AVI BioPharma will present data highlighting improved analogues of AVI’s phosphorodiamidate morpholino oligomer (PMO) chemistry. The data will be presented by Hong Moulton, director of Discovery Research.
At the 3rd Intracellular Delivery of Therapeutic Molecules: From Bench to Bedside, taking place in Montpellier, France, Dr Moulton will give a presentation entitled Targeted Gene Expression in vivo: Cell Penetrating Peptides Make Antisense Work as part of the session on applications of delivery systems on September 2, 2009.
At the Targeted Drug Delivery Conference, taking place in Lausanne, Switzerland, Dr Mouton will present on Cell Penetrating Peptidomorpholino Conjugates on September 3, 2009 as part of the peptide-bases delivery session.
Both presentations focus on AVI’s next generation chemistry, peptide-conjugated PMO (PPMO), which improves delivery to nuclei of muscle cells and so is particularly applicable to drugs that work by exon skipping.
The company claims, with appropriate dosing, PPMO compounds restored dystrophin to nearly normal levels in skeletal and cardiac muscles in a mouse model of Duchenne muscular dystrophy without inducing toxicity or an immune response against the new dystrophin expressed. As a result, force generation and function of both skeletal and cardiac muscles were improved.