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By Kaylen Auer
PBN Web Editor
By Kaylen Auer
PBN Web Editor
(Updated, 3:51 p.m.)
PROVIDENCE – NsGene Inc., a biotech company based in Denmark and Providence, has received a $545,000 grant from Swedish nonprofit Karin & Sten Mortstedt CBD Solutions to investigate the application of NsGene’s BrainRepair therapeutic platform for the treatment of cortico-basal degeneration, also known as CBD.
The BrainRepair technology was developed to treat a variety of neurological disorders by delivering therapeutic antibodies and proteins directly to the brain. The device consists of a slender catheter-like tube about a millimeter in diameter, ending in a semi-permeable hollow fiber that contains human cells engineered to produce therapeutic agents targeting a given disease.
In the case of CBD – a rare neurodegenerative disorder characterized by atrophy and nerve-cell loss in the brain – the BrainRepair device will secrete therapeutic antibodies that will lower the levels of the protein Tau in the brain. “Misfolded,” or incorrectly structured Tau proteins, render brain neurons dysfunctional in patients of CBD and other neurological diseases such as Alzheimer’s disease.
The device is surgically implanted in the brain and secured out of sight beneath the skull, delivering therapeutic concentrations high enough to be effective while avoiding the risks associated with introducing therapeutic agents at high concentrations peripherally. According to NsGene, the BrainRepair platform promises to be an effective treatment option for patients with neurodegenerative disorders and less invasive than existing technologies.
“This is really truly exciting, and I think it could change the lives of millions of people,” said Denice Spero, vice president of strategic alliances for NsGene. “When we did our Alzheimer’s trial, you know, these trials are usually just to make sure the device is safe, but we could see that [some] patients were actually getting better.”
The BrainRepair device has undergone clinical tests for the treatment of Alzheimer’s disease and safety trials for Parkinson’s disease, and the new grant from CBD Solutions will allow NsGene to develop a BrainRepair treatment for CBD, in partnership with Swedish neurological research foundation AlzeCure and Hofstra University professor of pathology and neuroscience Dr. Peter Davies.
The $545,000 grant from CBD Solutions will fund the staff of scientists who will design and run experiments to engineer the therapeutic antibodies specific to CBD treatment, and will also fund the building of the BrainRepair devices themselves. Spero said the two-year grant provides for the basic science phase of development. If the basic science sees successful results, NsGene will seek additional funding for the safety trial and clinical trial phases.
Between 2,000 and 3,000 people in the United States suffer from CBD. Onset of the disease typically occurs at about 60 years of age, and symptoms are similar to the symptoms of Parkinson’s disease, including impaired balance, muscle rigidity, halting speech, and cognitive and visual-spatial disorientation.
In 2011, NsGene received a $682,000 grant from the Michael J. Fox Foundation to develop the BrainRepair platform for the treatment of Parkinson’s disease. NsGene will begin clinical trials of the Parkinson’s disease treatment next year.
The development of a BrainRepair application for Alzheimer’s disease – which has already undergone one clinical trial and is gearing up for a second – is funded by grants from ALF and Swedish Brain Power.
NsGene is also in the early stages of developing the BrainRepair device to treat hearing loss and epilepsy.
NsGene Inc. was founded in Providence in 2011 by Dr. Lars U. Wahlberg, who was also co-founder, executive vice president and chief operating officer of NsGene’s Danish parent company NsGene A/S. The company works with research and development teams in biopharmaceutical companies and academic institutions to identify therapeutic agents and develop technologies to target protein therapeutics directly to diseased nerve cells.