Sumitomo Pharma Co., Ltd. has secured approval from the U.S. Food and Drug Administration to initiate a Phase 1/2 clinical trial. This study will explore the efficacy of an allogeneic induced pluripotent stem (iPS) cell-derived retinal sheet, known as DSP-3077, in treating retinitis pigmentosa, a leading cause of inherited blindness.
Clinical Trial Details and Collaboration
The Investigational New Drug (IND) application, submitted on October 25, 2024, successfully passed the FDA’s 30-day review period. Sumitomo Pharma is finalizing preparations to commence the study, which plans to utilize fresh, non-frozen three-dimensional retinal tissues. Collaborations with Massachusetts Eye and Ear in Boston aim to begin patient transplants within the fiscal year 2025. Additionally, a prospective observational study (NCT06517940) is underway to identify optimal ophthalmic endpoints, enhancing both the current and future clinical developments.
Preceding Research and Technological Foundation
Prior to this initiative, Sumitomo Pharma conducted pioneering transplants at Kobe City Eye Hospital starting in 2020, marking the first global use of allogeneic iPS cell-derived retinal sheets in patients. These early trials confirmed the safety and engraftment of the retinal sheets over a two-year period. The underlying technology leverages the SFEBq self-organizing cell culture technique, initially developed by Dr. Yoshiki Sasai’s team at RIKEN, and further refined through Sumitomo’s collaboration with RIKEN from 2010 to 2014. This foundation has enabled the establishment of a robust manufacturing process for generating retinal sheets.
- Expansion of treatment options for a diverse genetic background of RP patients.
- Potential reduction in progression to severe visual impairment.
- Advancement in stem cell therapy applications beyond ophthalmology.
The clinical trial is meticulously designed as an unmasked, single-arm, dose-escalation study involving 12 participants. Primary objectives focus on assessing the safety and tolerability of DSP-3077, while secondary goals include evaluating engraftment success, immune responses, and overall efficacy in halting or reversing retinal degeneration.
By spearheading this clinical study, Sumitomo Pharma aims to offer a groundbreaking therapeutic option for individuals suffering from retinitis pigmentosa. The company’s extensive research and development efforts underscore its commitment to addressing significant unmet medical needs in the realm of genetic retinal diseases.
Advancements in iPS cell technology not only pave the way for innovative treatments for retinitis pigmentosa but also hold promise for a wide range of degenerative conditions. Continuous collaboration with leading research institutions and rigorous clinical evaluation are essential steps toward realizing the full potential of stem cell-based therapies. Patients and healthcare providers alike stand to benefit from the progress made in this field, anticipating improved quality of life and preservation of vision.
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