San Diego-based Ryne Biotechnology announced last week that it has received a $4 million Clinical Stage Research Program grant from the California Institute for Regenerative Medicine (CIRM) to move RNDP-0001 into clinical testing.
RNDP-0001 is an investigational dopaminergic neuron-replacement therapy for Parkinson’s disease that is developed using induced pluripotent stem cell (iPSC) technology. iPSC involves the reprogramming of skin or blood cells into an embryonic-like state and then developing them into another type of human cell, including neurons.
RNDP-0001 has completed preclinical efficacy and safety studies, where it demonstrated the ability to restore lost motor function when injected into the brains of rats. According to Ryne Bio, RNDP-0001 is optimized for innervation, or the ability to create nerve branches once it is surgically delivered to the brain.
The CIRM grant will allow Ryne to prepare and submit an Investigational New Drug (IND) application to the FDA in the next year. “A dramatic shift in the standard of care for patients with neurodegenerative disease is long overdue. We are thrilled to be developing groundbreaking therapies for patients in need of better treatment options,” said Nick Manusos, Chief Executive Officer of Ryne Bio.