ReviewThe AAPS journal2026
Industry Perspective on Translational and Clinical Pharmacology Aspects of Viral-based Gene Therapies and Vaccines-Key Considerations and Learnings From Approved Products.
Review in The AAPS journal, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Viral-based gene therapies and vaccines represent a rapidly evolving modality for treating a wide range of diseases. However, these novel modalities pose novel and complex challenges for translational PK/PD scientists and clinical pharmacologists during preclinical and clinical development. To support accelerated development of these transformative therapies, experts in the field formed a working group under the umbrella of AAPS and through this review provide a comprehensive overview of translational and clinical pharmacology considerations for viral vector-based gene therapies, including adeno-associated virus (AAV), oncolytic virus, adenovirus (AdV) and herpes simplex virus (HSV), and viral-vector based vaccines; with the help of robust discussion of the literature and learnings from the currently approved products. We discuss key developmental challenges, such as preclinical characterization, including dose-response evaluations and biodistribution assessments. The manuscript highlights how preclinical data, particularly the minimum effective dose (MED) and maximum tolerated dose (MTD) in relevant animal models, inform the selection of the First-in-Human (FIH) dose. We explore the challenges and strategies for clinical study design, including use of open-label, dose-escalation trials and the importance of monitoring for immunogenicity. The text also reviews clinical pharmacology considerations, such as vector shedding and tissue biodistribution, which are critical for determining product safety and efficacy. Finally, we discuss the emerging role of modeling and simulation approaches to improve preclinical-to-clinical dose translation and clinical dose optimization. By synthesizing lessons from approved products, this review aims to provide a valuable framework for navigating the complex translational and clinical pharmacology landscape of viral vector-based gene therapies and vaccines.
Indexed as
Identifiers
42618860What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.