Published as an invited Opinion in the Nature Index Nanoscience and Nanotechnology supplement, Dr Shadi Farhangrazi and Professor Moein Moghimi call for a new model for funding, investing in and developing therapies for rare pediatric diseases. They propose a dedicated rare-disease accelerator that brings together patient communities, scientists, investors, industry and other partners, while directing greater investment towards novel therapeutic and delivery platforms with the potential to overcome current limitations. The article argues that coordinated funding, shared expertise and solutions to critical challenges such as drug delivery are essential to transform promising innovations into safe and effective therapies for children.

 

Published in Nature Communications, this study established the scientific foundation of SMDG’s NanoLigand Carriers™. Following intravenous administration, the self-assembling peptide nanosystems engaged transferrin receptor and RAGE at the cerebral endothelium, crossed the blood–brain barrier and reached neurons and microglia. The findings demonstrate the platform’s potential for targeted delivery of nucleic acid medicines to the brain.

 

This Nature Bioengineering Community article explores the scientific thinking and development behind the self-assembling phage-mimetic peptides that became SMDG’s NanoLigand Carriers™. It explains how the platform was designed to cross the blood–brain barrier through receptor-mediated transport and enable nucleic acid medicines to reach neurons and microglia.

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Published in Therapeutic Delivery, this invited editorial by Professor Moein Moghimi and Dr Shadi Farhangrazi examines the biological and technological barriers that have limited effective drug delivery to the brain. It calls for more effective brain-targeting systems capable of crossing the blood–brain barrier and reaching the specific cells where treatment is required, highlighting the potential of receptor-targeted phage-mimetic nanosystems.

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