AAV Library Services for Gene Therapy Research in Southeast Asia

AAV Library Services for Gene Therapy Research in Southeast Asia

Are you looking to:

Optimize tissue specificity and serotype tropism?
Enhance gene expression levels and tissue specificity?
Discover novel therapeutic targets through gene editing?

Packgene’s comprehensive AAV library design services are tailored to address your specific research needs.

Our AAV Library Design Expertise

  • Capsid Library Design
  • Gene of Interested (GOI) Library Design
  • Gene Editing Libraries

Generate diverse AAV serotypes for targeted gene delivery using techniques like DNA shuffling.

Our capsid library design focuses on helping you find AAV serotypes to optimise for the enhancement of tissue specificity and efficiency of gene delivery (serotype tropism). This can help you minimise the risk of off-target effects and bypass innate immune responses in gene therapy applications.

Optimize gene expression and tissue specificity through promoter engineering.

Our Gene of Interest (GOI) library ensures that your therapeutic genes are expressed at the right levels in the desired tissue.

Design custom CRISPR libraries for high throughput screening to achieve precise gene editing and functional genomics. Identify critical genes and pathways involved in various diseases and uncover potential therapeutic targets.

Our libraries include custom CRISPR sgRNA libraries, CRISPR activation libraries, CRISPRi libraries, CRISPR knockout libraries and shRNA libraries for:

  • Gene function screening
  • Gene knockdown studies
  • Disease modeling
  • Drug Target Discovery
  • Cancer Research

Our Library Design Capabilities

1. Random Peptide Library

Random Peptide Library Design Services

Create libraries with diverse peptide sequences for exploring protein function and interaction.

How do we do it?

Up to 20 amino acids at each site with the random peptide insertion. Combinatorial mutation throughout the peptide insertion.

2. DNA Shuffling Library

Packgene DNA Shuffling Library Services

Generate combinatorial libraries to optimize protein properties.

How do we do it?

Pre-synthesized gene library ligate together to create a combinatorial gene shuffling library. This will generate ~100 ready-to-use capsid genes.

3. Precision Library

Packgene Precision Library Services

Design libraries with precise mutations at specific sites.

How do we do it?

A short precision oligo pool that encodes gRNA library or other predesigned sequence insert into the vector. Each variant sequence can be predefined and precisely synthesized

4. Barcoded Library

Packgene Barcoded Library Services

Track individual cells and their genetic modifications.

How do we do it?

Your variant will be labelled with a barcode that is adjacent or distant from the variable region. The barcode will enable easy variant identification by NGS or track cell lineage.

5. Site Saturation Library and Saturation Scanning Library

Explore the functional impact of amino acid substitutions.

Packgene Site Saturation Library Services
Site-Saturation Library

How do we do it?

Up to 20 amino acids at single sites with 1 amino acid change per variant

Saturation Scanning Library Service
Saturation Scanning Library

How do we do it?

Up to 20 amino acids at a series of sites that you choose with 1 amino acid change per variant

6. Combinatorial Mutant Library

Packgene Combinatorial Mutant Library Service

Generate libraries with multiple mutations to identify synergistic effects.

How do we do it?

You define the amino acid or nucleotide components and position; we generate the library from the template. Multiple mutations per variant in a combinatorial way.

Our commitment to Quality

High Library Coverage

Near 100% coverage of our precision library

Ensure comprehensive coverage of the desired sequence space.

Exceptional Library Uniformity

Minimize bias and maximize the effectiveness of your screening experiments.

Low Mispackaging Rates

Mismatched capsid versus genome can be assessed by introducing a low frequency of capsid genes with early stop codons. The frequency of these stop codons indicates the mismatched rate. Our typical mispackaging rate is less than 0.1%.

Our advanced AAV packaging protocols minimize the risk of incorrect packaging, ensuring accurate and reliable results.

Screening Results

Final validation of novel muscle targeting Capsid screened in Cynomolgus Monkey

Packgene AAV Services Case Study

  • Case 1
  • Case 2
  • Case 3
  • Case 4
Screenshot of a mother’s testimonial in Chinese dated September 19, 2024, describing her two children’s improvements after 13 days of gene therapy and anti-epileptic medication. She shares that one child no longer has seizures for 11 days, the other can now walk, eat, and ride a bicycle, and her child has started practicing climbing stairs again.
  • Spinal muscular atrophy with myoclonic epilepsy (SMA-PME, ASAH1 gene defect)
  • Single dose, intrathecal and intracerebroventricular injection.
Screenshot of a research article titled “AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial,” reporting that in six children with OTOF-related hereditary deafness, five showed significant improvement in hearing and speech after AAV1-hOTOF treatment, demonstrating good safety and efficacy.
  • OTOF biallelic mutations cause severe hearing impairment or complete hearing loss.
  • Single dose, cochlear injection.
Screenshot of a Chinese news article reporting that GenAns Biotech (Jienda Jiuzhou) achieved a breakthrough with its GA001 gene therapy. A patient with total blindness regained light perception under the guidance of Professor Zhang Xuxiang (张旭乡), showing early evidence of safety and efficacy in treating retinal degenerative diseases.
  • Advanced blindness caused by retinitis pigmentosa.
  • Single dose, intraocular intravitreal injection.
Screenshot of a Chinese news article reporting that SineuGene Therapeutics announced positive clinical progress for its SNUG01 gene therapy in amyotrophic lateral sclerosis (ALS). The study, led by Professor Fan Dongsheng at Peking University Third Hospital, showed no serious adverse reactions and early signs of safety and therapeutic potential.
  • Amyotrophic lateral sclerosis (ALS).
  • Single dose, intrathecal injection.

Download To View More AAV Preclinical Case Studies

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