Goodwin Biotechnology, Inc., a biological Contract Development and Manufacturing Organization (CDMO) that specializes in bioprocess development and GMP manufacturing of biopharmaceuticals utilizing Mammalian Cell Culture expression systems and Bioconjugation technologies, has partnered with Q Therapeutics, Inc., a clinical-stage developer of novel cellular therapies for central nervous system (CNS) diseases to provide GMP antibodies for manufacture of clinical trial materials. To do so, Goodwin Biotechnology completed Process Development, Scale Up, and cGMP manufacturing of an IgM antibody and an IgM: Ligand Conjugate used in the isolation of Q-Cells®, the first patented cellular therapeutic product candidate from Q Therapeutics under development for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease.
“Q-Cells are glial-restricted progenitor cells (GRPs) – early descendants of neural stem cells that produce only ‘glia’ – which make up 50 percent of cells in the brain, and are essential for supporting, maintaining and even restoring neuron health. Based on the recently cleared Investigational New Drug Application (IND) from the U.S. Food and Drug Administration (FDA), we’re now on the cusp of performing groundbreaking human studies for the treatment of patients with ALS,” said Deborah Eppstein, PhD, President & Chief Executive Officer at Q Therapeutics. “This important milestone is a testament to the commitment and dedication of all of our scientific co-workers and supporters, that include the National Institute of Neurological Disorders and Stroke Translational Research Program (U01-NS06713) at the National Institutes of Health, the Maryland Stem Cell Research Fund, Bosarge Life Sciences, Nicholas Maragakis, MD, at Johns Hopkins University, MPI Research, Inc., the Biologics Consulting Group, and the Cell Therapy and Regenerative Medicine Facility (CTRM) at the University of Utah.”
“We are very pleased with the highly skilled scientists at Goodwin Biotechnology, who met the challenge of developing and scaling up the process to manufacture a monoclonal antibody using their perfusion cell culture process, and utilizing their bioconjugation expertise to prepare the IgM-ligand conjugate needed for purification of our Q-Cells®, while overcoming the many process and product-related challenges associated with an IgM antibody,” added James Campanelli, PhD, Vice President of Research and Development at Q Therapeutics. “We also were very pleased with their preparation of the portions of the CMC section concerning this antibody for our IND submission, which has recently received clearance by the FDA, to proceed with Phase 1/2a clinical trials to evaluate the use of Q-Cells® in treating ALS patients. This is a significant milestone for these patients and their families, as well as for both companies.”
“This is an excellent example that utilized the breadth of our capabilities at Goodwin Biotechnology,” noted Muctarr Sesay, PhD, Chief Scientific Officer at Goodwin Biotechnology. “The technical team at Goodwin prides themselves in all facets of this project and being able to minimize the potential aggregation, stability, formulation, bioconjugation, and purification issues associated with a hybridoma IgM monoclonal antibody reflects our solutions oriented approach that we apply to all projects. We are proud to have partnered with and provided consultation to Q Therapeutics over the last 6 years where we manufactured the IgM and conjugated the antibody with a ligand and leveraged the regulatory skill sets of our team to prepare the antibody portion of the CMC section for their IND submission.”
ALS is a devastating condition caused by degeneration of motor neurons, the nerve cells in the brain and spinal cord that control muscle movement. ALS affects more than 30,000 people in the U.S. at any given time and nearly half a million people worldwide. To date, there has been no effective therapy for ALS and it is 80% fatal within five years of diagnosis.
Q Therapeutics selected ALS as the first clinical indication for Q-Cells based on a combination of the large unmet medical need and the significant scientific rationale supporting the multiple pathways by which healthy glial cells are believed to protect and preserve the function of motor neurons.