AAVnerGene


AAVnerGene is a leading biotech company specializing in advanced gene therapy technologies using the AAV platform. With over 20 years of expertise, they develop innovative solutions including AAV vector design, packaging, capsid library construction, and screening. Their proprietary ATHENA platform enables efficient identification and evolution of AAV capsids for targeted gene delivery, addressing challenges such as immune responses and high vector doses. The company collaborates with academic and biotech partners to push the frontiers of gene therapy, offering a comprehensive range of AAV products, services, and custom solutions to accelerate therapeutic development.

Industries

biotechnology
life-science
manufacturing

Nr. of Employees

small (1-50)

AAVnerGene

9620 Medical Center Dr, Suite 100, Rockville, MD 20850


Patents

Modified plasma clotting factor VIII and method of use thereof

US-12583907-B2

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Modified plasma clotting factor VIII and method of use thereof

US-11795207-B2

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Products

Plasmid sets for single-, dual- and triple-plasmid production workflows

Transfer and helper plasmid sets formatted for single-, dual- and triple-plasmid AAV production workflows and for capsid/library construction.

Premade AAV viral vectors (multiple serotypes, promoters, reporters)

Ready-to-use AAV vectors supplied in multiple serotypes with defined genomes and characterized titers and QC for gene delivery in vitro and in vivo.

AAV reference materials (full, truly-empty, byproduct-empty)

Standardized AAV reference materials for assay calibration and QC, including fully packaged genomes, truly-empty capsids and byproduct-empty particles with documented characterization.

Capsid testing kits and barcode kits

Kits to enable capsid-barcode testing and pooled serotype/variant evaluation to support empirical selection using barcode sequencing and NGS.

Premade high-complexity capsid libraries

Ready-to-use capsid libraries with random peptide insertions at selected capsid variable regions, provided in multiple serotypes and library sizes for directed evolution.

Live-cell cAMP biosensor GPCR assay kits

Assay kits based on live-cell cAMP biosensors for rapid high-throughput functional screening of GPCRs.

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Services

Consultation, sequence design and construction of AAV transfer plasmids with selection of regulatory elements and optimization for target tissues and applications.

Custom packaging of AAV vectors across multiple serotypes using plasmid-based production workflows with defined QC testing and turnaround options.

Laboratory-based analytical testing for genome quantification, capsid titer, full/empty ratio, purity and standard safety endpoints to support release and downstream studies.

Comparative testing of capsids and regulatory elements across cell types and animal models (in vitro and in vivo) to guide capsid selection for specific tissues and applications.

Design, construction, production and selection/evolution of barcoded and random-insertion capsid libraries with support for pooled NGS-based screening in small and large animal models.

Project consulting covering experimental planning, construct design, transfection and assay configuration support for AAV-based studies.

Expertise Areas

  • AAV transfer vector design and construct optimization
  • Plasmid-based AAV manufacturing and scale-up (single/dual/triple plasmid workflows)
  • Capsid engineering including directed evolution, peptide insertion libraries and DNA shuffling
  • High-complexity capsid library generation and barcoded pooled screening
  • Show More (4)

Key Technologies

  • Next-generation sequencing (barcode-seq) for pooled screens
  • Barcoded capsid libraries
  • Random-peptide insertion libraries (NNK insertion) with >1e9 complexity
  • DNA shuffling / recombination libraries
  • Show More (13)

News & Updates

The AAVone system generates over 5E15 VG/L AAV9 vectors in suspension HEK 293 cells, showcased at ASGCT 2025.

The AAVone system achieved high productivity in large-scale testing (XDR200).


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