Porton Advanced and BioMap Unveil AI-Powered AAV Solution, Pioneering a New Era in Gene Therapy

Since November 2023,Porton Biologics Ltd. Announces Strategic Partnership with BioMapBoth parties are committed to utilizing cutting-edge large-scale life science models to assist in the development of Adeno-Associated Virus (AAV)-related algorithms. After six months of close collaboration, Porton Biologics Ltd. and BioMap achieved a significant milestone in July 2024.Successful Launch of AI-Driven AAV Solution Opens a New Chapter in Gene Therapy。

Co-create a New Chapter in AAV Design

Based on BioMap's AIGP platform and xTrimoPGLM large model, combined with Porton Biologics Ltd.'s rich professional experience in AAV libraries and experimental validation, a set of efficient and precise one-click AAV library design services has been constructed.This innovative achievement enables users to efficiently design and optimize AAV libraries covering more than 10 serotypes, including point mutations, short peptide insertions, and fragment recombination, to achieve higher transduction efficiency, tissue and organ specificity, and immune escape capabilities.

BioMap's xTrimoPGLM large model, which integrates a vast amount of multimodal protein data (sequence, structure, and other properties), provides a unified pre-training framework and foundational model for AAV design. Based on this large model, we utilize a series of AI models to comprehensively optimize key metrics such as assembly efficiency, tissue targeting specificity, and immune escape capability, ensuring that the generated AAV sequences exhibit higher functional performance and diversity, significantly improving library screening success rates. The platform demonstrates strong generalization capabilities, enabling high-precision AAV function prediction even in unseen mutation regions and serotypes.

Moreover, in response to customers' personalized needs and specific data, BioMap and Porton Biologics Ltd. will alsoProvide customized AI design services, we will fine-tune the model based on the customer's unique data to achieve more accurate and personalized design optimization.

xTrimoAAVViability v1.0 Assembly Efficiency Model,

Assist in the Construction of Library Screening and Candidate Variant Characterization

Based on BioMap's xTrimo, a life science foundational large model with hundreds of billions of parameters, and the 20 million AAV assembly efficiency data provided by Porton Biologics Ltd., BioMap has launched an industry-leadingAssembly Efficiency Prediction Model —— xTrimoAAVViability v1.0This model can help users quickly and accurately develop AAV vectors that meet specific application needs, effectively reducing R&D costs and time while enhancing the market competitiveness of gene therapy products.

This R&D achievement was showcased at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in 2024., has now been officially launched in the market, providing professional services to industry clients and research institutions.

In the model performance test, we used mutation data from the VR2 region of the AAV5 serotype for training. The results showed that the model achieved a Pearson correlation of 0.9 within the same serotype and mutation region, approaching the upper limit of biological reproducibility. Moreover, even in unseen mutation regions of the same training set AAV5 serotype or other serotypes not covered by the training set, the model still demonstrated excellent generalization capabilities, achieving Pearson correlations of 0.71 and 0.59, respectively. Additionally, BioMap's general protein naturality model xTrimoStability (https://aigp.biomap.com/best-practices/1), without exposure to any AAV data, reached a Pearson correlation of 0.53 in this predictive task, fully demonstrating the foundational generalization ability brought by large-scale protein language models, and providing an excellent basis for targeted fine-tuning training.

Through the precise prediction by the xTrimoAAVViability assembly efficiency model, and relying on Porton Biologics Ltd.'s extensive experience in AAV capsid library plasmid construction, a mature AAV virus packaging system, and various purification processes, stable and efficient delivery can be ensured. We canIn vivo (NHP, mice) or in vitro (3D culture, organoids, solid-phase screening) AAV capsid library screening is performed according to requirements, and candidate variants are obtained after several rounds of cycling.For the characterization of candidate variants, gene synthesis is performed based on the sequence, and plasmids of candidate variants are constructed. Viral packaging containing Barcode sequences is carried out using reporter genes or specified genes. Animal experiments or in vitro affinity assays are conducted using the virus, followed by NGS sequencing analysis to evaluate the targeting ability of the variants.

AIEmpower: Co-create a New Era of AAV Design

The success of this collaboration heralds a significant developmental phase in the field of AAV design. By integrating AI technology with biopharmaceutical practices, BioMap and Porton Biologics Ltd. have not only greatly enhanced the efficiency and success rate of AAV screening but also provided a powerful tool and solid support for research and applications in gene therapy and the biopharmaceutical industry.

Looking ahead, the two parties will continue to strengthen cooperation, leveraging the accumulation and training of vast amounts of high-quality data to further refine and optimize processes, rapidly transforming innovative concepts into feasible candidate variants. They will further expand and enhance AAV design services, providing the biopharmaceutical industry with more efficient and precise AAV solutions, jointly advancing innovation and development in gene therapy and biomedicine.

If you have any cooperation intentions or need further information about our services and products, please feel free to contact us. Contact information: marketingchina@portonadvanced.com

About BioMap

BioMap is a life science AI large model company with disruptive technology, founded by Robin Li and Wei Liu in 2020. The company is committed to combining cutting-edge AI and biotechnology, building the xTrimo platform, a foundational large model in life sciences centered on protein language, with hundreds of billions of parameters. It models biological evolution, decodes the laws of life, and brings generative capabilities to the frontier discovery issues of the life science industry. It has achieved a series of breakthrough results in innovative drug design, target discovery, de novo design of proteases, green technology, industrial innovation, consumption upgrade, and other fields, thereby contributing to human health and sustainable social development. For more information, please visit: www.biomap.com

About Porton Biologics Ltd.

Porton Biologics Ltd., established in December 2018 and located in Suzhou Industrial Park, relies on the listed company—Chongqing Porton Pharmaceutical Co., Ltd. (Stock Code: 300363). It has built CDMO platforms for plasmids, viral vectors, cell therapy, gene therapy, and nucleic acid therapy, providing end-to-end services from library construction, process and analytical method development, cGMP production to formulation filling, covering different stages of drug development to help customers accelerate the drug research and market launch process.

Porton Biologics Ltd. focuses on gene and cell therapy, building on the successful experience of Porton Pharma Solutions. With top international professionals at its core, adhering to the service principle of “Customer First,” and guided by the brand philosophy of “Compliance, Professionalism, Dedication, and Open Collaboration,” the company continues its parent company’s robust IP protection, project management mechanisms, and comprehensive quality management system. Leveraging the core team's successful experience in advancing gene and cell therapy products from clinical trials to market approval, it provides high-quality and efficient services to global customers, enabling innovative medicines to benefit the public sooner. For more information, please visit: www.portonbio.com