BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapyApr 30, 2026

New advances in using fat-based particles to deliver gene and biological treatments into the brain

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated May 1, 2026

Abstract

Lipid nanoparticles (LNPs) are emerging as a promising alternative for delivering complex therapeutics to the central nervous system (CNS).

Peripherally administered therapeutics face significant challenges in accessing the CNS due to anatomical and physiological barriers.
The blood-brain barrier regulates the entry of blood constituents into the CNS, impacting the delivery of therapeutics.
Recent advancements in blood-brain barrier delivery technologies have been established in pre-clinical models and are being validated in clinical studies.
LNPs have gained attention for their ability to deliver various gene therapy modalities, including messenger RNA and small interfering RNA.
Functionalizing LNPs with specific components may enhance their ability to penetrate the blood-brain barrier.
Innovations in LNP-based delivery systems could facilitate the development of more effective CNS-targeted gene therapies.

Simplified

Peripherally administered therapeutics for neurological indications are challenged with anatomical and physiological barriers that limit their ability to access their site of action in the central nervous system (CNS). This is particularly true for complex therapeutics such as antibodies, immunotherapeutics, and gene therapies. The blood-brain barrier is the specialized structure that functionally regulates the ability of blood constituents to access the CNS. Blood-brain barrier delivery technologies for protein therapeutics have been established in pre-clinical models and are beginning to be verified in clinical studies. Technologies reliant on the transcellular pathway across the blood-brain barrier utilize the receptor-mediated transcytosis mechanism. Research into the use of lipid nanoparticles (LNPs) to deliver complex therapeutics has tremendously expanded in recent years. Lipid nanoparticles represent a compelling alternative to viral vectors for the delivery of various gene therapy modalities, including messenger RNA, small interfering RNA, and antisense oligonucleotides. Functionalization of LNPs with blood-brain barrier-penetrant moieties is being explored as a means to enable CNS delivery of LNP-based therapeutics. The recent innovations and validation of LNP-based delivery systems have hastened the fulfillment of the promise of facile CNS-targeted gene therapies. This review focuses on functional aspects of the blood-brain barrier and how they relate to recent advances in LNP technologies for CNS delivery, as well as their potential impact on gene therapy.

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Funding

Competing interests

Declarations. Funding: No funding was received to conduct this study or assist with the preparation of the article. Conflict of Interest: Umar Iqbal and Will J. Costain are employees of the Government of Canada. Roy W. Hwang is a Ph.D. candidate at the University of Ottawa. None has any existing conflict of interest that is directly relevant to the content of this article. Ethics Approval: Not applicable. Consent to Participate: Not applicable. Consent for Publication: Not applicable. Availability of Data and Material: Not applicable. Code Availability: Not applicable. Authors’ Contributions: All authors contributed to conceptualizing and writing this manuscript.

PubMed

New advances in using fat-based particles to deliver gene and biological treatments into the brain

Abstract

Full Text

Funding

Competing interests

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