When Ron Cohen, M.D., CEO of Oryon Cell Therapies, recently dug into the company’s Parkinson’s disease data, he told Fierce Biotech it “sang” to him. The former internist-turned-biotech CEO knew a thing or two about Parkinson’s—an area Oryon is targeting with an autologous neuron replacement therapy designed to restore dopaminergic function.
Cohen founded Acorda Therapeutics more than three decades ago. As CEO, he led the development of Inbrija, an approved inhaled treatment for Parkinson’s patients whose other medications wear off prematurely. After selling the company's commercial drugs to Merz, he jumped at the opportunity to once again lead a Parkinson’s-focused venture—particularly as the son of a neurologist.
Oryon is emerging from stealth with a $21 million tranche of series A round, bringing its total funding—including grants—to $42 million. The funding was a key factor in Cohen’s decision to join as CEO. Investors include Neuro.VC, Byers Capital and others.
Cohen said he was particularly impressed by the company’s data. Its lead program is in an ongoing phase 1b/2a trial in which participants received a unilateral neuronal implant. According to Cohen, the therapy has shown improvements in motor function, along with neuroimaging evidence suggesting restoration of dopaminergic signaling in the transplanted brain region.
Oryon is far from the only company pursuing stem cell therapies for Parkinson’s by targeting dopamine production. Aspen Neuroscience recently announced plans for a phase 3 trial after reporting one-year data showing improved symptoms across all patients in its phase 1/2a study. Hope Biosciences has also linked its stem cell candidate to improved motor function in a phase 2 trial, while Bayer has moved into late-stage testing with a therapy aimed at reducing Parkinson’s-related involuntary movements.
Oryon’s approach, however, is differentiated and includes a built-in control. Cohen explained that most therapies rely on neuronal progenitor cells, which are implanted into the brain, where they multiply and eventually become A9 neurons—the primary dopamine-producing cells that degenerate in patients with Parkinson’s and drive the condition’s motor symptoms.
By contrast, Oryon implants fully developed A9 neurons from the outset, aiming to restore dopamine production more directly. Another key distinction is its unilateral approach: The company treats only one side of the brain, whereas most competing programs use bilateral treatment.
Cohen noted that this strategy allows comparisons not only with bilateral programs but also within the same patient—against the untreated side of the brain. The company has measured increasing signals of dopamine synaptic activity on the implanted side compared to the untreated half of the brain, and says the treatment corresponds with the functional improvements in patients.
Using a DaTscan, which evaluates the brain’s dopamine system, Oryon measures dopamine restoration on the treated side while tracking motor recovery. This creates something close to a built-in control arm, as outcomes can be compared to the untreated hemisphere.
Because Parkinson’s and other neurological diseases are particularly susceptible to placebo effects, Cohen said the imaging data are critical for assessing true efficacy.
The company presented interim trial data at the AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Diseases on March 21. Cohen said the results show unilateral treatment comparing favorably with bilateral approaches, and he expects the next phase of the trial to incorporate bilateral treatment.
“One of the big questions we now have to answer is whether we’ll see meaningfully improved results over what we observed with unilateral implants,” Cohen said. “If we do, that’s very meaningful and exciting.”
The strong placebo effect in Parkinson’s also raises the question of whether future trials should include sham surgeries. But that’s a complicated proposition considering the treatment procedure involves drilling a small hole in the skull and transplanting A9 neurons via a cannula. The absence of a sham control was a central issue when the FDA rejected uniQure’s data package for a closely watched gene therapy for Huntington’s disease.
“If you don’t do a sham, you’re shortchanging everyone in the process,” Cohen said. “You have to be ethical—and if patients are in the placebo group and the trial succeeds, they should be eligible to receive the actual therapy.”
Cohen recalled the first time, as a medical student, he walked into a hospital room and a patient addressed him as “doctor”—a moment that underscored the privilege of helping others. That sense of purpose, he said, is magnified in drug development, where the impact can reach tens of thousands of patients.
“When patients come up and tell us what our treatment has meant to their lives—that’s why I couldn’t stay away.”