Inherited retinal diseases: Progress being made with treatment modalities


The delivery of lipid nanoparticle-based mRNA to the retina is encouraging, according to researchers.

Inherited retinal diseases: Progress being made with treatment modalities

Progress is being made in the treatment modalities for inherited retinal diseases. Specifically, the delivery of lipid nanoparticle (LNP)-based mRNA seems promising to treat these diseases, according to Marco Herrera-Barrera, MD, from the Department of Pharmaceutical Sciences, College of Pharmacy, Robertson Life Sciences Building, Oregon State University, Portland, USA.

Until now, LNP-mediated mRNA delivery could only be achieved to the retinal pigment epithelium (RPE) and Müller glia. “LNPs must overcome ocular barriers to transfect neuronal cells critical for visual phototransduction, the photoreceptors,” the investigators explained.1

They hypothesised that chemically decorating LNPs with a short 7-nucleotide oligomer peptide would facilitate penetration into the neural retina. Previous studies have shown that by crossing biologic barriers, peptides enhanced drug delivery, imaging agents and nanoparticle drug targeting.2,3

In this study, they used a peptide library to identify peptide sequences that bind the neural retina in vivo. Decoration of the peptides on the surface of LNPs with varying surface densities resulted in successful delivery of mRNA to the neural retina in a mouse model, they reported.

“These results translated to the more clinically relevant nonhuman primate, where robust protein expression was observed in the photoreceptors, Müller glia, and RPE. Overall, we identified peptide-conjugated LNPs that can facilitate the delivery of mRNA to the neural retina, expanding the utility of LNP-mRNA therapies for inherited blindness,” they concluded.

  1. Herrera-Barrera M, Ryals RC, Gautam M, et al. Peptide-guided lipid nanoparticles deliver mRNA to the neural retina of rodents and nonhuman primates. Sci Adv. 2023;9; doi: 10.1126/sciadv.add4623
  2. Albertini B, Mathieu V, Iraci N, et al. Tumor targeting by peptide-decorated gold nanoparticles. Mol Pharm. 2019;16:2430–2444.
  3. Hennrich U, Kopka K. Lutathera: The first FDA- and EMA-approved radiopharmaceutical for peptide receptor radionuclide therapy. Pharmaceuticals. 2019;12:114.
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