Beijing BYONGEN Technology Co., Ltd

R&D Overview

Powered by the DynEvoLib^® discovery platform, Byongen Therapeutics has built a comprehensive R&D ecosystem integrating technical services, asset licensing, and innovative pipeline development. We are committed to overcoming gene delivery bottlenecks to provide safer, more precise therapies for patients worldwide.

Discovery Services: Leveraging proprietary PEARS and CALIBRATOR technologies, we offer customized services from high-quality AAV library preparation to multi-level, single-animal validation.

Capsid Licensing: We offer global licensing for our MyoCAP^® (muscle-tropic) and SynCAP™ (CNS-tropic) variants, both rigorously validated in non-human primate (NHP) models.

Therapeutic Pipeline: We accelerate the clinical translation of genetic medicines for cardiovascular, CNS, and neuromuscular diseases through independent R&D and strategic partnerships.

Discovery Services
Capsid Licensing
R&D pipeline

We provide comprehensive AAV library preparation services, including random AAV capsid peptide display libraries, surface-informed AAV capsid peptide display libraries, and internally calibrated barcoded libraries, alongside our specialized capsid screening and characterization services. We offer high-standard foundational support for the precision screening of high-performance capsids and vector development. We welcome the opportunity to discuss your specific custom requirements.

AAV Library Preparation

Defining excellence from the source to build libraries with superior evolutionary potential.
By integrating the rational design of PEARS with the precision calibration of CALIBRATOR, we systematically solve industry challenges such as low effective variant ratios and poor evaluation accuracy.
Core Technologies
PEARS (Peptide-Driven AAV Directed Evolution): Combines the diversity of random peptide libraries with surface-informed peptide libraries from tens of thousands of cell-surface interacting proteins to build variant libraries with high biological activity.
CALIBRATOR (Calibration System): Utilizes multi-gradient internal controls and co-purification processes to establish tissue-specific standard curves for the quantitative assessment of transduction efficiency.
Service Advantages
Enrichment of High-Function Sequences: Unlike traditional random libraries, PEARS incorporates cell-surface interaction data at the design phase, significantly reducing inactive sequences and boosting screening efficiency.
Elimination of Packaging & Detection Bias: Our normalization algorithms effectively bypass abundance variations during preparation, ensuring fair and authentic performance evaluation within complex tissue environments.
Maximum Diversity Guarantee: We provide expansive candidate libraries covering a vast sequence space, supporting multi-target and multi-species screening needs from the very start of R&D.

Capsid Screening

Enhanced Sensitivity for Low-Abundance Signals
Utilizing the CAPTURE module alongside PEARS and CALIBRATOR to enable high-sensitivity, parallel screening across multiple tissues.
Core Technology
CAPTURE (Automated High-Throughput Signal Enrichment): Leverages automated sequence-specific capture to maximize signal extraction efficiency. It supports simultaneous multi-tissue detection and identification of low-abundance signals, enabling high-efficiency, precision AAV variant screening.
Service Advantages
Superior Sensitivity: Effectively extracts and identifies sparse signals from complex tissue samples.
Automated High-Throughput: Supports batch parallel processing of gram-level tissue samples, significantly shortening screening timelines.

Capsid Characterization

Single-Animal Multi-Level Validation: Comprehensive Evaluation from DNA to Protein
Leveraging the CAMEL module, we achieve high-throughput validation of AAV variants through integrated, multi-level testing within a single animal.
Core Technology
CAMEL (Multiplexed Epitope Tag Evaluation): Each variant is encoded with a unique Epitope Tag and a DNA Barcode, enabling multiplexed, simultaneous validation of numerous candidates within a single subject.
Service Advantages
Multi-Level Parallel Data: Simultaneously capture comprehensive data across the entire delivery process—from vector DNA internalization and mRNA transcription to protein expression—all within a single animal.
Breaking Throughput Bottlenecks: Move beyond the inefficient "one sequence per animal" model. This significantly reduces the number of experimental animals required and shortens validation timelines.
Eliminating Individual Variability: Conduct comparative analysis of multiple candidate variants within the same biological background, resulting in more authentic and persuasive data.
Universal Applicability: Particularly suited for the in-depth evaluation of rare and precious animal models, such as Non-Human Primates (NHP).

Developed via the DynEvoLib^® platform, our engineered AAV capsids feature precisely refined tissue tropism. Compared to traditional serotypes like AAV9, these vectors offer significant technical advantages: lower effective dosages, reduced off-target toxicity, and an expanded therapeutic index. These improvements directly enhance the clinical translation potential of gene therapy pipelines.We offer the following lead vector assets rigorously validated in non-human primate (NHP) models. We look forward to exploring licensing and customized collaboration opportunities to empower your research.

MyoCAP^® (Muscle-tropic)

Application Scenarios

Specifically developed for gene therapies targeting neuromuscular diseases, cardiomyopathies, and the cardiovascular system. It provides an alternative solution for R&D pipelines currently constrained by the delivery efficiency, liver toxicity risks, and effective dosage limits of traditional AAV9.

Technical Features

Enhanced Tissue Tropism: Significantly improves transduction efficiency in skeletal muscle in Non-Human Primate (NHP) models.

Liver De-targeting: Optimized via systematic design to reduce uptake in non-target tissues. In NHP models, the muscle-to-liver expression ratio is increased by 10–20 fold compared to AAV9.

Administration & Efficacy: Supports systemic intravenous (IV) injection. By enhancing transduction efficiency in target tissues, it enables "dose sparing," which helps broaden the therapeutic window.

CMC Manufacturability: Production titers are comparable to AAV9, making it compatible with mainstream large-scale manufacturing processes.

SynCAP^™(CNS-tropic)

Application Scenarios

Designed for neurodegenerative diseases (such as Ataxia and ALS) and various CNS gene therapy projects. It is ideal for developing therapies that require broad distribution across the central nervous system (CNS) and high-efficiency transduction of specific cell lineages.

Core Advantages

Clinical-Grade Administration Route: Optimized for intrathecal (IT) injection, featuring superior cerebrospinal fluid (CSF) diffusion capabilities to achieve effective coverage across the entire CNS.

Robust Expression Efficiency: Achieved an average 30-fold increase in CNS mRNA expression levels compared to AAV9, significantly broadening the therapeutic window.

Deep Tissue Penetration: Highly efficient penetration into the spinal cord, cerebellum, and deep brain regions, overcoming the delivery barriers that traditional vectors face in deep-seated tissues.

Superior Cell Lineage Coverage: Strong neuronal tropism enables high-ratio coverage of cerebellar Purkinje cells, while also ensuring highly efficient infection of glial cells.

Non-Human Primate (NHP) Validation: Preliminary validation of core variants has been completed in NHP models. Their transduction profiles provide critical benchmarks for predicting clinical dosages in the future.

Byongen is dedicated to developing innovative gene therapies based on engineered AAV vectors, with a primary focus on areas of high unmet medical need, including hereditary neuromuscular diseases, cardiomyopathies, and Central Nervous System (CNS) degenerative diseases. Leveraging our capsid development platform validated in Non-Human Primate (NHP) models, we advance the clinical translation of our pipeline assets through both independent R&D and collaborative development.
Byongen actively seeks global partners for collaboration in areas such as target development, clinical translation, and capsid licensing. By providing high-performance engineered vector technology, we are committed to enhancing the delivery efficiency and commercial value of our partners' R&D pipelines.

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