Grant funding recommended for Targeted Genetics

Seattle, USA-based Targeted Genetics says that the Department of Defense Amyotrophic Lateral Sclerosis Research Program of the Office of the Congressionally Directed Medical Research Programs has recommended grant funding to the company of up to $2.4 million, to be applied towards preclinical development of new therapies for ALS.

Targeted Genetics plans to develop a small-molecule for an Investigational New Drug submission. This is based on the observations, by the firm's collaborator, John Engelhardt at the University of Iowa, that genetic mutations known to be associated with ALS, lead to over-activation of NADPH oxidase which is associated with pathogenesis of ALS. The work proposed under this grant includes formulation development and preclinical pharmacokinetic and safety studies.

"Our candidate product has shown a potential to delay the onset and progression of disease in animal models of ALS and, if this ability translates to human subjects, the molecule could have a dramatic impact on survival and quality of life," said principal investigator Barrie Carter, chief scientific officer of Targeted Genetics.