THE 30STARTUPS OF KANSAI

Rege Nephro is a clinical-stage biotechnology company developing innovative treatments for kidney diseases. Our lead programs are RN-014, a small-molecule drug candidate for autosomal dominant polycystic kidney disease (ADPKD) that has completed a Phase 2 clinical trial in Japan, and RN-032, an iPS cell-derived kidney progenitor cell therapy candidate being developed for chronic kidney disease (CKD). Building on our expertise in nephrology and proprietary iPS cell technologies, we are also expanding our drug discovery and regenerative medicine activities to address other kidney diseases with significant unmet medical needs.

Kidney disease can progressively impair patients’ health, independence, and quality of life, while dialysis creates substantial physical, emotional, and financial burdens for patients, families, caregivers, and healthcare systems. ADPKD, the most common monogenic kidney disease, causes progressive kidney cyst growth and can ultimately lead to kidney failure requiring dialysis or transplantation. In Japan alone, approximately 337,000 patients receive chronic dialysis, with annual dialysis-related medical expenditure estimated at about US$9.8 billion. Our pipeline candidates are being developed to slow disease progression, preserve kidney function, and prevent or delay dialysis, helping patients remain healthier and independent for longer.

Our core technologies originate from research conducted at the Center for iPS Cell Research and Application (CiRA), Kyoto University. We have established proprietary methods to efficiently generate kidney progenitor cells from iPS cells and create kidney disease models for drug discovery. This platform supports a distinctive dual approach: developing regenerative cell therapies and identifying small-molecule drug candidates, including RN-014. Combined with our deep expertise in kidney biology and expanding intellectual property portfolio, this integrated approach gives us a strong competitive advantage in creating disease-modifying treatments that may delay or reduce patients’ dependence on dialysis.

Our near-term priority is to advance RN-014 into global Phase 3 development and toward commercialization, working with a pharmaceutical partner for markets outside Japan to leverage partner’s strong global clinical, regulatory, and commercial capabilities. We also intend to accelerate RN-032 toward clinical development and expand our iPS cell-based drug discovery platform across additional kidney diseases. By combining our scientific and development capabilities with strategic partnerships and support from leading international investors, we aim to build a sustainable global biotechnology company and deliver transformative treatment options to patients with serious unmet medical needs.