Approach and Technologies
Unleashing the therapeutic power of human factors
Millions of years of evolution have resulted in human proteins with optimal activity and specificity for neuronal function and repair. Our core strategy is to leverage this power in novel reparative therapies for both peripheral and central nervous systems disorders. The development of proteins as therapeutics is however often posed with manufacturing and drug delivery challenges, in particular in context of brain disorders.
We have generated novel proprietary technologies and solutions for both manufacturing and drug delivery, which we apply in the development of efficacious recombinant proteins and gene-based therapies for multiple disorders of the peripheral and central nervous systems.
Gene Therapy Device Platform
Combines the strengths of direct gene therapy with the safety of a medical device
Our proprietary gene therapy device platform, encapsulated cell biodelivery (ECB), targets the brain directly with powerful protein therapeutics. Using minimally invasive surgery, slender EC-devices can be introduced into diseased areas of the brain where the permeable distal tip secretes therapeutic protein(s) synthesized by an encapsulated engineered cell line.
The cells have been shown to survive and secrete for a year or longer, a sufficient amount of time for diseased brain cells to recover function. EC devices can produce biologics such as neurotransmitters, small peptides, enzymes, growth factors, and antibodies. They are also capable of secreting multiple protein therapeutics simultaneously, opening the door for novel disease targeting.
