Plasmid Design and Preparation
2-4 Weeks
One-Stop AAV Drug Discovery Service
An AAV drug discovery platform designed to help gene therapy developers progress from DNA construct design to preclinical candidate selection. ProBio’s one-stop discovery workflow that reduces complexity, accelerates timelines, and generates data needed to advance gene therapy programs toward IND-enabling development.
Accelerate Gene Therapy Development with End-to-End AAV Discovery Services
One Stop AAV Discovery Service
An integrated platform including RUO AAV production, in vitro bioassays and in vivo pharmacology evaluation, reducing technology transfer and project handoffs.
High-Quality RUO AAV Production
RUO AAV production, comprehensive QC and customizable AAV packaging and vector design support to support improved expression, serotype selection and vector optimization.
Integrated Pharmacology Expertise
Multidimensional in vitro bioassays, comprehensive in vivo platforms and MOA-driven pharmacology strategies, reducing candidate risk selection and translational uncertainty.
RUO AAV Packaging in as Fast As 4 Weeks
AAV Packaging
1 Week
AAV Purification (optional)
0.5 Weeks
AAV QC
0.5 Weeks
AAV In Vitro Bioassay
AAV In Vivo Pharmacology
Plasmid Design and Preparation
AAV Packaging
AAV Purification (optional)
AAV QC
AAV In Vitro Bioassay
AAV In Vivo Pharmacology
2-4 Weeks
1 Week
0.5 Weeks
0.5 Weeks
RUO AAV Production
High-Quality AAV Vectors: Manufactured to rigorous standards with titers ≥1E+13 GC/mL, purity >95%, empty capsid ratios.
Flexible Serotype Options: Available across a broad range of AAV serotypes and customizable for engineered or specialty variants.
Comprehensive Quality Control: Extensive testing for titer, sterility, purity, endotoxin, genome quantification, and advanced vector characterization.
Flexible Serotype Options: Available across a broad range of AAV serotypes and customizable for engineered or specialty variants.
Comprehensive Quality Control: Extensive testing for titer, sterility, purity, endotoxin, genome quantification, and advanced vector characterization.
In Vitro Bioassay Platform
AAV Transduction: Assess vector infectivity, transgene expression, and gene delivery efficiency using Luc and GFP reporter assays.
Transgene Expression: Evaluate transgene activity at the DNA, RNA, and protein levels using gene-editing analysis, RNAScope, qPCR, ddPCR, FISH, FACS, WB, ELISA, IF, IHC, and BLI.
Target-Specific Functional Bioactivity: Evaluate biological activity using cell cytotoxicity, cell proliferation, cell cycle, and reporter gene assays.
Transgene Expression: Evaluate transgene activity at the DNA, RNA, and protein levels using gene-editing analysis, RNAScope, qPCR, ddPCR, FISH, FACS, WB, ELISA, IF, IHC, and BLI.
Target-Specific Functional Bioactivity: Evaluate biological activity using cell cytotoxicity, cell proliferation, cell cycle, and reporter gene assays.
In Vivo Bioassay Platform for AAV Drug Discovery
Efficacy: Evaluate therapeutic efficacy in disease-relevant animal models, supported by diverse AAV dosing routes and surgical capabilities.
PK & Bioanalysis: Characterize AAV biodistribution, viral shedding, transgene expression, and immunogenicity through comprehensive ADME and anti-AAV antibody assessments.
Toxicology: Assess safety through anti-AAV antibody screening, bioanalytical method development, toxicokinetic studies, immunogenicity testing, and toxicity evaluations.
PK & Bioanalysis: Characterize AAV biodistribution, viral shedding, transgene expression, and immunogenicity through comprehensive ADME and anti-AAV antibody assessments.
Toxicology: Assess safety through anti-AAV antibody screening, bioanalytical method development, toxicokinetic studies, immunogenicity testing, and toxicity evaluations.
Case Studies
Optimizing AAV Serotype Selection Through Integrated Biodistribution Analysis
AAV-Mediated Bispecific Antibody Delivery Demonstrates Dose-Dependent Tumor Growth Inhibition In Vivo
Resources
FLYER
In Vivo Pharmacology
Solutions including efficacy, biological Activity, DMPK, bioanalysis and toxicity.
BROCHURE
Biologics Discovery Services Brochure
Tailored discovery strategies for diverse biologics modalities, designed to enhance success rates.
FLYER
Comprehensive Target Based Bioassay Overview
Learn more about our full cell-based bioassay list for different targets.
BROCHURE
In Vitro Pharmacology Solutions
Comprehensive bioassay offerings, helping smooth the journey from discovery to lead molecule.
Frequently Asked Questions