The United States Food and Drug Administration (FDA) granted Beijing InnoVec Biotherapeutics’ InnoVex IVB102 injection the Rare Pediatric Disease Designation (RPDD) on January 10, 2024. Innovec is now one step closer to being eligible for the Rare Pediatric Disease Priority Review Voucher (PRV) for the treatment of rare pediatric diseases with the InnoVex IVB102 injection in the United States.

As a partner of Innovec in the drug development, Cyagen extends our heartfelt congratulations on this wonderful news and we look forward to Innovec's continued progress and success in the field of new drug development. We are honored to have been part of the drug development process for IVB102 injection, providing services such as mouse breeding, ocular injection dosing, and pharmacodynamic monitoring, which have significantly contributed to the clinical translation of gene therapy for X-linked retinoschisis (XLRS).

InnoVex IVB102: Injectable XLRS Gene Therapy Drug

InnoVex IVB102 is a gene therapy drug designed to treat X-linked retinoschisis (XLRS) and is reportedly based on a novel carrier independently-developed by InnoVec. Preclinical data indicates that animal models treated with IVB102 can have their visual electrophysiological signals restored to levels comparable to those of wild-type animals, suggesting that the drug holds 'best in class' potential.

The Rare Pediatric Disease Designation (RPDD) is granted by the FDA for drug applications intended for pediatric rare diseases where the patient population in the United States is fewer than 200,000 individuals under the age of 18. If the biologics license application (BLA) for IVB102 injection is approved by the FDA, Innovec will become eligible to receive a Priority Review Voucher (PRV) for the InnoVex IVB102 injectable gene therapy drug. PRVs are highly valuable, as they can be used to expedite the review process for any subsequent product marketing applications and can also be sold to third parties, with recent transfer prices reaching as high as $350 million.

Ophthalmic Gene Therapy CRO Service Platform

Drawing upon our accumulated data and extensive experience in bioinformatics and gene editing projects, Cyagen has established a public platform for Gene Therapy Drug Screening Technical Services. This platform not only caters to general preclinical gene therapy research and development, but also offers specific technical services in various disease areas such as ophthalmology, neurology, and oncology. Building on our gene therapy drug screening capabilities, Cyagen has developed a comprehensive set of gene therapy solutions for ophthalmic diseases. Our ophthalmic gene therapy CRO platform hosts a team of technical experts skilled in ophthalmic precision operations, internationally renowned precision small animal ophthalmic instruments and equipment, and a standardized service system for all preclinical ophthalmic gene therapy research. This allows us to provide clients with one-stop solutions for ophthalmic gene therapy research, including the construction of ophthalmic disease models, ocular injection dosing, in vivo ocular phenotype analysis, and molecular pathology phenotypic drug efficacy testing.

AI-Assisted AAV Vector Development

  • Building an AI model to efficiently screen AAV mutants for multi-target gene therapy drugs, achieving predictions for various site-specific targeting such as brain (blood–brain barrier permeating), whole-eye transduction capability, and retinal penetration.

Variety of Ophthalmic Disease Models

  • Our current mouse resource repository includes models for ophthalmic diseases such as LCA2, LCA10, retinal pigment degeneration, retinal vascular proliferation, and corneal endothelial malnutrition.

  • We also offer customization or collaborative development of gene-edited mouse models, including gene knockouts, gene knock-ins, point mutations, humanized mouse models, as well as surgical disease models in both mice and rats.

Specialized and Efficient Ophthalmic Pharmacology Analysis Platform

  • Ocular Injection Dosing: Subretinal, intravitreal, subconjunctival, anterior chamber, etc.

  • Ocular Phenotypic Testing: Fundus fluorescein angiography (FFA), anterior segment photography, optical coherence tomography (OCT), electroretinogram (ERG), flash visual evoked potentials (F-VEP), laser photocoagulation, visual water maze, light-dark box, OptoTrack small animal visual stimulus dynamic tracking, etc.

  • Molecular Pathology Phenotypic Testing:
  1. Isolatable Tissues: Cornea, lens, retina, RPE (Retinal Pigment Epithelium), choroid + sclera, optic nerve.
  2. Testing Methods: Western Blot, qPCR (quantitative polymerase chain reaction), enzyme-linked immunosorbent assay (ELISA), retinal/choroidal flat-mount, retinal sectioning, hematoxylin and eosin (H&E) staining, immunohistochemistry, immunofluorescence, and more.


About InnoVec Bio

Established in 2021, Beijing InnoVec Biotherapeutics Inc. (InnoVec Bio) is a company dedicated to gene editing delivery technology and drug development. Guided by the philosophy of "In China For Global," the company focuses on developing precise, safe, and efficient tissue-targeted vectors. Its mission is to address the challenge of gene delivery to all tissues and organs in the human body, enabling the widespread application of gene therapy across various diseases and populations.

In the field of gene therapy delivery technology, InnoVec Bio has developed various AAV vectors for intraocular injection, extensively validated in both small and large animals. Building on these vectors, the company is advancing clinical research. Additionally, carriers targeting organs such as the liver, heart, nerves, and muscles are also in the development phase. To overcome the toxicity associated with viral vector immunogenicity and the challenge of repeat injections, InnoVec Bio is working on developing fully human-derived viral delivery vectors, using human proteins to compose nucleic acid delivery carriers, theoretically minimizing or eliminating immunogenicity issues related to delivery vectors.

Building on the self-developed intraocular injection vectors, InnoVec Bio is actively advancing product development. This includes genetic mutations causing hereditary retinal diseases such as X-linked retinoschisis (XLRS), complete color blindness (CNGA3), retinol dehydrogenase 12 (RDH12), juvenile macular dystrophy (ABCA4), and common retinal diseases like retinal detachment and age-related macular degeneration. Additionally, InnoVec Bio is actively exploring gene therapy for diseases beyond the field of ophthalmology, including Alzheimer's disease, neurodegenerative diseases, and muscular atrophy.

Innovec is located in Area A of Haixinyu Pharmaceutical Industrial Park, Haidian District, with 3,000 square meters of R&D and office space. It has been officially recognized as a high-tech enterprise in Zhongguancun, categorized as a technology-based small and medium-sized enterprise (SME), and an innovative SME. In the "Chuangke Beijing" entrepreneurship and innovation competition, INNOVEC achieved third place in the innovation category, and in the fourth edition of the "Entrepreneurship Beijing" entrepreneurship and innovation competition, it also received a third-place award in the innovation category. Furthermore, the company has secured support funds for high-quality development from the Zhongguancun National Independent Innovation Demonstration Zone and has undertaken various municipal-level innovation projects under its leadership.

About Cyagen

Founded in 2006, Cyagen is an innovative Contract Research Organization (CRO) that accelerates drug discovery through data, algorithms, and modeling. Leveraging animal models and incorporating artificial intelligence, Cyagen is at the forefront of the industry in the field of gene-edited animal models. They offer comprehensive, intelligent solutions, including animal model resource libraries, customized models, breeding and husbandry, germ-free mouse technology services, and phenotype functional validation, catering to the full spectrum of customer needs in animal models for basic research and drug development.

Simultaneously, Cyagen continuously enriches its product offerings, strengthens its data and modeling capabilities, and proactively invests in the fields of gene therapy and cell therapy. By combining target prediction and validation platforms, virus vector development platforms, evaluation model construction platforms, and effectiveness assessment platforms, the company is poised for further growth in the rapidly evolving landscape of the industry.

Cyagen currently has over 900 employees and a total footprint of more than 40,000 square meters. The company has wholly-owned subsidiaries in Guangzhou, Suzhou, and Gu'an, as well as offices in Chengdu, Beijing, Shanghai, Wuhan, Changsha, and subsidiary companies in Santa Clara, USA, and Tokyo, Japan. Cyagen has established extensive collaborations with scientists, research institutions, and businesses in over 100 countries and regions worldwide. Its products and technologies have been directly applied in more than 10,000 academic papers, including prominent journals like CNS (Cell, Nature, Science).

Driven by the vision of creating endless possibilities in biotechnology, Cyagen is committed to partnering with pharmaceutical researchers worldwide to shape a brighter future together!