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Biodegradable nanoparticles for targeted gene delivery
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Get them answered by the team at Daiichi Sankyo.
Background

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.

What we're looking for

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.

Solutions of interest include:
  • Biodegradable nanoparticles
  • Polymer-based nanoparticles
  • Dendrimer nanoparticles
  • Peptide-based carriers
Our must-have requirements are:
  • Preliminary data demonstrating a high capacity for mRNA/DNA (~15 kb) and/or proteins
  • Initial findings suggesting effective gene delivery
  • Reasonable expectation of low toxicity and immunogenicity, for frequent administrations
  • Potential for patentability
Our nice-to-have's are:
  • 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

What's out of scope:
  • Lipid nanoparticles
  • Silica-based nanoparticles
  • Research focused solely on developing ligands for targeted organ delivery
  • Nanoparticles with complex manufacturing processes that cannot be scaled up for production
Acceptable technology readiness levels (TRL):
Levels 3-5
What we can offer you
Eligible partnership models:
Sponsored research
Benefits:
Sponsored Research
Up to €0.3 million per year for 2 years with potential follow-on funding for 1 year, including indirect costs.
Expertise
Regular meetings will be conducted to review progress and explore research directions, with the participation of industrial mentors.
Who we are

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/

 Learn more
Reviewers
MN
Masatoshi Nagamochi
Associate Director
MK
Mikio Kato
senior director
TA
Takahide Aburatani
Senior Director
Q&A with Daiichi Sankyo

Ask the team at Daiichi Sankyo any questions you have about this RFP.

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Q.
I am interested in applying to the "Sponsored Research Project". I am working with biodegradable polymers for transfering IVT mRNA into the lung by nebulisation or intratracheal application. We do have good preclinical data in mouse models. Our Polymers can not be used for intravenous application. Can I apply or are you only intersted in intravenous applications? Thank you very much for answering my questions.
1
A.
Dear Dr. Rosenecker, Thank you very much for expressing your interest in the Sponsored Research Program and for your inquiry. While it is acceptable for the method of administration to be nebulisation or intratracheal application when applied to the lungs, we hope that the nanoparticle itself could be administered intravenously, considering potential applications to other organs. If your nanoparticle is applicable for IV administration, we would be delighted to receive your application. Thank you very much for your question. Best regards,
TA
Takahide Aburatani, Senior Director, Daiichi Sankyo
November 7, 2024
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Deadline: December 31
Seeking partners focused on
Biopharmaceutics
Biophysics
Immunology
Molecular Medicine
Nanoengineering
Nanomaterials
Nanotechnology
Polymer Behavior
Gene Therapy
Drug Delivery
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Biotechnology
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