Foundation Fighting Blindness (FFB) and BlueRock Therapeutics LP, an independently-operated subsidiary of Bayer AG, are collaborating to launch a new cohort to FFB’s natural history study of inherited retinal diseases (IRDs)
I’m familiar with FFB, but not this company.
Founded in 2016 and based in Cambridge, Massachusetts, BlueRock Therapeutics is a clinical-stage cell therapy company focused on developing regenerative medicines for intractable diseases.
Its science: cell differentiation technology (cell + gene platform) that uses pluripotent stem cells (PSCs) to make highly specific cell types “with the intention to replace those lost or damaged to disease,” per the company.
These PSCs are also known to possess the ability to self-renew or differentiate into specific cell types.
What else should I know about this science?
BlueRock’s pipeline is focused on induced PSCs (iPSCs)—”blank canvas” cells, as they’re called—that can be reprogrammable into specific cell types lost to a patient with a degenerative disease.
The how: The company is using its cell + gene platform to turn fully developed healthy adult donor cells into iPSCs to treat diseases such as dry age-related macular degeneration (AMD) and IRDs (i.e. retinitis pigmentosa [RP], cone and rod dystrophy).
The therapeutic targets: neurology, ophthalmology, cardiology, and immunology.
Let’s zero in on this ophthalmology program.
Originally developed in partnership with Opsis Therapeutics and FUJIFILM Cellular Dynamics, BlueRock’s OpCT-001 program—an iPSC-derived cell therapy candidate currently in preclinical development—is being investigated for the treatment of primary photoreceptor diseases.
Note: Primary photoreceptor diseases are a subgroup of IRDs including RPand cone and rod dystrophies.
There’s more: BlueRock announced in January 2024 that it had exercised its option to exclusively license OpCT-001 from its partner companies.
And the goal: to potentially restore IRD-related vision loss by replacing degenerative tissue in the retina with functional cells.
Plus: The company is planning to file an investigational new drug (IND) application later in 2024.
Alrighty. Now explain this natural history study.
Launched in 2022, the two-part Uni-Rare Study (NCT05589714) is enrolling an estimated 1,500 patients with one of over 300 rare genes associated with IRDs.
The design: Conducted by FFB’s international Clinical Consortium, the Jaeb Center for Health Research (JCHR) is also serving as the coordinating center.
The lead investigator: Heading up the study is José-Alain Sahel, MD, distinguished professor and chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine.
When: Per Clinical Trials, the study is slated to conclude by December 2026.
And its purpose?
As FFB originally reported, the study aims to not only better understand the course of retinal degeneration and vision loss for patients with mutated genes that haven't been well characterized in clinic, but also help the therapeutic research community:
- Identify more patients for clinical trials of therapies
- Determine the design of clinical trials
- Identify therapeutic targets for more patients
So why collaborate?
As part of this partnership, a new cohort added to the Uni-Rare study will include patients with IRDs caused by rare single gene mutations.
BlueRock will then use the data from this to support the identification and development of sensitive, reliable outcome measures for clinical trials and to enable the possible discovery and development of new therapies for treating IRDs, the company reported.
Lastly, any input from these organizations?
According to FFB’s Todd Durham, senior vice president of Clinical and Outcomes Research, the Uni-Rare study is an ideal platform for FFB to collaborate with companies such as BlueRock to advance the goal of “better informed clinical trial designs for treatments of IRDs.”