The San Diego therapeutics company – which is leveraging induced pluripotent stem cell (iPSC) technology to discover and develop off-the-shelf neuron replacement therapies for neurological disorders – will use the funds to advance its lead candidate.
RNDP-001 is an iPSC-derived dopamine neuron progenitor for the treatment of Parkinson’s disease, and the CIRM grant will help advance the candidate through submission of an Investigational New Drug (IND) application within the next 12 months.
RNDP-001 has already completed preclinical efficacy and safety studies: and Ryne Bio will now be able to finalize its IND-package including the production of GMP-grade materials to enable the evaluation of RNDP-001 in Phase 1 clinical trials for both inherited and idiopathic forms of Parkinson’s disease.
The underlying cause of Parkinson’s disease is progressive degeneration of a patient’s dopamine neurons – and Ryne Bio believes it can directly replace these neurons by utilizing precision manufacturing techniques.
Beyond RNDP-001, Ryne Bio has two other Parkinson’s candidates in the discovery phase: targeting mild to severe rapid progressing Parkinson’s as well as young onset Parkinson’s.
“We appreciate CIRM’s partnership in our vision to reverse degenerative conditions of the brain by developing off-the-shelf cell replacement therapies,” said Nick Manusos, Chief Executive Officer of Ryne Bio. “A dramatic shift in the standard of care for patients with neurodegenerative disease is long overdue.”
In addition to funding from CIRM, Ryne Bio was launched and seeded in 2022 by Saisei Ventures, an emerging venture capital firm focused on building revolutionary advanced medicine companies.