BlueRock Therapeutics LP has received Orphan Drug designation (ODD) from the FDA for OpCT-001, its investigational cell therapy for retinitis pigmentosa (RP).
Let’s start with a look at BlueRock and its science.
As a wholly owned, independently-operated subsidiary of Bayer AG, the clinical-stage cell therapy company is developing regenerative medicines for intractable diseases.
Its science is based on a proprietary cell differentiation technology that uses fully developed and healthy adult donor pluripotent stem cells (PSCs) to make reprogrammable induced PSCs (iPSCs).
- Referred to as “blank canvas cells,” these iPSCs can be reprogrammed into specific cell types that have been lost or damaged in patients with a degenerative disease (such as RP).
Before we get to this cell therapy, talk about ODD.
ODD is a special regulatory status granted by the FDA for certain investigational drug candidates that demonstrate promise for diagnosing, treating, or preventing so-called “orphan” diseases.
- To note: These are classified as rare, serious, or life-threatening diseases or conditions (such as Stargardt) that affect <200,000 patients in the United States.
And what advantages come with this designation?
While candidates deemed to be “orphan drugs” undergo the same scientific review process as any other drug seeking regulatory approval for commercial licensing in the United States, they are also qualified to receive:
- Tax credits for qualified clinical trials
- User fees exemption
- Potential 7 years of market exclusivity following approval
Duly noted. Now to OpCT-001.
What it is: An iPSC-derived cell therapy candidate designed to restore vision loss in both pediatric and adult patients diagnosed with primary photoreceptor diseases (PPDs; a subgroup of inherited retinal diseases)—including RP.
- Some background: OpCT-001 was originally developed in partnership with Opsis Therapeutics and FUJIFILM Cellular Dynamics before being licensed by BlueRock (more details here).
And on the U.S. regulatory front: The cell therapy already received Fast Track designation (in February 2025) from the FDA for the treatment of PPDs.
So what has its clinical experience encompassed thus far?
The asset is currently under evaluation in the ongoing CLARICO trial (NCT06789445), a phase 1/2a study initiated following the FDA’s clearance of an investigational new drug (IND) application in September 2024.
And what do we know about that study?
The design: First-in-human (FIH), multisite, two-part interventional trial.
- The purpose: To evaluate the safety, tolerability, and effect of OpCT-001 when administered in several dose levels as a subretinal injection.
The participants: An estimated 52 adults (aged 18+) diagnosed with PPD.
- See here for inclusion and exclusion criteria.
And the setup?
As we mentioned, it’s a two-parter:
Phase 1: Four planned dose levels administered across four cohorts (cohorts 1-4), with dose escalation conducted using a standard 3+3 scheme (see here for a cohort breakdown).
- This involves a total of 12 to 24 legally blind participants (~3 to 6 per cohort) receiving escalating doses of OpCT-001 after acceptable safety signals with the prior lower dose.
Phase 2: Will enroll a maximum of 30 participants randomized 1:1 across two cohorts (cohorts 5 and 6) to gather additional safety data and assess two dose levels of (based on phase 2 data).
- Its purpose: To evaluate OpCT-001’s effect on measures of:
- Visual function
- Functional vision
- Anatomical measures of engraftment in different clinical subgroups
To note: Participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments for phase 2.
Speaking of measurements ... what is the entire study looking at?
The primary outcome: Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAs).
- The timeframe: From the onset of OpCT-001 administration to the Week 52 visit.
And the secondary outcome: Change from baseline (through Week 52) in outer retinal layer thickness of the treated study eye, as measured via spectral-domain optical coherence tomography (SD-OCT).
- The timeframe: From enrollment through the Week 52 visit.
So when is a data readout expected for the first part of the study?
Good question … the last clinical update on this study was in July 2025, when BlueRock reported the first patient dosing of OpCT-001.
- Per Clinical Trials: The estimated study completion date is October 2030.
Stay tuned for results in the meantime!