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One-Stop AAV Drug Discovery Service

From DNA to Preclinical Candidate

Adeno-associated viruses (AAVs) have emerged as leading vectors in gene therapy due to their safety and ability to support long-term gene expression. However, AAV development presents significant challenges—including serotype selection, vector design, expression efficiency, and in vivo targeting.

ProBio‘s integrated AAV drug discovery service accelerates the R&D process with high-quality Research Use Only (RUO) AAV packaging and integrated pharmacology evaluation.

Features

One-Stop AAV Discovery Service
- RUO AAV production
- In vitro bioassay
- In vivo pharmacology
High-Quality RUO AAV Packaging
- Customizable for various special variants
- Comprehensive AAV QC methods
- Guarantee high-quality delivery
Integrated Pharmacology Platform
- Multidimensional in vitro activity evaluation
- Comprehensive and integrated in vivo platforms
- MOA-based pharmacology strategies for AAV Drug

RUO AAV Production

Plasmid Design

Plasmid Construction

AAV Packaging

AAV Purification (optional)

2-4 weeks

1 week

0.5 week

Service Highlights

High quality
- Titer>=1E+13 GC/ml
- Purity > 95%
- Empty Capsid Ratio< 10%
- Endotoxin < 10 EU/ml
Available for a wide range of serotypes
- AAV1, 2, 5, 6, 7, 8, 9, PHP.eB, AAV2.7m8, rh10, DJ, DJ-8, AAV2-retro, etc.
- Customizable for various special variants
Comprehensive AAV quality control methods
- Physical and functional titer
- Bacteria/fungi/mycoplasma
- Purity, ddPCR, Endotoxin
- AUC, TEM, GOI sequencing, etc.

In Vitro Bioassay for AAV Drug Discovery

We can perform in vitro activity evaluation of AAV drugs from multiple dimensions.

Transduction efficiency	Target gene expression	Functional evaluation
AAV Transduction ✔ AAV-based Luc/GFP reporter assay	Transgene Expression ✔ DNA: DNA editing efficiency ✔ RNA: RNAScope, qPCR, ddPCR, FISH ✔ Protein: FACS, WB, ELISA, IF, IHC, BLI	Target-specific functional bioactivity ✔ Cell cytotoxicity ✔ Cell proliferation ✔ Cell cycle ✔ Reporter gene assay

In Vivo Pharmacology for AAV Drug Discovery

Our rich expertise and resources empower us to craft customized pharmacology solutions that align with the characteristics of each AAV therapy

Efficacy Study	Various animal models for major AAV therapeutic areas （Rodent and Non-rodent）	Genetic disease Neurological disease Cardiovascular and metabolic disease Oncology and autoimmune disease Infectious disease Lung, kidney and GI disease
Efficacy Study	Surgical Techniques for Various AAV Dosing Routes	IC,ICV,IT,IN,IVT,IVG,IV,SC,IP,IM,FP,ID
PK and Bioanalysis	Comprehensive PK and bioanalysis platform	ADME: Functional protein detection & virus shedding & virus bio-distribution & GFP or Luc imaging Immunogenicity (Anti-AAV Ab screening & confirmation)
Toxicology	Toxicology studies for pre-CMC/IIT	✔ Anti-AAV Ab screening ✔ Bioanalytical method ✔ Toxicokinetics ✔ Immunogenicity ✔ Toxicity study ✔ FIH translational support