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更新于：2026-02-02
ADX-038
更新于：2026-02-02
概要
基本信息
非在研机构- |
权益机构- |
最高研发阶段临床2期 |
首次获批日期- |
最高研发阶段(中国)临床申请批准 |
特殊审评- |
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关联
3
项与 ADX-038 相关的临床试验NCT06990269
A Phase 2, Randomized, Masked, Placebo-Controlled Study of Subcutaneously Administered ADX-038 in Participants With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
NCT06989359
A Phase 2 Study to Assess ADX-038 in Participants With Complement-Mediated Kidney Disease
NCT05876312
A Phase 1, Randomized, Double Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Participants Followed by a Phase 2a Open Label Study in Participants With PNH and Residual Anemia to Evaluate the Safety, Tolerability, PK and PD of ADX-038
100 项与 ADX-038 相关的临床结果
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100 项与 ADX-038 相关的转化医学
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100 项与 ADX-038 相关的专利（医药）
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41
项与 ADX-038 相关的文献（医药）2026-01-01JOURNAL OF CONTROLLED RELEASE
Hypoxia-responsive modules via tunable hydrophobicity reversal enhance renal-targeted release of CD36-interfering nanoparticles to ameliorate acute kidney injury
Article
作者: Liao, Xiaohui ; Wei, Li ; Liu, Chen ; Su, Yu ; Chen, Yaxi ; Ruan, Xiong Z ; Zhao, Lulu ; Wen, Haobo ; Hu, Ke ; Zhao, Yunfei ; Zou, Gang ; Wang, Ping ; Zhang, Tao
RNA interference (RNAi) has led to multiple FDA-approved therapeutics and boasts a robust clinical pipeline. However, its transformation for kidney disease remains hindered by the absence of effective delivery vehicles, which are constrained by low delivery efficiency and non-target organ release. Herein, six novel modules of 2-(2-nitro-1H-imidazol-1-yl)ethanamine (IEN) with different carbon-chain linkers were designed and synthesized, then subsequently grafted onto chitosan (CS-Linker-IEN) to enable efficient delivery of RNAi in renal tissues. The CS-Linker-IEN self-assembles with siRNA into spherical nanoparticles (CS-Linker-IEN/siRNA) primarily through electrostatic, hydrogen-bond, and tunable hydrophobic interactions. This assembly enables the screening of an optimal size driven by tunable hydrophobicity and achieves efficient delivery to renal proximal tubule epithelial cells, with the smallest-sized CS-Hex-IEN/siRNA nanoparticles exhibiting the highest renal accumulation. Under hypoxic conditions characteristic of specific kidney diseases, the IEN moiety undergoes a structural transformation into 2-(2-amino-1H-imidazol-1-yl)ethanamine (AIE). This transformation reverses the hydrophobic-hydrophilic interactions, thereby enabling kidney-specific siRNA release. In a murine model of IRI-induced acute kidney injury (AKI), CS-Linker-IEN demonstrated targeted release of siCD36 to renal tubular epithelial cells, effectively mediating CD36 interference. This intervention conferred renoprotective effects through restoration of lipid metabolism, attenuation of oxidative stress, and suppression of the NF-κB signaling pathway. Given a plethora of kidney diseases that could benefit from RNAi, it is anticipated that this finding will expand the preclinical landscape of renal therapeutics.
2025-06-01JOURNAL OF CONTROLLED RELEASE
Hybrid micellar preparations for co-delivery of PARP-1 siRNA and quercetin for cataract treatment
Article
作者: He, Yao ; Zou, Zhilin ; Yalamarty, Satya Siva Kishan ; Li, Xiang ; Zhang, Jing ; Torchilin, Vladimir P ; Filipczak, Nina
Cataract remains a major cause of ocular blindness. Cyclic Arg-Gly-Asp-d-Phe-Lys (RGD) peptide was introduced to the surface of self-assembled hybrid micelles for the co-delivery of poly (ADP-ribose) polymerase 1 (PARP-1) small-interfering RNA (siRNA) and quercetin (Q/siP-c-M). Q/siP-c-M exhibited uniform particle size distribution, good dispersibility, high encapsulation efficiency, and strong stability for siRNA and quercetin. Q/siP-c-M significantly improved the transcorneal co-delivery of siRNA and quercetin to the deeper cornea and led to greater drug accumulation. In addition, Q/siP-c-M significantly increased the activity of catalase and the content of adenosine triphosphate (ATP), reduced the expression of PARP-1 protein, and effectively prevented lipid peroxidation in the lens. Among selenite-induced cataract rats, the Q/siP-c-M-treated rats produced higher levels of ATP and catalase, as well as lower levels of malondialdehyde and PARP-1 protein expression compared with those in the model group. Administration of quercetin further resulted in a decrease in neutrophil extracellular trap formation and downregulation of gene expression of related proteins and pro-inflammatory cytokines. These observations indicated that quercetin has the potential to serve as a therapeutic for alleviating an excessive inflammatory reaction characterized by an overabundance of neutrophil extracellular traps in the eyes. Therefore, this study highlights the potential of Q/siP-c-M against cataract development through the regulation of the immune response by regulating inflammatory conditions. Furthermore, Q/siP-c-M may offer benefits in terms of apoptosis attenuation for lens epithelial cells.
2024-09-01BIOMEDICINE & PHARMACOTHERAPY
Carboxylated mesoporous silica nanoparticle-nucleic acid chimera conjugate-assisted delivery of siRNA and doxorubicin effectively treat drug-resistant bladder cancer.
Article
作者: Shang, Zhenhua ; Chen, Wen ; Wang, Xu ; Wang, Qi ; Ou, Tongwen ; Liu, Biao ; Yang, Jintao ; Yan, Hao ; Li, Guangping
Chemotherapy is the main treatment for bladder cancer, but drug resistance and side effects limit its application and therapeutic effect. Herein, we constructed doxorubicin (DOX)/COOH-mesoporous silica nanoparticle/polyethylenimine (PEI)/nucleic acid chimeras (DOX/MSN/Chimeras) to reduce the toxicity of chemotherapy drugs and the resistance of bladder cancer cells. Transmission electron microscopy showed that PEI was coated on the DOX/MSN/BSA nanoparticles with a diameter of about 150 nm. DOX/MSN/PEI could control DOX release for over 48 h, and the sudden release rate was significantly lower than DOX/MSN. Immunohistochemical results showed that DOX/MSN/Chimera specifically bound to bladder cancer cells, and markedly inhibited PI3K expression and proliferation of DOX-resistant bladder cancer cells. DOX/MSN/Chimera promoted the apoptosis of drug-resistant bladder cancer cells, which was superior to DOX/MSN/Aptamer or DOX/MSN. We further carried out animal experiments and found that DOX/MSN/Chimera could reduce the volume of transplanted tumors in vivo. Compared with DOX/MSN/Aptamer group, the proliferation rate was significantly decreased and the proportion of apoptotic cells was highly increased. Through the histological observation of kidneys and lungs, we believed that DOX/MSN/Chimera can effectively reduce the damage of chemotherapy drugs to normal tissues. In conclusion, we constructed a COOH-MSN/nucleic acid chimera conjugate for the targeted delivery of siRNA and anti-cancer drugs. Our study provides a new method for personalized and targeted treatment of drug-resistant bladder cancer.
100 项与 ADX-038 相关的药物交易
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 年龄相关性黄斑变性 | 临床2期 | 美国 | 2025-11-10 | |
| 年龄相关性黄斑变性 | 临床2期 | 澳大利亚 | 2025-11-10 | |
| 年龄相关性黄斑变性 | 临床2期 | 加拿大 | 2025-11-10 | |
| 地图样萎缩 | 临床2期 | 美国 | 2025-11-10 | |
| 地图样萎缩 | 临床2期 | 澳大利亚 | 2025-11-10 | |
| 地图样萎缩 | 临床2期 | 加拿大 | 2025-11-10 | |
| 补体因子H缺乏症 | 临床2期 | 美国 | 2025-08-28 | |
| 补体因子H缺乏症 | 临床2期 | 澳大利亚 | 2025-08-28 | |
| 补体因子H缺乏症 | 临床2期 | 中国香港 | 2025-08-28 | |
| 补体因子H缺乏症 | 临床2期 | 韩国 | 2025-08-28 |
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临床结果
临床结果
适应症
分期
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临床1期 | 41 | 醖鬱鑰願積廠選鏇艱獵(蓋齋願蓋蓋遞餘顧艱簾) = The most common AE was upper respiratory tract infection, similar to placebo. There were no encapsulated bacterial infections nor clinically relevant changes in clinical lab tests, PE or vital signs. 鏇憲鬱艱繭顧範遞選夢 (醖餘窪艱餘願鏇範遞淵 ) | 积极 | 2025-11-07 | |||
placebo | |||||||
临床1期 | 15 | 齋顧積窪獵糧淵艱糧蓋(醖鑰鑰齋餘廠醖餘簾鬱) = Most common treatment-related AEs: fatigue (47%), fever/chills (33%), allergic (33%), constipation (33%), nausea/vomiting (20%), all g1-2. 1 pt had g3 anemia and 2 pts had g2 thrombocytopenia, all shortly after CALAA-01 infusions with rapid recovery. 1 pt had possibly-related sinus bradycardia (g2) 醖構構繭鑰顧構獵壓襯 (憲繭願蓋顧壓糧獵糧鬱 ) 更多 | - | 2010-05-20 |
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