Title : Lipid and carbohydrate based delivery vesicles for gene therapeutics: Approaching difficult diseases
RNA therapeutics is a broad group of RNA oligo- and polymers that knock down, insert or replace a disease associated RNA. RNA therapeutics act via diverse biological mechanisms including e.g. antisense oligonucleotides (ASOs), RNA interference oligonucleotides, mRNAs and sgRNA/CRISPR systems. Some RNA therapeutics have already reached clinical trials and have been approved by FDA. For instance, Eteplirsen, a 30nt long phosphorodiamidate morpholino oligomer (PMO), characterized as a splice switching oligo (SSO) and has been used for the excision of exon 51 in dystrophin RNA which results in production of functional dystrophin gene in Duchene muscular dystrophy (DMD) patients. Improved symptoms, however, were observed in only 16% of patients, pointing on unmet need for further improvements.
In my talk I will describe recent advances in RNA bioconjugation and nanoparticle technology for the delivery of appealing RNA therapeutics. In particular, I will describe the utility of novel lipid and carbohydrate conjugation technologies as promising approach for targeted delivery of RNA to human cells.
There is currently an unmet need for reliable, specific delivery options for gene therapeutics. Specifically, RNA targeted therapy has been dramatically developed over the last decade. Multiple cell types and tissues are especially hard to target by RNA therapeutics. This includes neuronal cells in brain, well protected by blood brain barrier, and metastatic, rapidly migrating malignant cells. Using conjugations is a modern paradigm that integrates an interdisciplinary chemistry and biology, to provide new delivery options for biological drugs including DNA and RNA therapeutics. Herein, I will give a review of our most recent acquired knowledge within the field of the RNA conjugates as gene therapeutics for treating HIV-1 in brain and metastatic non small cell lung cancer.
Audience take away:
• The audience will get an update on novel bioconjugation strategies for labelling of sensitive molecules
• New methods for drug design in context of RNA gene therapeutics will be discussed
• My presentation will also help the audience to make an efficient design of gene therapeutics targeting difficult cell types, including neuronal cells in brain and metastatic malignancies