The Programme for Manufacture of hPSC‑Based Products for AMD and PD.

Pluripotent stem cells (hPSCs) possess the unique ability to self-renew and differentiate into various functional cell types, positioning them as key "seed cells" in regenerative medicine. The development of PSC-based therapies offers new hope for treating complex diseases, and degenerative diseases, particularly those caused by the loss of specific cellular functions, such as macular degeneration, Parkinson's disease, spinal cord injury, diabetes, and cartilage damage. Cell replacement therapies using hPSC-committed cells can help halt disease progression or potentially cure these conditions. The Retinal Pigment Epithelium (RPE) is crucial for visual function and photoreceptor support, with its dysfunction implicated in age-related macular degeneration (AMD) and retinitis pigmentosa (RP). Parkinson's disease (PD), a rapidly progressing neurodegenerative disorder marked by the loss of midbrain dopaminergic neurons (mDA), remains one of the most difficult neurological conditions to treat. Traditional therapies for these diseases have largely been ineffective. RPE and mDA neurons differentiated from hPSCs offer promising solutions for treating AMD and PD, their treatment methods are both cell replacement therapy, and they are the products used in the first batch of stem cell clinical research registration projects in China, so we will put these two products together. This chapter summarizes the differentiation and quality control strategies for hESC-derived RPE and mDA neurons as examples of the potential hPSC-derived cell therapies, under development at the Chinese Academy of Sciences and Beijing Institute for Stem Cell and Regenerative Medicine, for these complex, treatment-resistant diseases.