Nucleic acid-based agents for difficult-to-treat diseases
In CNATM, we are moving away from traditional protein-centred drug development and developing nucleic acids as therapeutics. Nucleic acids are the most promising class of molecules to achieve rapid development of highly specific therapeutics - as already demonstrated by the mRNA-based COVID-19 vaccines. We are exploiting an even broader spectrum that includes chemically modified mono- and dinucleotides, oligonucleotides such as siRNAs or antimirs as well as aptamers. Infections, inflammations, tumours and cardiovascular diseases cause around 75% of all deaths in the EU. Available therapies are often ineffective or limited to symptom relief, so there is a significant medical need.
We aim to take active ingredients from development through validation to preclinical testing within a few years. Our 14 industrial partners are closely involved in the projects from the very beginning and make a significant contribution through research activities, syntheses and drug formulations.
Our cluster is divided into 4 collaborative projects. In collaborative projects 1-3, we are working on our research and development projects around 3 major overarching themes in 11 work packages. In our centrally coordinated joint project 4 we work on our innovation support measures.
Mono- and dinucleotides
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Decitabine analogues for the therapy of acute myeloid leukemia
Decitabine analogues for the therapy of acute myeloid leukemia: Decitabine is a synthetic nucleoside that can significantly slow the progression of acute myeloid leukaemia (AML) through an epigenetic
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cGAMP analogs for immune-mediated tumor therapy
cGAMP analogs for immune-mediated tumor therapy: Stimulator of Interferon Genes (STING) is a receptor that plays a significant role in inflammatory and degenerative diseases.
Oligonucleotides and their targeted delivery
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Chemical modification of siRNAs for optimized delivery into target cells
Chemical modification of siRNAs for optimized delivery into target cells: While mRNA vaccines were important in the containment of the COVID-19 pandemic, no antivirals with
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Library and machine learning assisted evolution of nanoparticles
Library and machine learning assisted evolution of nanoparticles: One of the key challenges for nucleic acid therapeutics is their delivery into cells. The embedding of nucleic acids in liposomal nanoparticles is often chosen
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microRNA-based therapy of COPD
microRNA-based therapy of COPD: Chronic obstructive pulmonary disease (COPD) is the third leading cause of death worldwide. Currently, there is no therapy that can interfere with the underlying inflammatory processes of COPD.
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ncRNA targeting in tumors and inflammation
ncRNA targeting in tumors and inflammation: WP7 focuses on the observation that macrophages may have a significant role in the development of certain tumors
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Peptide-coupled antisense oligonucleotides as a new class of antibiotics
Peptide-coupled antisense oligonucleotides as a new class of antibiotics: The widespread use of classical antibiotics carries the risk of multi-resistance and also leads to unwanted side effects such as
mRNA agents and other principles
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Evolution of mRNA vaccines
Evolution of mRNA vaccines: The replacement of uridine by 1-methylpseudouridine helped mRNA vaccines against COVID-19 to achieve significantly improved pharmacological properties. However, the limited shelf life and laborious formulation
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Aptamers against cardiovascular diseases
Aptamers against cardiovascular diseases: WP8 addresses two significant and often fatal cardiovascular diseases: Carotid artery stenoses and aneurysms of the abdominal aorta. In both cases, changes in
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Gene editing in Duchenne muscular dystrophy
Gene editing in Duchenne muscular dystrophy: WP9 is dedicated to cardiovascular diseases, in this case in the context of skeletal muscle disease. Specific guide RNAs for CRISPR/Cas-based gene therapy of DMD will be developed
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mRNA-based therapy of hepatitis B
mRNA-based therapy of hepatitis B: Hepatitis B is a very common chronic viral disease that is difficult to cure. The established therapy is based on the use of