Spotlight on Gene Therapy: finding a Cure

Our gene therapy trial for canine degenerative myelopathy was featured yesterday on TuftsNow. 

"A gene-silencing therapy under development for people with amyotrophic lateral sclerosis (ALS) is being tested at Tufts in dogs with degenerative myelopathy, a fatal paralytic disease that is similar to ALS."

This gene therapy trial for canine degenerative myelopathy is related to the development of our gene therapy candidate for SOD1-linked ALS.

"“What’s so extraordinary is that the dog is a naturally occurring model of one form of the human disease,” said Robert H. Brown Jr., a UMass Chan Medical School neurologist who is one of the world’s foremost experts on ALS and other neurodegenerative diseases. "

The canine trial follows the same delivery protocol than the future human trial.

"The dogs in the Tufts trial receive a single spinal fluid injection of an engineered adenovirus—from a family of viruses that can infect the nervous system, but is best known for causing the common cold. The engineered virus was designed to breach the blood-brain barrier to deliver DNA particles that turn off—or silence—the mutated gene. [...] The therapy has shown promising results in mice genetically engineered to have a condition similar to ALS, he said, and it also was safe when tested in normal monkeys."

The trial started in December 2016 and is currently ongoing. No results are available yet. 

"“Does it work? That’s the question I wake up and go to bed with every day,” Brown said."

Read the entire article here.

You can also read more about our human program here.