ALS TDI and Asklepios to Develop New Therapeutic Delivery Options for Treating Lou Gehrig...( Collaboration to evaluate gene delive...),Health

Collaboration to evaluate gene delivery vectors for ALS

CAMBRIDGE, Mass. and CHAPEL HILL, N.C., Jan. 20 /PRNewswire-USNewswire/ -- ALS Therapy Development Institute (ALS TDI) and Asklepios BioPharmaceutical, Inc. announced today their collaboration aimed at developing a panel of viral vectors that could be used to deliver therapeutics based on targets identified by ALS TDI that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease). The collaboration between the two research centers will focus on screening and characterization of optimal delivery vectors.

"This project is an aggressive and proactive effort to develop the safest alternative therapeutic delivery options available today," reported Dr. Steve Perrin, Chief Scientific Officer at ALS TDI after a recent conference call with more than 140 members of the ALS community. "By focusing on developing therapeutic strategies while operating the largest discovery biology program for this disease, we are preparing to rapidly move potential therapeutics through the drug development process with today's patients in mind."

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 A
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