AVI BioPharma, Inc and Charley's Fund Inc (CFI), a not for profit organization, announced that AVI and CFI have amended their existing sponsored research agreement to provide for an additional $3 million in sponsored research funds, for a total of $5 million in support of the development of AVI-5038 through to IND. The drug candidate is based on proprietary PPMO chemistry and has the potential to skip exon 50 in certain patients with Duchenne muscular dystrophy (DMD). AVI's first contract with the Fund was initiated in October 2007 and partly supported the research that identified AVI-5038.
"We are excited that the research supported by our Fund allowed AVI to identify this drug candidate for further development. We are pleased to extend additional support to AVI to help the company reach its clinical goals in this devastating disease so that more children with DMD can be treated" said Benjamin Seckler, M.D., president of Charley's Fund Inc.
"Our new drug candidate - AVI-5038 - is based upon novel PPMO chemistry, which will potentially enhance the bioavailability and potency of exon-skipping drugs," said Leslie Hudson, president and chief executive officer of AVI BioPharma. "We appreciate the support and commitment of Charley's Fund Inc to help advance this promising drug candidate towards the clinic."
AVI has selected and begun preclinical development on a lead molecule, based on AVI's proprietary PPMO chemistry that has the potential to skip dystrophin exon 50 and so not only restore the proper RNA reading frame but also produce functional dystrophin in patients with certain types of mutation. This therapeutic approach is similar to that of AVI-4658, which AVI has in more advanced development to potentially treat DMD patients with mutations that could benefit from skipping exon 51 of the dystrophin gene.
Charley's Fund is a not-for-profit foundation that finances therapeutics development for Duchenne muscular dystrophy.
AVI BioPharma is focused on the discovery and development of RNA-based drugs utilizing proprietary derivatives of its antisense chemistry (morpholino-modified phosphorodiamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms of action.