September 08, 2026

Accelerating the Path to IND: Cyagen Empowers Global Biopharma with Integrated Preclinical CRO Solutions

From custom disease modeling to IND-enabling pharmacology, our integrated CRO platforms have successfully driven breakthroughs in gene therapy, cell therapy, and rare diseases toward clinical validation.

Guangzhou, China, September 8, 2026 - Cyagen Biosciences has accelerated the preclinical development and clinical translation of multiple innovative therapeutic programs across ophthalmology, metabolic and cardiovascular diseases, hematological disorders, cell therapy, and rare diseases. Through integrated CRO platforms and customized disease models, Cyagen provides research support spanning disease model development, animal studies, pharmacology and efficacy evaluation, and IND-enabling research.

With capabilities covering target validation, disease model development, in vivo efficacy evaluation, and regulatory submission support, Cyagen partners with biopharmaceutical companies to address key challenges in preclinical development and facilitate the advancement of innovative therapies toward clinical development.

Ophthalmic Gene Therapy

Cyagen's in vivo expertise has directly accelerated multiple gene therapies targeting retinal diseases into the clinic.

IVB102 (XLRS): For InnoVecBio's investigational X-linked retinoschisis therapy, Cyagen delivered longitudinal efficacy evaluations and ocular administration. IVB102 became the first XLRS gene therapy developed in China to receive U.S. FDA IND clearance and Rare Pediatric Disease Designation (RPDD).
IVB103 (nAMD): Cyagen engineered the mouse choroidal neovascularization (CNV) model and executed efficacy evaluations for this neovascular age-related macular degeneration program. IVB103 secured both U.S. FDA IND and China CDE clinical authorization in 2024.

Metabolic and Cardiovascular Therapies

For HRS-5346, an oral small-molecule inhibitor of lipoprotein(a) [Lp(a)] developed by Hengrui Pharmaceuticals, Cyagen provided end-to-end preclinical support, from the construction and breeding of Lp(a) conditional knock-in mouse models to pharmacology and efficacy evaluation.

This seamless IND-enabling support helped advance the program toward a monumental exclusive licensing agreement with Merck, featuring a $200 million upfront payment and potential milestones exceeding $1.77 billion.

Hematology Programs

For SNH-119014, an investigational small-molecule therapy developed by Sino-Hub Pharmaceutical (Chengdu), Cyagen conducted thalassemia mouse model development and in vivo pharmacology and efficacy evaluation, enabling the program through preclinical research and IND submission. The program subsequently received approval from China's National Medical Products Administration (NMPA) to initiate clinical trials.

Cyagen's hematology research capabilities include customized disease model development and pharmacological validation, enabling partners to conduct preclinical studies tailored to specific therapeutic programs.

Cell Therapy

Cyagen provided animal models and integrated CRO services to advance the development of ZLT-001, a second-generation tumor-infiltrating lymphocyte (TIL) therapy developed by Guangzhou Zhiling Biopharmaceutical Co., Ltd.

The collaboration focused on animal model development to evaluate TIL tumor infiltration and related preclinical studies, contributing to ZLT-001's advancement to NMPA clinical trial approval in 2023 for the treatment of advanced recurrent or metastatic cervical cancer.

Ultra-Rare Diseases

Cyagen contributed to the preclinical development of a gene therapy program for GM2 gangliosidosis, AB variant, an ultra-rare genetic disorder, through disease model development and efficacy validation.

Cyagen's preclinical research, including AAV-mediated administration, safety assessment, and target protein expression analysis, helped advance the program to the first reported clinical administration of gene therapy for GM2 gangliosidosis AB worldwide in 2024.

An Integrated CRO Platform for Translational Drug Development

Across these programs, Cyagen combines specialized disease models, gene-editing technologies, pharmacology and efficacy evaluation, and therapeutic-area-specific CRO capabilities to accelerate drug development from early research through IND-enabling studies.

Spanning ophthalmology, metabolic and cardiovascular diseases, hematology, cell therapy, gene therapy, oncology, and antibody drug development, Cyagen integrates customized animal models with standardized preclinical workflows to deliver tailored solutions and help translate promising discoveries into clinical-stage programs.

About Cyagen Biosciences

Founded in 2006, Cyagen Biosciences is an AI-enabled biotechnology company dedicated to accelerating biomedical innovation through proprietary gene-editing technologies, advanced disease models, computational platforms, and integrated preclinical research solutions.

Cyagen develops and applies cutting-edge technologies in gene editing, humanized models, AAV engineering, and AI-driven biological research to support global drug discovery and translational medicine programs. The company's research capabilities span multiple therapeutic areas, including ophthalmology, oncology, neuroscience, metabolic diseases, autoimmune disorders, and other fields.

With advanced research platforms and global collaboration networks, Cyagen is committed to empowering researchers and biopharmaceutical companies with innovative solutions that bridge scientific discovery and therapeutic development.