGene to simplify AAV manufacturing, improve productivity and vector quality, and accelerate the development of scalable and cost-effective AAV production processes. By optimizing the producer cell, culture medium, transfection reagent, production enhancer, and AAV plasmid system as an integrated platform, AAVnerGene aims to provide a simplified and scalable solution for next-generation AAV manufacturing.

AAV Analysis and Characterization

AAVnerGene provides analytical services to compare vector quality and manufacturing performance across different systems and production conditions. Available analyses may include:

• Vector genome titer
• Capsid titer
• Full-to-empty capsid ratio
• Genome integrity
• Infectious titer and potency
• Residual plasmid DNA and process-related impurities
• Capsid protein analysis
• Comparative lot characterization

Flexible Collaboration Models

Every project is different. We offer flexible engagement models based on the scientific goals, development stage, and commercial needs of each partner.

Why Collaborate with AAVnerGene?

AAVnerGene combines scientific innovation with practical experience in AAV development and manufacturing.

Our partners benefit from:

• Deep expertise across the complete AAV workflow
• Proprietary AAV production and capsid engineering platforms
• Flexible project structures
• Rapid feasibility testing
• Direct access to experienced AAV scientists
• Support from early research through commercial development
• A collaborative approach focused on solving real technical challenges

Examples of Potential Collaborations

We are interested in discussing projects involving:

• High-yield AAV manufacturing
• Improved full capsid packaging
• Difficult-to-produce AAV serotypes
• Novel tissue-targeting capsids
• CNS and choroid plexus targeting
• Immune-cell targeting, including T cells, NK cells, and macrophages
• Liver-detargeted or tissue-restricted vectors
• Gene therapy for rare and common diseases
• Gene editing and in vivo delivery
• AAV potency and infectivity assays
• Cell-line and reagent development
• AAV analytical method development
• Technology licensing and commercialization

Let’s Build the Next AAV Innovation Together

We welcome early-stage ideas, defined development programs, technology evaluations, and long-term strategic partnerships.

Whether you already have an AAV candidate or are just beginning to explore a new concept, we would be pleased to learn about your research goals and discuss how AAVnerGene can support your program.

Contact Us

Please share a brief description of your project, including:

• Target disease or application
• AAV serotype or capsid, if known
• Therapeutic gene or payload
• Current development stage
• Desired collaboration model
• Expected timeline

Contact AAVnerGene today to explore a potential collaboration.

Together, we can accelerate AAV innovation and bring better gene therapies to patients.