BioPharmaTrend
Latest News
All Topics
  • AI in Bio
  • Tech Giants
  • Next-Gen Tools
  • Biotech Ventures
  • Industry Movers
Interviews
Companies
  • Company Directory
  • Sponsored Case Studies
  • Create Company Profile
More
  • About Us
  • Our Team
  • Advisory Board
  • Citations and Press Coverage
  • Partner Events Calendar
  • Advertise with Us
  • Write for Us
Newsletter
Login/Join
  • AI in Bio
  • Tech Giants
  • Next-Gen Tools
  • Biotech Ventures
  • Industry Movers

Advancements in Lipid Nanoparticles for Targeted Gene Therapy in the Lungs

by Dana Sokolova  (contributor )   •   April 5, 2023  

Disclaimer: All opinions expressed by Contributors are their own and do not represent those of their employers, or BiopharmaTrend.com.
Contributors are fully responsible for assuring they own any required copyright for any content they submit to BiopharmaTrend.com. This website and its owners shall not be liable for neither information and content submitted for publication by Contributors, nor its accuracy.

   Biopharma insight   
Share:   Share in LinkedIn  Share in Bluesky  Share in Reddit  Share in Hacker News  Share in X  Share in Facebook  Send by email   |  

Researchers from MIT and the University of Massachusetts Medical School have developed a new type of lipid nanoparticle designed to deliver messenger RNA (mRNA) to the lungs. This innovative approach could potentially provide an inhalable treatment for cystic fibrosis and other lung diseases.

#advertisement
AI in Drug Discovery Report 2025

The team, led by Daniel Anderson, a professor in MIT's Department of Chemical Engineering, demonstrated highly efficient delivery of RNA to the lungs in mice. The study, published in Nature Biotechnology, showed that these lipid nanoparticles can be used to deliver mRNA encoding CRISPR/Cas9 gene-editing components. This development could pave the way for therapeutic nanoparticles capable of replacing disease-causing genes.

A major challenge in deploying mRNA as a therapeutic for treating diseases caused by faulty genes has been delivering it to the right part of the body without off-target effects. Lipid nanoparticles, with their fatty sphere structure, protect mRNA from being broken down prematurely and assist in its entry into target cells. However, until now, specifically targeting the lungs has been difficult.

In this study, the researchers developed lipid nanoparticles with a unique molecular structure consisting of a positively charged headgroup and a long lipid tail. The positive charge of the headgroup allows interaction with negatively charged mRNA and assists in mRNA escape from cellular structures. The lipid tail structure enables the particles to pass through the cell membrane.

RELATED: Current Trends in Nanomedicine: Notable FDA Approvals and Clinical Trials to Follow

After screening various combinations of these structures in mice, the researchers identified the most likely candidates to reach the lungs. Tests revealed that after a single dose of mRNA, approximately 40% of lung epithelial cells were transfected. This percentage increased to over 50% after two doses and up to 60% following three doses. Importantly, the cells that were successfully edited are the most relevant for treating lung disease, such as club cells and ciliated cells.

These newly designed lipid nanoparticles have several advantages over existing delivery methods. They break down quickly, enabling clearance from the lung within a few days and reducing the risk of inflammation. They can also be administered multiple times to the same patient if needed. In comparison, adenoviruses, another mRNA delivery method, can't be given repeatedly due to their induction of an immune response.

As part of their ongoing research, the scientists are working on enhancing the stability of their nanoparticles to allow for aerosolization and inhalation using a nebulizer. They also plan to test the lipid nanoparticles in a mouse model of cystic fibrosis, as well as develop treatments for other lung diseases like idiopathic pulmonary fibrosis and mRNA vaccines that can be delivered directly to the lungs.

This work in lipid nanoparticles for targeted gene editing in the lungs has the potential to improve treatment options for various lung diseases, providing new hope to patients suffering from these debilitating conditions.

 

Share:   Share in LinkedIn  Share in Bluesky  Share in Reddit  Share in Hacker News  Share in X  Share in Facebook  Send by email
#advertisement
ThermoFisher Scientific: Integrated genetic technologies for cell therapy development

BiopharmaTrend.com

Where Tech Meets Bio
mail  Newsletter
in  LinkedIn
x  X
rss  RSS Feed

About


  • What we do
  • Citations and Press Coverage
  • Terms of Use
  • Privacy Policy
  • Disclaimer

We Offer


  • Newsletter
  • BioTech Scout
  • Interviews
  • Partner Events
  • Case Studies

Opportunities


  • Advertise
  • Submit Company
  • Write for Us
  • Contact Us

© BPT Analytics LTD 2025
We use cookies to personalise content and to analyse our traffic. You consent to our cookies if you continue to use our website. Read more details in our cookies policy.