ProNAi Announces DNA Interference Drug Development Strategy for 2007
News Jan 12, 2007
ProNAi Therapeutics, Inc., a biopharmaceutical has announced its drug development strategy for 2007, and its plans to bring its lead therapeutic candidate, PNT2258, to IND submission by mid-year.
DNAi® is an approach to targeting genomic DNA using sequence-specific therapeutic agents, employing single strands of DNA to target and treat genes responsible for complex genetic diseases, such as cancer.
By acting at the DNA level, where only one or two copies of the gene exist per cell, treatment can be targeted by DNAi® drugs. With fewer targets, the activity of a DNAi® drug is expected to last longer at lower doses, and reduce some of the toxicity issues prevalent with other marketed therapies.
Dr. Richard D. Gill, President and CEO of ProNAi, said, “For many, many years, the promise of DNA-related therapies has gone largely unfulfilled, due to a daunting number of obstacles – lack of specificity and potency, high manufacturing costs, as well as the inability to deliver effective doses of DNA-based drugs – a problem that also still affects RNAi-based approaches currently in vogue. ProNAi has worked steadily and methodically to overcome each of these barriers to nucleic acid-based therapeutics and we fully expect to have our lead drug candidate, PNT2258, ready for IND submission by mid-year.”
Dr. Gill added, “ProNAi is looking forward to demonstrating the viability of DNAi®-based drugs in the coming year, as we announce our very promising preclinical results for PNT2258, which has shown exciting in vivo efficacy in xenograft mice for a number of cancers.”
“Our first drug formulations show promising utility against multiple target genes, and we are targeting well-documented, non-translated genomic sequences. We are also examining the potential for a Dx/Rx combination, and will further develop that possibility.”
Back in 2009, researchers identified a herd of Awassi sheep suffering from "day blindness". As that term implies, these sheep were blind during the day (in bright light) but could see at night, in low-light conditions. After identifying the genetic basis of this blindness, researchers have now successfully used gene therapy to restore their daytime vision.READ MORE