Ryne Biotechnology has received a $4m Clinical Stage Research Program (CLIN1) grant from the California Institute for Regenerative Medicine (CIRM) to advance its lead candidate RNDP-001.
RNDP-001 is an iPSC-derived dopamine neuron progenitor intended to treat inherited as well as idiopathic forms of Parkinson’s disease.
The company will submit an investigational new drug (IND) application within the next 12 months to advance the therapy which has concluded preclinical efficacy and safety trials.
With this CIRM grant, Ryne Biotechnology will be able to finalise its IND-package including GMP-grade materials production for evaluating RNDP-001 in Phase I trials.
These trials will evaluate RNDP-001 for both inherited and idiopathic forms of Parkinson’s disease.
Ryne Biotechnology CEO Nick Manusos said: “We appreciate CIRM’s partnership in our vision to reverse degenerative conditions of the brain by developing off-the-shelf cell replacement therapies.
“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.”
The company is also developing a platform of drug candidates, including gene-modified programmes that can modify and reverse disease progression in Parkinson’s disease and other moderate to severe disorders related to central nervous system.
Ryne Biotechnology chief medical officer, CLIN1 award scientific co-founder and principal investigator Howard Federoff said: “The underlying cause of Parkinson’s disease is progressive degeneration of a patient’s dopamine neurons.
“Ryne Bio is able to directly replace dopamine neurons that have been lost by utilising precision manufacturing techniques.”
Last year, Ryne Bio was launched and seeded by venture capital firm Saisei Ventures.