Armatus Bio has partnered with Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization to accelerate manufacturing of an Armatus gene therapy treatment for Charcot-Marie-Tooth Type 1A. The goal is to maximize program efficiency and speed to clinical studies of this novel, precision approach. The decision to initiate manufacturing is based on promising preclinical data and is a critical step in moving toward clinical studies.

The potential therapy is based on research that CMTRF funded by renowned neuromuscular disease expert, Dr. Kleopas Kleopa in collaboration with RNAi-based treatments for neuromuscular disorders expert, Dr. Scott Q. Harper and his team at Nationwide Children’s Hospital. The resulting program was licensed by Armatus Bio for further development and potential clinical trials.

Backstory: CMTRF approached Dr. Kleopa (who had already studied viral gene therapy in two other forms of demyelinating CMT) about the relevance of his approach to CMT1A. After subsequent discussions with our Scientific Advisory Board the CMT Research Foundation funded the project.

In collaboration with Dr. Harper, Dr. Kleopa’s team developed a vectorized RNAi for CMT1A that is aimed at reducing or eliminating disease symptoms by lowering levels of PMP22, the culprit behind CMT1A. The resulting program was licensed by emerging biotechnology innovator Armatus Bio.

Brian Price, PhD, Chief Technical Officer of Armatus says, “We look forward to collaborating with Andelyn on this program as we work toward supporting this population that urgently awaits innovative solutions.”

As part of the agreement, Armatus will leverage Andelyn’s extensive experience in adeno-associated virus (AAV) production and its proprietary suspension platform for development activities, plasmid manufacturing, and viral vector toxicology and GMP clinical manufacturing. The partnership strengthens the working relationship and offers great hope for patients suffering from neuromuscular disease.