Recent advances in gene therapy have enabled treatments for previously untreatable diseases. Two of the most promising platforms in this area are Adeno-Associated Virus (AAV) and Lipid Nanoparticle-messenger RNA (LNP-mRNA). AAVs are small, non-pathogenic viruses that can be engineered to deliver therapeutic genetic material into specific cells. Their ability to precisely target and insert functional genes into a patient's DNA has made them an invaluable tool for treating genetic disorders. LNP-mRNA technology, on the other hand, works by encapsulating messenger RNA (mRNA) inside lipid nanoparticles, which then deliver the mRNA into cells, prompting the production of therapeutic proteins. This approach bypasses the need for altering the patient's genetic material and can rapidly respond to various disease types.
Despite these breakthroughs, both AAV and LNP-mRNA platforms face significant challenges. Immunogenicity remains a major obstacle, as the body’s immune system often recognizes and attacks these delivery vehicles, reducing the efficacy of the therapy. Inflammatory toxicity is another concern, and manufacturability presents a critical bottleneck.
We are seeking research on novel nanoparticle technologies to overcome these limitations. Developing delivery systems that are safer and less immunogenic would bring us closer to realizing the full potential of gene therapy, potentially unlocking solutions for diseases currently beyond our reach.
We are looking for research on a high-capacity biodegradable nanoparticle technology, excluding lipid nanoparticles, capable of supporting long-term, multiple administrations of therapeutic agents. The solution should enable targeted organ delivery following IV administration and carry long nucleic acids.
Approaches for efficient gene delivery in vivo
Unique targeting mechanisms
Targeted delivery to specific organs (e.g., muscle, kidney, brain, and lung)
Scalable manufacturing potential
At Daiichi Sankyo, we attach significant importance to working with academic institutions, startups and bioventure companies to discover new therapeutics in the place where hypotheses are brought and tested in order to expand possibilities for scientific innovation breakthrough. We build sustainable relationships with partner institutions and companies through open and fair alliance management and trust based on mutual respect as the foundation for effective collaborations. Our goal is to jointly create new value for patients by maximizing each other’s expertise and strengths.
https://www.daiichisankyo.com/rd/strategy_operations/open_innovation/
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