Ultra-rare neurological diseases are often without any treatment options. Even if a biopharmaceutical company decided to pursue one of these conditions, it’s unlikely that a child already diagnosed would be able to get treatment in time to prevent irreversible damage. Charles River Laboratories has embarked on a three-pronged strategy to work with families of children with these conditions to rapidly develop customized therapies. This involves the simultaneous pursuit of drug repurposing, antisense oligonucleotides, and gene therapies customized to individual patients. The company is using induced pluripotent stem cells to create differentiated cells with a patient’s genetics to study the disease and screen treatments. We spoke to Roxana Redis, Associate Science Director of Advanced Modalities for Charles River Laboratories, about the potential to rapidly develop so-called N-of-1 therapies, how Charles River does this, and why its pursuit of multiple modalities at once is designed to meet the urgency of these patients.
