Ryne wins $4M to advance cell replacement therapy for Parkinson’s

Funding is through a CIRM Clinical Stage Research Program grant

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Ryne Biotechnology has been awarded $4 million to advance RNDP-001, its investigational dopamine-producing, or dopaminergic, neuron replacement therapy for Parkinson’s disease.

The funding comes in the form of a Clinical Stage Research Program grant from the California Institute for Regenerative Medicine (CIRM).

Ryne plans to use the award to finalize the package for an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) within the year to test the therapy in trials, having completed preclinical safety and efficacy studies.

This will include producing Good Manufacturing Practice (GMP)-grade RNDP-001 to ensure the therapy meets FDA quality control standards before it can be used in people. Greenlighting the IND would set the stage for Phase 1 clinical testing of RNDP-001 in people with familial and idiopathic (without a known cause) forms of 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, Ryne Bio’s CEO, in a company press release. “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 progressive loss of neurons that produce dopamine, an important brain signaling chemical, is a hallmark of Parkinson’s disease. As such, a number of Parkinson’s treatments are focused on boosting dopamine levels in the brain.

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Replacing lost cells with progenitor cells

RNDP-001 seeks to replace the lost cells by providing patients with dopaminergic neuron progenitor cells, which are generated through induced pluripotent stem cells (iPSCs).

Producing iPSCs involves obtaining fully matured cells, such as those in the skin or blood, and treating them with reprogramming factors that revert them to a stem cell-like state, where they can be transformed into almost any cell type in the body.

When treated with certain molecules, iPSCs will develop into dopamine-producing neuron progenitors. Rather than being made on demand, the so-called “off-the-shelf” therapy can be produced ahead of time and then preserved and stored at low temperatures.

After being surgically delivered directly into the brain, the progenitor cells should innervate, or supply neurons branches, to various brain regions, restoring more dopamine signaling circuits. The treatment is expected to aid functional recovery in both idiopathic and inherited forms of Parkinson’s.

“Ryne Bio is able to directly replace [dopaminergic] neurons that have been lost by utilizing precision manufacturing techniques,” said Howard Federoff, MD, PhD, Ryne Bio’s chief medical officer, scientific co-founder, and principal investigator on the CIRM grant.

Preclinical data showed the treatment was able to innervate multiple brain regions in rats, including the striatum, where dopaminergic inputs are characteristically lost in Parkinson’s.

In a rat model of parkinsonism, the delivery of a higher number of progenitor cells was associated with greater innervation and restoration of motor function, and better survival.

RNDP-001 leads to better innervation and more potent functional recovery than competing products, according to Ryne.

“The potential of off-the-shelf cell replacement therapies is on the cusp of being realized for complex and intractable disease,” said Jonathan Yeh, PhD, a managing partner at Saisei Ventures, which provided seed funding for Ryne Bio’s launch last year. “This funding decision from CIRM provides robust validation of the Ryne Bio approach, and supports the delivery of this best-in-class therapy to patients.”

RNDP-001’s development for inherited Parkinson’s is also supported by the Michael J. Fox Foundation for Parkinson’s Research. Ryne is also working on next-generation, gene-modified iPSC therapies for rapidly progressing Parkinson’s and early-onset Parkinson’s. Both are still in early discovery phases.