Targeted Therapies and Drug Delivery for Neurodegenerative Diseases

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Drug Targeting and Design".

Deadline for manuscript submissions: 28 February 2026 | Viewed by 495

Special Issue Editors


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Guest Editor
Department of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy
Interests: neurodegenerative diseases; pharmacology; drug discovery; drug repurposing; neuroinflammation
Special Issues, Collections and Topics in MDPI journals

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Guest Editor
Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia, 41100 Modena, Italy
Interests: neurodegenerative disease; epilepsy; pharmacology; neuroinflammation
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The treatment of neurodegenerative diseases faces two major hurdles: the complexity of disease mechanisms and the challenge of efficiently delivering therapeutics to the central nervous system. Disorders such as Alzheimer’s Disease (AD), Parkinson’s Disease (PD), Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), and epilepsy involve intricate pathological cascades, including protein misfolding, neuroinflammation, oxidative stress, and synaptic dysfunction. Moreover, the blood–brain barrier (BBB) severely limits the passage of most pharmacological agents, hindering the efficacy of potential treatments.

Recent breakthroughs in targeted therapies and advanced drug delivery systems are reshaping the therapeutic landscape, offering unprecedented precision in modulating pathological processes while overcoming biological barriers. Nanoparticle-based carriers, ligand-directed approaches, exosome-mediated delivery, and biomimetic platforms have shown great promise in enhancing drug bioavailability and selectivity. In parallel, gene- and peptide-based therapeutics are emerging as powerful tools to correct disease mechanisms at the molecular level, paving the way for disease-modifying treatments.

This Special Issue seeks cutting-edge contributions on innovative strategies that push the boundaries of neurodegenerative disease treatment. We invite original research and review articles focusing on novel drug formulations, advanced delivery platforms, and BBB-targeting mechanisms and translational studies bridging preclinical and clinical applications. By fostering interdisciplinary collaboration and highlighting the latest advancements in the field, this issue aims to accelerate the development of transformative therapies and improve patient outcomes in the fight against neurodegeneration.

We look forward to receiving your contributions.

Dr. Ramona Meanti
Dr. Chiara Lucchi
Guest Editors

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Keywords

  • targeted drug delivery
  • blood–brain barrier
  • neurodegenerative disease treatment
  • precision medicine
  • neuroscience

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Published Papers (1 paper)

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Review

24 pages, 580 KiB  
Review
Overcoming the Blood–Brain Barrier: Advanced Strategies in Targeted Drug Delivery for Neurodegenerative Diseases
by Han-Mo Yang
Pharmaceutics 2025, 17(8), 1041; https://doi.org/10.3390/pharmaceutics17081041 - 11 Aug 2025
Viewed by 299
Abstract
The increasing global health crisis of neurodegenerative diseases such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis, and Huntington’s disease is worsening because of a rapidly increasing aging population. Disease-modifying therapies continue to face development challenges due to the blood–brain barrier (BBB), which prevents more [...] Read more.
The increasing global health crisis of neurodegenerative diseases such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis, and Huntington’s disease is worsening because of a rapidly increasing aging population. Disease-modifying therapies continue to face development challenges due to the blood–brain barrier (BBB), which prevents more than 98% of small molecules and all biologics from entering the central nervous system. The therapeutic landscape for neurodegenerative diseases has recently undergone transformation through advances in targeted drug delivery that include ligand-decorated nanoparticles, bispecific antibody shuttles, focused ultrasound-mediated BBB modulation, intranasal exosomes, and mRNA lipid nanoparticles. This review provides an analysis of the molecular pathways that cause major neurodegenerative diseases, discusses the physiological and physicochemical barriers to drug delivery to the brain, and reviews the most recent drug targeting strategies including receptor-mediated transcytosis, cell-based “Trojan horse” approaches, gene-editing vectors, and spatiotemporally controlled physical methods. The review also critically evaluates the limitations such as immunogenicity, scalability, and clinical translation challenges, proposing potential solutions to enhance therapeutic efficacy. The recent clinical trials are assessed in detail, and current and future trends are discussed, including artificial intelligence (AI)-based carrier engineering, combination therapy, and precision neuro-nanomedicine. The successful translation of these innovations into effective treatments for patients with neurodegenerative diseases will require essential interdisciplinary collaboration between neuroscientists, pharmaceutics experts, clinicians, and regulators. Full article
(This article belongs to the Special Issue Targeted Therapies and Drug Delivery for Neurodegenerative Diseases)
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