I received a few links and some information from Charley’s Fund that I’d like to share. The fund supports a broad array of research and some of that is outlined on the “Charley’s Fund” page of this blog. This post will focus on AVI BioPharma and the work of this company towards finding an effective therapy for Duchenne Muscular Dystrophy. AVI BioPharma holds the
predominant patent estate for exon skipping technology. The relationship between Charley’s Fund and AVI BioPharma is an excellent example of how a not for profit can spur development at a for profit. I really admire the way that Charley’s Fund approaches these types of contracts. They are aggressively funding research into therapies that can make a difference in treating Duchenne Muscular Dystrophy in the very near future. The work is urgent and important and I find it very inspiring. We can all make a difference by downloading the Live Inspired application, donating money, spreading the word etc.
Below is an excerpt from a press release issued on June 4 that demonstrates the commitment of both parties to bring this therapy from the lab to patients.
AVI BioPharma Signs an Additional Drug Development Contract with Charley’s Fund Inc. for Duchenne Muscular Dystrophy
Fund has committed total of $5Million to AVI-5038
For Immediate Release
PORTLAND, Ore., and SOUTH EGREMONT, Mass. — June 4, 2009 — AVI BioPharma, Inc. (Nasdaq: AVII) and Charley’s Fund Inc. (CFI), a not for profit organization, today 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, Ph.D., 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.
Below is an excerpt from AVI’s about us page.