First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Patients With Retinitis Pigmentosa (RP)

hRPC is a cell therapy for retinitis pigmentosa. This is a first-in-human, dose escalation study in which participants with retinitis pigmentosa will receive a single subretinal injection of hRPC cells in one eye to evaluate safety and tolerability.
Participants will be followed for two years to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC.

开始日期2015-12-01

申办/合作机构

ReNeuron Ltd.

100 项与 Human retinal progenitor cells (ReNeuron Ltd.) 相关的临床结果

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100 项与 Human retinal progenitor cells (ReNeuron Ltd.) 相关的转化医学

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100 项与 Human retinal progenitor cells (ReNeuron Ltd.) 相关的专利（医药）

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项与 Human retinal progenitor cells (ReNeuron Ltd.) 相关的文献（医药）

2019-10-01·Biomaterials1区 · 工程技术

RBC membrane camouflaged prussian blue nanoparticles for gamabutolin loading and combined chemo/photothermal therapy of breast cancer

1区 · 工程技术

Article

作者: Jian, Yuqing ; Liu, Bin ; Xie, Qian ; Long, Ying ; Fan, Jialong ; Tong, Chunyi ; Wang, Wenmiao ; Daniyal, Muhammad ; Xiao, Feng ; Ma, Xiaochi ; Li, Bin ; Wang, Wei

Due to the non-targeted release of anti-cancer agent gamabufotalin (CS-6), conventional chemotherapy using this drug can cause serious side effects, which accordingly result in poor therapeutic efficiency. Recently, the development of smart nanodrug systems has attracted more and more attention due to their significant advantages of high loading efficiency, controllable release behavior and targeted accumulation at tumor sites. In this study, a nanodrug system named as HA@RBC@PB@CS-6 NPs (HRPC) was constructed. In this system, Prussian blue nanoparticles (PB NPs) with hollow porous structure were used as the carrier for CS-6 and photothermal sensitizer simultaneously. The result indicated that the encapsulation of erythrocyte membrane on the PB NPs prolonged the blood circulation life to 10 h and improved the immune evasion ability for more than 60%, as well, which is beneficial for the targeting molecule (HA) to achieve high concentration accumulation of HRPCs at tumor sites. Moreover, we also disclosed that loading drug of CS-6 performed its ultra-strong anti-tumor function partly through markedly suppressing the expression of HSP70, which conversely amplified the efficiency of photothermal therapy. The in vivo study demonstrated the outstanding performance of HRPC in synergistic photothermal/chemotherapy of cancer without side effect to normal tissues.

2016-07-14·Translational Vision Science & Technology3区 · 医学

Efficacy and Safety of Human Retinal Progenitor Cells

3区 · 医学

ArticleOA

作者: Vugler, Anthony ; Semo, Ma'ayan ; Brooke, Gary ; Carter, David ; Young, Michael ; Sinden, John ; Stevanato, Lara ; Patel, Sara ; Coffey, Peter ; Haamedi, Nasrin

PURPOSEWe assessed the long-term efficacy and safety of human retinal progenitor cells (hRPC) using established rodent models.METHODSEfficacy of hRPC was tested initially in Royal College of Surgeons (RCS) dystrophic rats immunosuppressed with cyclosporine/dexamethasone. Due to adverse effects of dexamethasone, this drug was omitted from a subsequent dose-ranging study, where different hRPC doses were tested for their ability to preserve visual function (measured by optokinetic head tracking) and retinal structure in RCS rats at 3 to 6 months after grafting. Safety of hRPC was assessed by subretinal transplantation into wild type (WT) rats and NIH-III nude mice, with analysis at 3 to 6 and 9 months after grafting, respectively.RESULTSThe optimal dose of hRPC for preserving visual function/retinal structure in dystrophic rats was 50,000 to 100,000 cells. Human retinal progenitor cells integrated/survived in dystrophic and WT rat retina up to 6 months after grafting and expressed nestin, vimentin, GFAP, and βIII tubulin. Vision and retinal structure remained normal in WT rats injected with hRPC and there was no evidence of tumors. A comparison between dexamethasone-treated and untreated dystrophic rats at 3 months after grafting revealed an unexpected reduction in the baseline visual acuity of dexamethasone-treated animals.CONCLUSIONSHuman retinal progenitor cells appear safe and efficacious in the preclinical models used here.TRANSLATIONAL RELEVANCEHuman retinal progenitor cells could be deployed during early stages of retinal degeneration or in regions of intact retina, without adverse effects on visual function. The ability of dexamethasone to reduce baseline visual acuity in RCS dystrophic rats has important implications for the interpretation of preclinical and clinical cell transplant studies.

2014-03-01·Journal of Biological Chemistry2区 · 生物学

Human Retinal Progenitor Cell Transplantation Preserves Vision

2区 · 生物学

Article

作者: Hong Ouyang ; John Sinden ; Nicole Sfeir ; Peter Shaw ; John Quach ; Petr Baranov ; Kang Zhang ; Austin Qiu ; Jing Luo ; Sherrina Patel ; Jessica Lu ; Cindy Wen ; Victoria Reade ; Caroline Hicks ; Sara Patel ; Hongrong Luo ; Meixia Zhang ; Jing Zeng ; Frances Wu ; Michael Young ; Xiaodong Sun ; Jing Zhu

Cell transplantation is a potential therapeutic strategy for retinal degenerative diseases involving the loss of photoreceptors. However, it faces challenges to clinical translation due to safety concerns and a limited supply of cells. Human retinal progenitor cells (hRPCs) from fetal neural retina are expandable in vitro and maintain an undifferentiated state. This study aimed to investigate the therapeutic potential of hRPCs transplanted into a Royal College of Surgeons (RCS) rat model of retinal degeneration. At 12 weeks, optokinetic response showed that hRPC-grafted eyes had significantly superior visual acuity compared with vehicle-treated eyes. Histological evaluation of outer nuclear layer (ONL) characteristics such as ONL thickness, spread distance, and cell count demonstrated a significantly greater preservation of the ONL in hRPC-treated eyes compared with both vehicle-treated and control eyes. The transplanted hRPCs arrested visual decline over time in the RCS rat and rescued retinal morphology, demonstrating their potential as a therapy for retinal diseases. We suggest that the preservation of visual acuity was likely achieved through host photoreceptor rescue. We found that hRPC transplantation into the subretinal space of RCS rats was well tolerated, with no adverse effects such as tumor formation noted at 12 weeks after treatment.

100 项与 Human retinal progenitor cells (ReNeuron Ltd.) 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
视网膜色素变性	临床2期	英国	ReNeuron Ltd.	2015-12-01
视网膜色素变性	临床2期	西班牙	ReNeuron Ltd.	2015-12-01
视网膜色素变性	临床2期	美国	ReNeuron Ltd.	2015-12-01