Induced Photoreceptor Cells and Methods for Their Production

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This patent describes a method for efficiently generating induced photoreceptor cells, such as rods and cones, from starting cells like human induced pluripotent stem cells (iPSCs). The innovation centers on introducing certain transcription factors, especially GON4L (with or without OTX2 and NEUROD1), to reprogram the initial cells into photoreceptor cells. The resulting induced photoreceptors can be produced quickly and in high purity, making them suitable for therapeutic applications such as cell transplantation to treat retinal degenerative diseases, as well as for research and drug testing purposes.

Use CasesContent extracted from patent full text and abstract with AI.

• Cell therapy for treating retinal degenerative diseases, including age-related macular degeneration and retinitis pigmentosa.
• Personalized medicine, using patient-specific iPSC-derived photoreceptors for autologous transplantation.
• Development of cell lines for research into retinal biology and disease mechanisms.
• High-throughput drug screening and toxicity testing targeting photoreceptor function or survival.
• Disease modeling using patient-derived iPSCs to understand genetic retinopathies and retinal degeneration.
• Basic neuroscience studies into the development and function of photoreceptor cells.

BenefitsContent extracted from patent full text and abstract with AI.

• Rapid generation of human photoreceptor cells (within 10 days) compared to traditional methods.
• High purity and homogeneity of the induced photoreceptor cell populations, enabling reliable downstream use.
• Protocol adaptable to various initial cell types, especially expandable sources like iPSCs and fibroblasts.
• Ability to generate large quantities of photoreceptors from a small starting population, making clinical applications feasible.
• Potential for personalized cell therapies using patient-derived cells, reducing risks of immune rejection.
• Scalable production process suitable for research, clinical, and pharmaceutical applications.

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Inventors & Applicants

Patent Abstract

The invention relates to a method for producing induced photoreceptor cells from an initial cell, the method comprising providing one or more transcription factors (TFs) comprising at least GON4L to the initial cell. In preferred embodiments of the invention, the initial cell is a human induced pluripotent stem cell (i PSC). In other embodiments the method comprises providing the TFs OTX2 and/or NEUROD1 to the initial cell. The invention further relates to the cells produced and obtainable by the method of the invention, the use of these cells as a medicament in the treatment of retinopathy, vectors for inducing the photoreceptor cells of the present invention and combinations of transcription factors intended for this use.