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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
Sources: ClinicalTrials.gov, Pharmsynthez

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

Related Research Articles

PubMedInternational journal of nanomedicine2026-09-20

Biotinylated ε-Polylysine-Cyclodextrin-Coated Mesoporous Silica Nanoparticles for Targeted pH-Responsive Baicalin Delivery.

Liao Rongqiang R, Ruan Yi Y, Zhang Ke K, Liu Maoxia M

Baicalin (BAI) is a natural flavonoid with antitumor potential, but its poor water solubility and low bioavailability limit clinical use. Mesoporous silica nanoparticles are promising drug carriers due to their large surface area and tunable pore structure, yet premature drug leakage remains a key challenge. A multifunctional nanoplatform, designated as BPCD@BAI@BMSN, was constructed to enable efficient baicalin loading, pH-responsive gated release, and biotin-mediated active tumor targeting. A novel biotin-ε-polylysine-cyclodextrin (BPCD) conjugate was synthesized via EDCI/NHS coupling and characterized by NMR and GPC. Hollow mesoporous silica nanoparticles (BMSN) were prepared by the Stöber method, surface-modified with benzothiazole. The BPCD conjugate was coated onto the nanoparticle surface via cyclodextrin-benzothiazole host-guest self-assembly. The resulting nanoparticles were characterized by TEM, DLS, zeta potential, FTIR, and TGA. Drug loading, pH-responsive release, cytotoxicity against SMMC-7721 cells, cellular uptake evaluated by confocal microscopy and flow cytometry, and in vivo antitumor efficacy in nude mice were systematically evaluated. BPCD@BAI@BMSN exhibited near-spherical morphology with a particle size of approximately 200 nm, a positive zeta potential of +10 mV, a drug loading of 19.85%, and an encapsulation efficiency of 89.6%. Cumulative baicalin release reached approximately 62% at pH 5.5, whereas only about 6% was released at pH 7.4, confirming acid-triggered gated release. The blank carrier showed no significant cytotoxicity with cell viability above 95%, while drug-loaded nanoparticles exhibited enhanced cytotoxicity at pH 6.8. Biotin-functionalized nanoparticles demonstrated significantly higher cellular uptake and tumor accumulation compared to non-targeted controls. In vivo, BPCD@BAI@BMSN achieved a tumor growth inhibition rate of 72.5% compared to the control group, with no obvious toxicity to major organs observed during the 21-day treatment period. BPCD@BAI@BMSN integrates high drug loading, pH-responsive supramolecular gating, and active tumor targeting into a single nanoplatform, offering a promising strategy for baicalin delivery with enhanced antitumor efficacy and favorable short-term biosafety.

PMID 42763689
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PubMedInternational journal of nanomedicine2026-09-20

Glutathione-Responsive Disulfiram Delivery from Thiol-Functionalized N-Doped Carbon Nanodots: An in vitro Proof-of-Concept Study.

Panaei Mohadese M, Mahani Mohamad M, Divsar Faten F, Khakbaz Faeze F

To develop and evaluate a glutathione (GSH)-responsive "Off-On" drug delivery system based on thiol-functionalized N-doped carbon dots (SNCDs) for controlled intracellular release of disulfiram (DSF). SNCDs were synthesized using a solvothermal technique and subsequently characterized using dynamic laser scattering, high-resolution electron imaging, UV-Vis spectrophotometry, and Fourier-transform infrared spectroscopy (FTIR). DSF was immobilized onto SNCDs through redox-cleavable disulfide linkages, and loading efficiency was determined by UV-Vis analysis. The in vitro drug release kinetics were examined under simulated physiological (pH 7.4) and tumor-relevant acidic (pH 5.4) conditions, evaluating the impact of glutathione (GSH) by performing experiments with and without its inclusion. Cytotoxicity and compatibility studies in MCF-7 breast tumor cells, evaluated via MTT test, were used to investigate SNCD biocompatibility and the anticancer performance of DSF-SNCDs. The FTIR spectra confirmed effective thiol functionalization and revealed oxygenated and nitrogenous groups on the particle surface suitable for disulfide linkage formation. DSF loading efficiency reached approximately 75%. Under non-reducing conditions, DSF-SNCDs exhibited minimal premature release (≤30% over 24 h at pH 7.4 and 5.4), demonstrating an "Off" state. In contrast, the presence of 5% (w/v) GSH at pH 5.4 triggered rapid DSF release, achieving approximately 80% cumulative release within 24 h, corresponding to the "On" state through reductive disulfide bond cleavage. Bare SNCDs showed high biocompatibility (>85% cell viability at 50 µg/mL), whereas DSF-SNCDs produced significantly enhanced cytotoxicity under GSH conditions, confirming redox-activated drug release. SNCDs provide an effective GSH-responsive nanoplatform for DSF delivery, combining high drug loading, excellent stability under non-reducing conditions, and efficient intracellular drug release under reductive environments. This strategy minimizes premature drug leakage while enhancing anticancer activity, demonstrating strong potential for cancer therapy.

PMID 42763685
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PubMedVeterinary research2026-09-20

Bovine viral diarrhea virus (BVDV) relies on cellular lipid droplet biogenesis and lipolysis to provide energy for viral replication.

Xiong Xiaoran X, Liu Yi Y, Zhu Yaohong Y, Wang Jiufeng J et al.

Bovine viral diarrhea virus (BVDV), one of the most important viral pathogens in cattle, causes serious economic losses due to immunosuppression and persistent infections. Previous studies have shown a tight connection between virus infection and lipid metabolism, particularly in the formation and degradation of lipid droplets (LDs). However, the pathogenic mechanism of BVDV infection and the molecular mechanisms by which viral proteins reprogram lipid metabolism remain unclear. We found that BVDV increased cellular LD accumulation by enhancing the production of new lipids, accelerating exogenous fatty acid uptake, and elevating diacylglycerol acyltransferase (DGAT)-dependent esterification of fatty acids (FAs). The generated LDs subsequently release free fatty acids (FFAs) via adipose triglyceride lipase/hormone-sensitive lipase (ATGL/HSL)-dependent lipolysis. This lipolysis-dependent release of FFAs is accelerated and transferred to mitochondria for oxidation by increasing contact between LDs and mitochondria, thereby promoting viral replication. Furthermore, BVDV core protein targets the surface of LDs, increasing and recruiting fatty acid synthase (FASN) and ATGL to promote LD mobilization. Meanwhile, the core protein interacts with mitochondria, linking mitochondria to LDs and facilitating the release of FAs for efficient fatty acid oxidation. Collectively, this study demonstrates that the BVDV core protein regulates cellular lipid metabolism to support BVDV replication, contributing to understanding the pathogenetic mechanisms by which BVDV interacts with host cells.

PMID 42763408
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PubMedCommunications biology2026-09-20

Beyond ER-Golgi trafficking: unconventional protein secretion as a new design frontier for synthetic secretion switches in mammalian cells.

Shi Bingshun B, Qiu Xinyuan X, Jiang Yongheng Y, Cai Jiabo J et al.

Mammalian gene switches enable programmable cell behavior. However, current switches on transcriptional and translational layers require de novo RNA and protein synthesis, and secreted outputs must additionally undergo folding, post-translational processing, and intracellular trafficking, imposing delays that limit rapid extracellular responses. Rapid secretion-control switches instead act on pre-synthesized proteins by controlling retention, trafficking, storage, or release. Most current platforms exploit the classical endoplasmic reticulum (ER)-Golgi pathway, including engineered stimulus-secretion coupling in specialized secretory cells and ER retention, retrieval-signal cleavage, or synchronized trafficking in general mammalian hosts. Although these strategies improve response kinetics, they remain limited by ER dependence, host-cell specificity, cargo compatibility, basal leakage, and post-release transport delays. Here, we review current secretion-control architectures and highlight unconventional protein secretion as an underexplored source of design principles for positioning regulatory control closer to terminal protein export and expanding the architectures available for mammalian secretion control.

PMID 42763312
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PubMedBMC pediatrics2026-09-20

Evaluating the therapeutic efficacy of a modified ketogenic diet in children with autism spectrum disorder: a randomized controlled trial.

Liu Le L, Zhao Qingsong Q, Zhou Jing J, Liu Zhi Z et al.

Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder marked by persistent impairments in social communication and interaction, together with restricted and repetitive behavior or interests. Recent epidemiological data indicate that the prevalence of ASD has continued to increase worldwide. Current behavioral and pharmacological interventions remain limited in efficacy, particularly for associated comorbidities. In this context, metabolic interventions, especially the ketogenic diet (KD), have attracted growing interest as a potential therapeutic strategy. A total of 62 young children with ASD admitted to Hefei Maternal and Child Health Hospital between January 2024 and January 2025 were prospectively enrolled and randomly assigned to a treatment group or a control group (31 per group). Both groups underwent hospital-based rehabilitation training on weekdays and home-based rehabilitation on weekends over a 2-month period. The treatment group additionally received a modified KD, comprising ketogenic nutritional powder and a structured dietary plan, with lunch provided at the hospital on training days while breakfast, dinner, weekend dietary implementation, and home monitoring were managed by caregivers. The control group followed a regular diet along with the same rehabilitation program. Changes in ASD-related symptoms were assessed using the Autism Behavior Checklist (ABC) and Childhood Autism Rating Scale (CARS). After two months of intervention, both groups showed significant reductions in ABC and CARS scores compared with baseline (P < 0.05). Between-group comparison of individual change scores showed larger reductions in the treatment group for both ABC (U = 324.000, P = 0.027) and CARS (U = 273.500, P = 0.003). In this cohort of young children with ASD, the modified ketogenic diet combined with rehabilitation training was associated with greater short-term reductions in ABC and CARS scores than rehabilitation alone. The intervention demonstrated acceptable short-term feasibility. The Project was registered on China Clinical Trial Registry (ChiCTR) with the identifier ChiCTR2300075057 ,Registered 24 August 2023, https://www.chictr.org.cn/ .

PMID 42763398
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PubMedJournal of perinatology : official journal of the California Perinatal Association2026-09-20

Definitions of neonatal Necrotizing Enterocolitis (NEC) in randomised controlled trials: a systematic review.

Ballantine Robert Seán RS, Campbell Ellen E, Croitoru Ovidiu O, Jackson Emma E et al.

Necrotizing Enterocolitis (NEC) is a significant cause of morbidity and mortality for preterm neonates. Although Bell's criteria are frequently used to assess stage of NEC, it was not a definition and, since its inception in 1978 and with the survival of neonates at earlier gestations, there is a requirement for an updated NEC definition. This systematic review aimed to evaluate the use of criteria for the diagnosis and staging of NEC in randomised clinical control trials. This systematic review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guidelines. A search was conducted through PubMed to identify Randomised Control Trials (RCTs) in the last twenty years that used NEC in the study title and in either the primary outcome or the inclusion criterion. The initial search identified fifty-six RCTs. Thirty-six RCTs were included for full-text analysis. Thirty-three trials used Bell's criteria or Modified Bell's criteria to define NEC and three RCTs unique, author-created definitions. There is consistent use of Bell's and Modified Bell's criteria of necrotizing enterocolitis in RCTs. However, international consensus on further modifications of the definition will greatly contribute to both research and clinical practice relating to NEC, in allowing greater consistency in the staging of NEC, and therefore optimal management.

PMID 42763344
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