AAVATAR THERAPEUTICS


R&D



R&D

PLATFORM


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

ProcessDescription

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

ProcessDescription

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.

ProcessDescription

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.



MON — FRI (8 AM ~ 5 PM)

TEL  |  070-4229-0321

EMAIL  |  information@aavatartps.com




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


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