Collaboration to evaluate gene delivery vectors for ALS
"This project is an aggressive and proactive effort to develop the safest alternative therapeutic delivery options available today," reported Dr.
The custom constructed Biological Nano Particle (BNP) vectors are designed using proprietary technology developed by Asklepios. ALS TDI will evaluate the BNP vectors in a tightly-managed mouse colony that is based on mutations in a specific mutated protein, which is a cause of a genetic form of disease. BNP vectors are of interest to drug development scientists because they generally do not elicit as aggressive an immune response often associated with other viruses, and have been clinically tested. Samples from animals treated with BNP vectors will be analyzed by a variety of molecular and immunohistochemistry analysis techniques at ALS TDI to determine in which cell types each vector becomes localized in various tissue samples. This project is seen as bolstering tools available as part of a key therapeutic strategy used by ALS TDI to develop effective therapeutics for ALS: viral gene therapy.
"There is a consensus that gene therapeutics are ideally suited to target the largely unmet medical needs of a variety of orphan diseases including ALS. I'm excited that AskBio has formed a partnership with ALS TDI, as it combines our experience in developing gene delivery technology with the expertise and capabilities that ALS TDI have in screening ALS therapeutic leads. Our main objective is to resolve issues that have limited successful gene delivery approaches to ALS in the past, and I look forward with a great deal of optimism to offering this initial step toward progress for the ALS community," said
Funding for this project is made possible as part of a major, three-year,
About the ALS Therapy Development Institute
The ALS Therapy Development Institute (ALS TDI) (www.als.net), based in
About Asklepios BioPharmaceutical, Inc.
AskBio is a private clinical-stage biotechnology company engaged in the development of novel, gene therapy mediated protein therapies using a proprietary Biological Nano Particle ("BNP(TM)") technology platform. BNPs may be used to deliver a broad variety of biological material to a cell, including therapeutic genes, monoclonal antibodies, RNAi, and vaccines, among others. The company's mature intellectual property portfolio is the result of over
About Augie's Quest
MDA (www.mda.org) is the world's largest provider of ALS services and funder of ALS research. Over the years, it has expended
SOURCE ALS Therapy Development Institute