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calcifediol (Replidea / JTT 762 / CTAP 101)

✓ Approved

Pharmsynthez · VDR · Small Molecule

What is calcifediol?

calcifediol is a small molecule developed by Pharmsynthez. It is approved for therapeutic indications via injectable (others) or intravenous (iv) or oral (po).

Drug Profile

Brand NamesReplidea, JTT 762, CTAP 101
CompanyPharmsynthez
Drug ClassSmall Molecule
Molecular TargetVDR
RouteInjectable (Others), Intravenous (IV), Oral (PO)
StatusApproved

Mechanism of Action

Molecular Targets

calcifediol acts on 1 molecular target:

VDRvitamin D receptor (NR1I1, PPP1R163)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

calcifediol is developed for 5 unique indications across 5 therapeutic areas.

Therapeutic AreaConditionPhase
Endocrine disordersHyperparathyroidism secondary✓ Approved
Metabolism and nutrition disordersVitamin D deficiency✓ Approved
Renal and urinary disordersEnd stage renal disease✓ Approved
Infections and infestationsCOVID-19Phase II
Immune system disordersCytokine release syndromePreclinical

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Mitochondria-Targeted Nanozyme Reprograms Dendritic-Cell Immunometabolism via Microenvironment-Responsive CO Release to Treat Periodontitis.

Shan Shangyan S, Liu Chengyu C, Ding Lina L, Zeng Weishu W et al.

Inflammatory diseases are characterized by overactivated immune responses and a disrupted metabolic equilibrium, particularly in dendritic cells (DCs), where mitochondrial reactive oxygen species (mtROS) burst and metabolic reprogramming drives pathological maturation. While modulating immunometabolism is a promising therapeutic avenue, achieving subcellular-targeted delivery of bioactive molecules remains a formidable challenge. Here, we report a mitochondria-targeted nanozyme designed to concurrently scavenge mtROS and reprogram DC metabolism for effective anti-inflammatory therapy in periodontitis. This system is constructed based on Prussian blue nanoparticles (PB NPs) loaded with manganese carbonyl, a high oxidative stress-responsive carbon monoxide (CO) donor, and further modified with triphenylphosphine for mitochondrial targeting. This nanozyme efficiently accumulates in the mitochondria of activated DCs, where it efficiently scavenges mtROS and concurrently delivers controlled CO release, synergistically modulating DC function. Metabolomics analysis reveals that CO suppresses DC maturation by reprogramming cellular metabolism, including inhibiting the tricarboxylic acid cycle, modulating glycolysis, and disrupting fatty acid synthesis. Consequently, the synergistic action of PB NPs and CO effectively reverses the pro-inflammatory phenotype of DCs, reshapes the immune microenvironment, and ultimately alleviates periodontal inflammation in vivo. This work presents a promising strategy for curing inflammatory diseases by targeting metabolic reprogramming at the subcellular level.

PubMedFrontiers in neuroscience2026-09-19

20 Hz tACS engages striatal THINs to enhance β-band synchrony and motor drive.

Zang Honggang H, Xie Wanxia W, Tian Lei L, Kang Wanrong W et al.

Transcranial alternating current stimulation (tACS) is a noninvasive neuromodulation technique that can enhance motor function. The striatum is a central hub for motor control and β-band oscillations, and its tyrosine hydroxylase-positive interneurons (THINs) are modulated by dopamine. However, whether THINs are associated with motor changes following tACS through β-band synchrony has not been investigated. Eighteen-month-old male mice received 20 Hz tACS or sham stimulation over M1 for 7 days under sevoflurane anesthesia. Motor performance, EEG, striatal dopamine release, and THIN activation were assessed at 3 min after awakening on day 7. THINs were optogenetically manipulated during the reaching task under the same post-anesthesia testing window. Transcranial alternating current stimulation was associated with higher post-anesthesia reaching success, increased β-band power, elevated dopamine release, and enhanced THIN activation compared to sham. Optogenetic THIN activation recapitulated motor improvement and β-band enhancement. Optogenetic THIN inhibition reduced successful attempts per minute and β-band power, but no statistically significant reduction in success rate was detected. These findings suggest that 20 Hz tACS over M1 may improve post-anesthesia motor performance in aged mice, with converging evidence implicating striatal dopamine release, THIN engagement, and β-band synchrony as functionally coupled elements in the context of post-anesthesia motor recovery in aged mice.

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Soft bioelectronics for wireless drug delivery: programmable, minimally invasive and closed-loop therapeutics.

Zhang Feng F, Sun Fuchang F, Yan Zheng Z

Soft bioelectronics is reshaping drug delivery from passive dosing to programmable, tissue-interfaced therapeutics. This Review examines the co-design of soft materials, wireless power, drug reservoirs, release mechanisms and feedback control for wearable and minimally invasive implantable therapeutic systems. We organize the field around key constraints including controllability, reversibility, payload capacity, dosing precision, drug stability and feedback latency, guiding the development of adaptive closed-loop drug delivery platforms for clinical translation.

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Higher early radiographic bone healing scores with popliteus muscle preservation during tibial plateau levelling osteotomy in dogs.

Sassaroli S S, Salvaggio A A, Roggiolani F F, Fordellone M M et al.

To compare early radiographic bone healing 45 days after tibial plateau levelling osteotomy performed with popliteus muscle detachment versus a modified technique preserving the muscle and to determine whether muscle preservation influences post-operative osteotomy stability through measurement of rock-back. In this prospective, blinded, randomised clinical study (2022 to 2025), 100 client-owned dogs with unilateral cranial cruciate ligament rupture were allocated to traditional surgery (n = 50) or popliteus-sparing surgery (n = 50). Radiographic assessment was performed preoperatively, immediately post-operatively and 45 days after surgery. Bone healing was evaluated using two validated radiographic scoring systems. Osteotomy stability was assessed by measuring the change in tibial plateau angle between the immediate post-operative and 45-day radiographs. Multivariable linear regression models were used to adjust for baseline clinical variables. A P-value <.05 was considered statistically significant. Dogs undergoing popliteus-sparing surgery demonstrated higher bone healing scores at 45 days, with an adjusted mean difference of 1.95 (95% confidence interval 0.91 to 2.99) on the 10-point bone healing scale and 1.87 (95% confidence interval 0.88 to 2.86) on the modified radiographic union scale. There was no clinically relevant difference in post-operative tibial plateau angle change between groups, with an adjusted mean difference of 0.26 degrees (95% confidence interval -0.10 to 0.63). No major intraoperative vascular complications were recorded. Tibial plateau levelling osteotomy performed without detachment of the popliteus muscle represents a safe and effective alternative to the traditional technique. Popliteus muscle preservation during tibial plateau levelling osteotomy was associated with higher radiographic bone healing scores at 45 days, without evidence of increased osteotomy instability. Whether this radiographic difference translates into improved clinical recovery or limb function remains unknown.

PubMedThe AAPS journal2026-09-19

Quantitative Characterization of Innate and Adaptive Pharmacology of Allogeneic anti-CD20 Chimeric Antigen Receptor (CAR) Vδ1 γδ T cells using Multiscale Mechanistic Modeling.

Desai Devam A DA, Elashkar Omar O, Cristofoletti Rodrigo R, Mugundu Ganesh G et al.

Gamma Delta (γδ) T Cells are currently being evaluated as a therapeutic alternative to traditional alpha-beta (αβ) T-cells due to their superior safety profile and enhanced tissue retention properties. The application of CAR technology to gamma delta (γδ) T cells presents a novel therapeutic avenue with the potential to overcome some limitations of conventional CAR T-cell therapies, such as targeting solid tumors and reducing on-target, off-tumor toxicities. The objective of this manuscript is development of a translational PK-PD framework to first characterize in vitro killing potential of un-transduced and CAR transduced anti- CD20 Vδ1 γδ T cells as well as development of an in vivo mechanistic CK-PD model designed to understand the complex dynamics of CAR γδ T cells and their interaction with IL-15 and tumor cells. All the preclinical and clinical datasets along with relevant information were digitized and obtained from the published work on Adicet Bio's AD-001. The developed model was able to estimate the in vitro killing potential of untransduced and CAR transduced anti- CD20 Vδ1 γδ T cells as well as expansion, tissue distribution, the impact of lymphodepletion and interleukin-15 (IL-15), and the tumor-killing potential of CAR-modified γδ T cells. These insights offer a deeper understanding of the potential therapeutic benefits and mechanisms of γδ T cells in immunotherapy, particularly in their application against various cancers. The development of this translational framework can be paramount in understanding the underlying dose-exposure-response relationship of CAR modified γδ T cell therapy and facilitate the discovery and development of these agents.

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RhoA in postnatal spinal motoneuron is essential for peripheral myelination.

He Ye Y, Xu Shuyi S, Zou Ying Y, Zhao Mengyao M et al.

Peripheral myelination requires precise axon-glia communication, yet the neuronal intrinsic machinery that governs the release of axonal signals remains incompletely understood. Here, we discover that RhoA, a classic cytoskeletal regulator, is highly expressed in postnatal spinal motoneurons and unexpectedly governs this axon-glia communication. RhoA conditional knockout in postnatal motoneurons causes profound peripheral hypomyelination without affecting neuronal survival, dendrites, or axonal caliber. Mechanistically, RhoA deficiency in postnatal spinal motoneurons attenuates ROCK2/p-Erk/SP1/BACE1 signaling and NRG1-Ⅲ secretion, then disrupts Schwann cells differentiation, lipid biosynthesis, and myelin formation. Together, this study reveals a novel, non-cell-autonomous role for RhoA and provides further insights into the complexity of neuronal control over peripheral myelination.

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