AAVATAR THERAPEUTICS
R&D
R&D
AAVATAR Therapeutics' AAV engineering platform leverages machine learning to efficiently generate novel AAV capsids. These innovative AAVs address the limitations of traditional gene delivery vectors by achieving high target specificity and stable expression at low doses.
Capsid Engineering
Process | Description |
1. Capsid Sequence Library | Hundreds of thousands of capsid sequences are designed, synthesized on a DNA printer, and assembled into a capsid library to prepare for screening. This allows the preparation of a training data set. |
2. NGS & Preditors | Among the libraries produced, AAVs with multiple characteristics important for treatment success are selected through next-generation sequencing. A process of selecting suitable AAV candidates is carried out based on analysis and prediction. |
3. Machine Learning Algorithms | A machine learning model is trained to predict the function of new base sequences, and a map suitable for AAV capsid selection is built. A more sophisticated and detailed database can be obtained through repeated learning. |
4. In vivo assay | Among the AAV candidates selected as suitable, the produced capsids undergo cross-validation model assessment selection training through in vivo assay. |
5. Hit NEW AAV Serotype | Through the above processes, you can obtain the most suitable candidate AAV that meets the customer's request based on the efficiency, specificity, immune response evasion, and manufacturability of in vivo delivery. |
Gene Regulation
Process | Description |
1. Promoter enginerring | Develop cell/tissue-specific promoters through machine learning-based screening. |
2. Gene auto regulation | Resolve the safety and sustainability of therapeutics through technology that can appropriately control the intracellular expression of gene therapy. |
Production
AAV CMC refers to the Chemistry, Manufacturing, and Control processes associated with Adeno-Associated Virus production.
In AAV gene therapy development, CMC processes encompass vector design, optimization of gene expression conditions, and validation of therapeutic efficacy.
Process | Description |
1. AAV vector design | Maximize specificity and expression efficiency for target tissues through AAV capsid selection, promoter optimization, and transgene size control. |
2. Screening and optimization of gene expression conditions | Test the expression of AAV vectors in various cell lines and culture conditions to determine optimal production conditions. Maximize AAV production by adjusting parameters such as media composition, temperature, and pH. |
3. In vitro/in vivo test | Evaluate the transduction efficiency and gene expression of AAV vectors in cell and animal models. Test various doses and administration routes to confirm the optimal therapeutic effect. |
4. Validation of effective substances | Aerify quality by analyzing the potency, purity, and integrity of AAV vectors. Quantify vector concentration and activity using methods such as qPCR and ELISA. |
5. Drug optimization | Optimize the structure and formulation of AAV vectors by considering stability, biodistribution, and immunogenicity. If necessary, improve pharmacokinetic characteristics through modifications such as PEGylation. |
* The Aavatar AAV CMC is designed based on DOE (Design of Experiment) and mechanical modeling, and is ready for scale-up and regulation.
Payload
Innovation in gene therapy, changing the lives of patients with rare and incurable diseases.
We are with you through the entire process of rare disease treatment, from the development of AAV platform technology to the production of customized treatments.
COMPANY | AAVATAR Therapeutics
CEO | Seunghee Cho
BUSINESS NUMBER | 235-81-02296
HQ
Unit 1023, Building B, 540 Misa-daero,
Hanam-si, Gyeonggi-do, Republic of Korea
BioHub
Room 203, R&D Center, Seoul Bio Hub, 117-3,
Hoegi-ro, Dongdaemun-gu, Seoul, Republic of Korea
Copyright ⓒ 2025 AAVATAR Therapeutics All rights reserved.
Innovation in gene therapy, changing the lives of patients with rare and incurable diseases.
We are with you through the entire process of rare disease treatment, from the development of AAV platform technology to the production of customized treatments.
MON — FRI (8 AM ~ 5 PM)
TEL | 070-4229-0321
EMAIL | information@aavatartps.com
Customer Center
070-4229-0321
MON~FRI 8:00 ~ 17:00
COMPANY | AAVATAR Therapeutics CEO | Seunghee Cho BUSINESS NUMBER | 235-81-02296
HQ | Unit 1023, Building B, 540 Misa-daero, Hanam-si, Gyeonggi-do, Republic of Korea
BioHub | Room 203, R&D Center, Seoul Bio Hub, 117-3, Hoegi-ro, Dongdaemun-gu, Seoul, Republic of Korea
Copyright ⓒ 2025 AAVATAR Therapeutics All rights reserved. | SITE BY. DOTCLE