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疾病领域	数量

内分泌与代谢疾病	3

排名前五的药物类型	数量

腺相关病毒基因治疗	3

排名前五的靶点	数量

mTOR(丝氨酸/苏氨酸蛋白激酶mTOR)	2

关联

3

项与 CdmoGen Co., Ltd. 相关的药物

rAAV2-shmTOR-GFP (CuroGene Life Sciences/University of Ulsan)

靶点

mTOR

作用机制

mTOR抑制剂

在研机构

University of Ulsan [+1]

原研机构

CuroGene Life Sciences Co., Ltd.

在研适应症

糖尿病性视网膜病变 [+1]

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期-

rAAV2-sVEGFRv-1 (CuroGene Life Sciences/University of Ulsan)

靶点-

作用机制

基因转移

在研机构

University of Ulsan [+1]

原研机构

University of Ulsan

在研适应症

脉络膜新生血管 [+1]

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期-

US20240218372

专利挖掘

靶点

mTOR

作用机制-

在研机构

CdmoGen Co., Ltd.

原研机构

CdmoGen Co., Ltd.

在研适应症

营养和代谢疾病

非在研适应症-

最高研发阶段药物发现

首次获批国家/地区-

首次获批日期-

100 项与 CdmoGen Co., Ltd. 相关的临床结果

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0 项与 CdmoGen Co., Ltd. 相关的专利（医药）

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9

项与 CdmoGen Co., Ltd. 相关的文献（医药）

2022-10-13·Advances in Cell and Gene Therapy

Intravitreally Administered Soluble VEGF Receptor-1 Variant Tested as a Potential Gene Therapeutic for Diabetic Retinopathy

作者: Choi, Jun-Sub ; Cha, Seho ; Kim, Hee Jong ; Woo, Ha-Na ; Kang, Im Kyeung ; Lee, Steven Hyun Seung ; Lee, Heuiran ; Park, Keerang ; Kim, Jin ; Lee, Joo Yong ; Lee, Kyoung Jin

In addition to laser photocoagulation, currently used therapeutic interventions for diabetic retinopathy (DR) include relatively short-lived anti-VEGF drugs targeting vascular endothelial growth factor (VEGF). The latter requires frequent administration via intravitreal injections to effect long-term VEGF suppression. However, due to the patient burden associated with this treatment modality, gene therapy may represent a preferable alternative, providing long-lasting yet patient-friendly effects. Here, we explore the therapeutic efficacy of rAAV2-sVEGFRv-1, a recombinant adeno-associated virus encoding a soluble variant of VEGF receptor-1, upon early DR processes. Bevacizumab, an anti-VEGF agent often prescribed off label to treat DR, was used as an experimental comparator. Administered by intravitreal injection to a streptozotocin-induced diabetic mouse model, rAAV2-sVEGFRv-1 was shown to effectively transduce the mouse retinas and express its transgene therein, leading to significant reductions in pericyte loss and retinal cell layer thinning, two processes that play major roles in DR progression. Acellular capillary formation, vascular permeability, and apoptotic activity, the latter being the cell death mechanism by which retinal neurodegeneration occurs, were also shown to be reduced by the therapeutic virus vector. Immunohistochemistry was used to visualize that rAAV2-sVEGFRv-1 has an effect on cell types important to DR pathophysiology, particularly the ganglion cell layer and glial cells. Combined with our previous work showing that the therapeutic virus vector reduces neovascularization, our current results reveal that rAAV2-sVEGFRv-1 addresses the early aspects of DR as well, thereby demonstrating its potential as a human gene therapeutic versus the condition as a whole.

2020-02-27·Investigative Opthalmology & Visual Science2区 · 医学

Inhibition of mTOR via an AAV-Delivered shRNA Tested in a Rat OIR Model as a Potential Antiangiogenic Gene Therapy

2区 · 医学

ArticleOA

作者: Lee, Joo Yong ; Park, Keerang ; Lee, Steven Hyun Seung ; Woo, Ha-Na ; Kim, Ji Hyun ; Kim, Hee Jong ; Chung, Sunho ; Chang, HeeSoon ; Choi, Jun-Sub ; Lee, Heuiran ; Lee, Kyoung Jin

PurposeRecent studies have shown that inhibitors of the mechanistic target of rapamycin (mTOR) play important roles in proliferating endothelial cells within the retinal vasculature. Here we explore the effects of inhibiting mTOR as a potential gene therapeutic against pathological retinal angiogenesis in a rat model of oxygen-induced retinopathy (OIR).MethodsSprague-Dawley pups were used to generate the OIR model, with a recombinant adeno-associated virus expressing an shRNA (rAAV2-shmTOR-GFP) being administered via intravitreal injection on returning the rats to normoxia, with appropriate controls. Immunohistochemistry and TUNEL assays, as well as fluorescein angiography, were performed on transverse retinal sections and flat mounts, respectively, to determine the in vivo effects of mTOR inhibition.ResultsCompared with normal control rats, as well as OIR model animals that were either untreated (20.95 ± 6.85), mock-treated (14.50 ± 2.47), or injected with a control short hairpin RNA (shRNA)-containing virus vector (16.64 ± 4.92), rAAV2-shmTOR-GFP (4.28 ± 2.86, P = 0.00103) treatment resulted in dramatically reduced neovascularization as a percentage of total retinal area. These results mirrored quantifications of retinal avascular area and vessel tortuosity, with rAAV2-shmTOR-GFP exhibiting significantly greater therapeutic efficacy than the other treatments. The virus vector was additionally shown to reduce inflammatory cell infiltration into retinal tissue and possess antiapoptotic properties, both these processes having been implicated in the pathophysiology of angiogenic retinal disorders.ConclusionsTaken together, these results demonstrate the strong promise of rAAV2-shmTOR-GFP as an effective and convenient gene therapy for the treatment of neovascular retinal diseases.

2019-09-01·Molecular Therapy - Methods & Clinical Development2区 · 医学

Effects of Stuffer DNA on the Suppression of Choroidal Neovascularization by a rAAV Expressing a mTOR-Inhibiting shRNA

2区 · 医学

ArticleOA

作者: Kim, Ji Hyun ; Choi, Jun-Sub ; Lee, Steven Hyun Seung ; Jung, Sang Joon ; Woo, Ha-Na ; Lee, Joo Yong ; Park, Keerang ; Chang, HeeSoon ; Kim, Jin ; Nah, Seung Kwan ; Lee, Heuiran ; Park, Tae Kwann ; Kim, Hee Jong

Choroidal neovascularization (CNV) is the defining characteristic of the wet subtype of age-related macular degeneration (AMD), which is a rapidly growing global health problem. Previously, we had demonstrated the therapeutic potential of gene therapy against CNV using short hairpin RNA (shRNA) delivered via recombinant adeno-associated virus (rAAV), which abrogates mammalian-to-mechanistic (mTOR) activity in a novel manner by simultaneously inhibiting both mTOR complexes. Both the target and use of gene therapy represent a novel treatment modality against AMD. Here, the xenogeneic GFP gene used as a reporter in previous studies was removed from the virus vector to further develop the therapeutic for clinical trials. Instead, a stuffer DNA derived from the 3' UTR of the human UBE3A gene was used to ensure optimal viral genome size for efficient rAAV assembly. The virus vector containing the stuffer DNA, rAAV2-shmTOR-SD, positively compares to one encoding the shRNA and a GFP expression cassette in terms of reducing CNV in a laser-induced mouse model, as determined by fundus fluorescein angiography. These results were confirmed via immunohistochemistry using anti-CD31, while a TUNEL assay showed that rAAV2-shmTOR-SD possesses anti-apoptotic properties as well. The qualities exhibited by rAAV2-shmTOR-SD demonstrate its potential as a human gene therapeutic for the treatment of wet AMD.

100 项与 CdmoGen Co., Ltd. 相关的药物交易

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100 项与 CdmoGen Co., Ltd. 相关的转化医学

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当前项目

药物(靶点)	适应症	全球最高研发状态

rAAV2-sVEGFRv-1 (CuroGene Life Sciences/University of Ulsan)	糖尿病性视网膜病变更多	临床前
rAAV2-shmTOR-GFP (CuroGene Life Sciences/University of Ulsan) ( mTOR )	糖尿病性视网膜病变更多	临床前
US20240218372 ( mTOR )	营养和代谢疾病更多	药物发现