P2, N=50, Active, not recruiting, Advenchen Pharmaceuticals, LLC. | N=30 --> 50 | Recruiting --> Active, not recruiting

After receiving targeted combination immunotherapy with sequential PD-1 inhibitors (toripalimab) plus anti-angiogenic agents (sunitinib, axitinib)-a regimen that enhances anti-tumor immunity but may disrupt pulmonary immune homeostasis-the patient gradually developed progressive dyspnea, chest tightness, hypoxemia, and anuria. Although PJP complicated by hydropneumothorax after immunotherapy is rare, it should be considered as a possible etiology when cancer patients develop progressive dyspnea with difficulty maintaining oxygen saturation after receiving immune checkpoint inhibitor-based therapy, particularly in the context of immune checkpoint inhibitor use. While biomarkers for predicting immunotherapy efficacy and irAEs are well-studied, the identification of specific biomarkers for predicting opportunistic infections like PJP in this context remains an area of active research.

P=N/A, N=500, Active, not recruiting, West China Hospital

Here, we evaluated whether normalizing tumor vasculature via chronic neoadjuvant VEGFR2 inhibition (aVEGFR2, DC101) improves subsequent FUS-mediated small molecule drug delivery...Our results indicate that neoadjuvant aVEGFR2 cooperates with FUS-mediated small molecule drug delivery through a unique biophysical mechanism. This mechanism may be leveraged to further augment the efficacy of combination therapies against GBM that entail blocking VEGF signaling.

IHC confirmed a decrease in microvessel density post-treatment and indicated larger hypoxic areas in treated tumors compared to controls at the experimental endpoint. The newly introduced approach thus facilitates experimental studies aiming at optimization of antiangiogenic treatment regimens and their subsequent combination with other treatment modalities, such as radiation therapy, where effectiveness may strongly depend on the vascular network condition and tumor oxygenation levels.

P1, N=12, Completed, Exegenesis Bio | Active, not recruiting --> Completed

P1, N=12, Active, not recruiting, Exegenesis Bio | Enrolling by invitation --> Active, not recruiting

P3, N=406, Recruiting, Bio-Thera Solutions

P2, N=20, Recruiting, M.D. Anderson Cancer Center | Trial primary completion date: Apr 2026 --> Dec 2027

P2, N=88, Not yet recruiting, Korea University Anam Hospital

Early clinical candidates, such as EZN-3042 and ALN-VSP, achieved target engagement and biological activity, while limitations included variable tumour uptake, dose-limiting toxicities, and complex pharmacokinetic behaviour. Translation to clinical practice will depend on optimised delivery platforms, reproducible pharmacokinetic/pharmacodynamic synchronisation, and rigorous evaluation of safety and off-target effects. Overall, current evidence highlights substantial potential for siRNA-drug co-delivery while emphasizing key challenges to overcome in achieving durable clinically meaningful outcomes.

P1, N=19, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Mar 2027