Revolutionizing Eye Disease Treatment with iPSC Therapy

The Cell & Gene Therapy Research & Development Congress Asia 2023 kicked off in Taipei, Taiwan today, experts in the field of cell and gene therapy gathered here to discuss the emerging advances in the field of cell and gene therapy, the regenerative potential of stem cells, the application of T-cells and CAR-T in the field of immuno-oncology, and more.

iPSC Therapeutics for Eyes Diseases

Xianmin Zeng, Professor at the University of Singapore and President & CEO of RxCell Inc. gave the talk about iPSC therapy treating retinal degenerative diseases. 

Two retinal diseases that lead to blindness are caused by loss of cells: age-related macular degeneration (AMD) and Retinitis pigmentosa (RP). For eye disorders, they are ideal indications for cell therapy regarding eyes’ accessibility, small and confined organ characteristics. 

To develop universal iPSC therapy, RxCell has combined Knock-out and Knock-in gene techniques to generate hypoimmunogenic iPSC cell lines. Mouse model and non-human primate studies show C-Rx-001 presents an effective way to avoid immune rejection. Using autologous cell transplantation and HLA haplobank approach, the single source cell platform possesses the ability to evade immune detection and cure retinal degenerative diseases.

Related Article: Cell & Gene Therapy Congress Asia Day One: Advances in the Research

Advancing Manufacturing Process of iPSC-derived Products

With clinical applications progressing, Zeng shared that RxCell has established a current Good Manufacture Practice (cGMP) iPSC master cell bank for clinical use. Besides, a GMP-compliant manufacturing process for RPC manufacture has been established. 

The cGMP-grade human iPSC-derived retinal photoreceptor precursor cells show no adverse effects in naïve NHP models. In addition, photoreceptor precursors injected into retinal degeneration NHP models demonstrated an ability both to survive and to mature into cone photoreceptors at 3 months post-transplant. 

Enabling Next-Generation Cell Therapies with Deep Tech

Peter Peumans, CTO of IMEC shared the application of deep tech to the healthcare field. As a world-leading R&D and innovation hub in nanoelectronics and digital technologies, IMEC connects digital technology to life science technology, including cell assays, computational microscopy, smart neuroprobes, and next-gen omics.

Peumans noted this new toolset will allow for high-throughput cell selection and editing based on cell surface repertoire or functional assays with single cell precision. For example, IMEC has developed a compact, disposable cell sorter. It enables the sorting of blood cells in minutes, reporting the result to point-of-care and early cancer diagnostics applications. 

Good Data Integrity in Cell & Gene Therapy Products

Following up, Anthony Colenburg Sr, Director & Site Head of Quality from Sutro Biopharma addressed the quality & regulatory expectations of cell & gene therapy products.

In Colenburg’s talk, he pointed out the importance of data integrity, since it affects product consistency, safety as well as efficacy. 

Colenburg also gave a brief introduction on good data integrity and documentation practice standard. Good Data Integrity and Documentation Practices describe standards by which records are created and maintained. It is required by the U.S. FDA, EMA, as well as other worldwide regulatory guidelines. Not only does it ensure the product safety, identity, strength, quality, and Purity/Potency (SISQP), it also provides evidence to the regulatory agencies that procedures were followed, and the products are being manufactured as claimed. Colenburg emphasized that it’s important to establish confidence in the quality of manufacturing, testing and final products at every stage/phase.

Author

Kathy Huang

We are, and will remain, perpetual newbies. - Kevin Kelly