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HSV-tk gene therapy (HSVtk / Zalmoxis / TK therapy)

✓ Approved

Megapharm Ltd · POLA1 · 细胞治疗

什么是 HSV-tk gene therapy？

HSV-tk gene therapy 是一种细胞治疗，由Megapharm Ltd研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名HSVtk, Zalmoxis, TK therapy
公司Megapharm Ltd
药物类别细胞治疗, 基因治疗
分子靶点POLA1
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, Megapharm Ltd

作用机制

分子靶点

HSV-tk gene therapy 作用于 1 个分子靶点：

POLA1DNA polymerase alpha 1, catalytic subunit (PDR, POLA)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

HSV-tk gene therapy 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Immune system disordersChronic graft versus host disease✓ Approved

相关研究文献

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Helicase-primase inhibitors as next-generation anti-HSV agents: from current clinical status to structure-activity relationships and patent coverage.

Gege Christian C, Kleymann Gerald G

Helicase-primase is an interesting target for small-molecule therapy of orthoherpesviruses in general and in particular herpes simplex virus (HSV) and varicella zoster virus (VZV) infections. With amenamevir already approved for the treatment of VZV and HSV in Japan and with pritelivir's granted breakthrough therapy designation and priority review for the treatment of acyclovir-resistant HSV infections in immunocompromised patients, other companies have also expressed interest in small-molecule helicase-primase inhibitors (HPIs). We review the helicase-primase inhibitor landscape across HSV and VZV, focusing on patent coverage, key structure-activity relationships and the clinical progress of leading candidates. Following the clinical validation of the helicase-primase target by amenamevir and pritelivir, substantial patent activity has emerged around structurally diverse scaffolds aimed at enhancing target residence time, improving resistance profiles, enabling prolonged antiviral exposure and optimizing tissue distribution, including nerve penetration. In parallel, the availability of several cryo-electron microscopy structures of the helicase-primase complex has enabled structure-based and computer-aided drug design, facilitating the exploration of alternative scaffolds beyond the established chemotypes. The potential opportunities and limitations of the current HPIs, as well as the different dosage forms, are discussed.

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PubMedJournal of drug targeting2026-07-27

Surface-Engineered Nanocarrier-Based Drug Delivery Systems for the Management of Herpes Simplex Virus.

Goswami Sahil S, Victor Janis J, Nath Anushka A, Mukherjee Anupam A et al.

Herpes simplex virus (HSV) infections are commonly treated with antiviral drugs; however, many of these therapeutics exhibit limited oral bioavailability. Consequently, high and frequent dosing is often required to maintain therapeutic plasma concentrations, which may increase the risk of adverse effects. In recent years, nanotechnology-based drug delivery systems have emerged as promising strategies to overcome these limitations by improving drug stability, bioavailability, and targeted distribution. A wide range of nanocarrier platforms, including nanoparticles (NPs), nanocomposites, nanodroplets, nanohybrids, solid lipid nanoparticles (SLNs), silver nanoparticles (AgNPs), and mesoporous silica nanoparticles (MSNs), have been investigated for HSV therapy. These nanocarriers enable controlled drug delivery and enhanced cellular uptake, while surface-engineering approaches further improve site-specific targeting, therapeutic efficacy, and cellular internalisation. This review provides a comprehensive overview of recent advances in nanotechnology-driven drug delivery strategies for HSV treatment, with particular emphasis on surface-engineered nanocarriers. A systematic literature search was conducted across major scientific databases and search engines, including Google Scholar, ScienceDirect, PubMed, and Embase, using keywords such as "nanoparticles", "blood-brain barrier", "nanocarriers", "surface modification", and "nanocarriers for HSV". We discuss current surface modification strategies applied to antiviral-loaded nanocarriers and highlight their potential to enhance bioavailability, improve targeting precision, and ultimately improve therapeutic outcomes in HSV management.

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PubMedZhonghua yu fang yi xue za zhi [Chinese journal of preventive medicine]2026-07-27

[Advances in research on the molecular mechanisms and gene therapy of hereditary hearing impairment].

Yang X P XP, Gao Y Q YQ, Huang L L, Zhao L J LJ et al.

Hereditary hearing impairment represents a significant etiology of language and social dysfunction in both children and adults, primarily caused by genetic factors. To date, over 150 genes have been identified in association with this disorder. The pathogenic mechanisms involve multiple molecular levels, including abnormalities in hair cell cytoskeleton and stereociliary structure, dysfunction of intercellular gap junctions (e.g., GJB2, GJB6), dysregulation of ion channels and transporters (e.g., SLC26A4, KCNQ4), alterations in extracellular matrix composition, and disruption of intracellular signaling pathways. In recent years, research has expanded to investigate the role of the inner ear immune microenvironment in this condition, with emerging evidence suggesting that immune dysregulation may contribute to disease initiation and progression. Therapeutically, novel strategies such as adeno-associated virus (AAV)-based gene replacement therapy, CRISPR/Cas-mediated gene editing systems, and lipid nanoparticle (LNP)-delivered mRNA therapeutics have demonstrated partial restoration of auditory function in animal models of hereditary hearing impairment involving genes such as TMC1, OTOF, and GJB2, with some approaches having advanced to clinical trial stages. This article systematically summarizes recent advances in the molecular mechanisms, immune microenvironment involvement, and gene therapy strategies for hereditary hearing impairment, delineates the research trajectory from gene discovery and mechanistic elucidation to therapeutic development, and discusses future translational research directions and clinical challenges.

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Storage and Stability of AAV-Containing Fibrin Hydrogels for Retinal Gene Therapy.

Berger Aubrey A, Knudsen Travis T, Kopp Francesca F, Korda David D et al.

Subretinal and intravitreal injection of retinal gene therapy is associated with serious adverse events and poor efficacy. We sought to improve retinal gene therapy delivery by developing fibrin hydrogel encapsulated adeno-associated virus (FE-AAV). Here we investigate conditions for storage and stability for FE-AAV. FE-AAV containing 1.9 × 109 genome copies of AAV2/2-CMV-GFP was manufactured using fibrinogen reconstituted in 0.01 M sodium citrate, pH 7 (NaC) or phosphate-buffered saline containing 0.001% (v/v) Pluronic F68 (F68). Samples were stored at either -80 °C or 4 °C for up to 16 weeks. Changes in transduction efficiency, and mechanical and physical properties were evaluated. In vitro transduction was significantly (p < 0.05) reduced for FE-AAV manufactured with NaC. In contrast, we observed no change in transduction through 16 weeks for FE-AAV made with F68. Physical changes occurred in FE-AAV stored at -80 °C. In contrast to FE-AAV formulated with NaC, FE-AAV formulated with F68 and stored at 4 °C for 16 weeks was essentially equivalent to freshly made FE-AAV and retained the ability to transduce retinal pigment epithelial (RPE) cells in the pig eye. We conclude that FE-AAV formulated with F68 and stored at 4 °C is stable and shows potential for retinal gene therapy for at least 4 months following manufacture.

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PubMedDiseases (Basel, Switzerland)2026-07-27

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Alvarado Ibarra Martha M, Mera-González Alma B AB, Tapia-Enriquez Ana L AL, Ramirez-Hoyos Ana P AP et al.

Hemophilia is an X-linked inherited bleeding disorder, classified as type A or type B. Therapeutic advances offer new treatment options that improve disease control and reduce associated complications, including inhibitor development and hemophilic arthropathy. This document aims to update the Mexican hemophilia consensus, reviewing current evidence on diagnosis and management, and addressing gaps in the treatment and follow-up of patients in Mexico, aligning local needs with international recommendations. A PubMed literature search covering the last five years (up to September 2025) was conducted, prioritizing consensus statements, guidelines, and systematic reviews. Using the Delphi methodology, a structured questionnaire was submitted electronically to forty-four experts. Aspects without initial agreement were discussed at an in-person meeting. Consensus was defined as at least 80% of votes in favor. Recommendations were issued across six domains: laboratory diagnosis, genetic testing, management of hemophilia A and B without and with inhibitors, adjuvant treatments, and gene therapy. The recommendations address prophylaxis with coagulation factor concentrates, non-factor therapies, immune tolerance induction, perioperative management, pain management, and eligibility criteria and follow-up protocols for gene therapy with adeno-associated viral vectors. This consensus provides updated, evidence-based recommendations adapted to the Mexican healthcare context, identifying priority areas, including timely access to non-factor therapies and gene therapy, development of a national referral network for complex cases, and inclusion of novel therapeutic agents in the institutional essential medicines list, with the aim of improving the quality of life of people with hemophilia in Mexico.

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Recent Insights into the Role of Herpesviridae in Alzheimer's Disease: A Structured Narrative Review Based on a Systematic Literature Search.

Plantone Domenico D, Manco Carlo C, Righi Delia D, Lago Stefania S et al.

Background/Objectives: Herpesviridae have been increasingly investigated as possible contributors to Alzheimer's disease (AD) because of their neurotropism, lifelong latency, and capacity to modulate inflammatory and amyloid-related pathways Methods: This structured narrative review, based on a systematic literature search, examined original studies published between December 2020 and December 2025 on the association between human herpesviridae and AD. Searches were conducted in PubMed, Web of Science, and Scopus using a PRISMA-informed screening framework. Results: The available evidence was heterogeneous across viruses and study designs. HSV-1 emerged as the most consistently implicated virus, supported by epidemiological, biomarker, neuroimaging, and experimental studies, although contradictory findings were also reported. VZV was mainly associated with AD through vascular, inflammatory, and vaccination-related evidence, with several studies suggesting lower dementia risk after zoster vaccination. CMV, EBV, and HHV-6 showed biologically plausible but less consistent associations, whereas HHV-7 and HHV-8 were supported only by limited or indirect evidence. Across studies, the proposed mechanisms included chronic neuroinflammation, vascular injury, amyloid and tau dysregulation, host immune responses, and cumulative infectious burden. Conclusions: Overall, the literature suggests that the relationship between herpesviridae and AD is not uniform and that HSV-1 appears to be the most relevant candidate. However, methodological heterogeneity, possible reverse causation, and unequal study intensity across viruses limit firm conclusions. More robust longitudinal and subtype-specific studies are needed to clarify causal relevance.

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