BlueRock Therapeutics LP announced the FDA has cleared its investigational new drug (IND) application for an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for treating primary photoreceptor diseases (PPDs)—a subgroup of inherited retinal diseases (IRDs).
First up, this company.
Founded in 2016 as a joint venture between Versant Ventures and Leaps by Bayer (an investing arm of Bayer AG, which then acquired BlueRock in 2019), the Cambridge, Massachusetts-based clinical-stage cell therapy company is developing regenerative medicines for intractable diseases.
The science: proprietary cell differentiation technology (cell + gene platform).
- Specifically: This tech uses fully developed and healthy adult donor pluripotent stem cells (PSCs) to make induced IPSCs (iPSCs)—referred to as “blank canvas cells”—that can be reprogrammed into specific cell types lost or damaged in patients with a degenerative disease.
Can I see a visual of this?
See here for a look at this scientific process, and here for details on BlueRock’s cell + gene platform.
And the therapeutic targets?
The company’s pipeline of investigational candidates centers on the following disease categories:
- Neurological
- Cardiovascular
- Immunological
- Ophthalmic
- PPDs
- Early / intermediate dry age-related macular degeneration (AMD)
- Late dry AMD / geographic atrophy (GA)
Now this candidate.
Currently in preclinical development, BlueRock’s OpCT-001 is an iPSC-derived cell therapy candidate originally developed in partnership with Opsis Therapeutics and FUJIFILM Cellular Dynamics.
Its purpose: To restore vision loss in both pediatric and adult patients diagnosed with PPDs.
- To note: OpCT-001 was the first candidate that BlueRock licensed via an exclusive option for treating PPDs, in which Opsis and FUJIFILM received an undisclosed license fee and became eligible to receive payments following certain development and commercial milestone achievements of the candidate.
And what did this exclusive licensing option give BlueRock?
The company was supported by Opsis and FUJIFILM via research, development, and the execution of “critical IND-enabling activities.”
These activities included OpCT-001’s clinical manufacturing, which was conducted at BlueRock’s Madison, Wisconsin-based current good manufacturing practice (CGMP) facility.
Nice! So what does the FDA’s IND clearance mean for OpCT-001?
BlueRock is now cleared to move forward with the candidate’s clinical testing—starting with initiating a phase 1/2a clinical study in the near future, according to Amit Rakhit, the company’s chief development and medical officer.
Any details on that study yet?
The trial is expected to be designed as a first-in-human study evaluating the safety and tolerability of OpCT-001 when administered in several dose levels as a subretinal injection in U.S.-based patients diagnosed with PPP diseases.
Plus: The study will also reportedly analyze the effect of OpCT-001 on the following:
- Retinal structure
- Visual function
- Functional vision
Any specific timeline yet?
Not quite … so stay tuned.
And the overarching significance?
With no treatment options currently available for restoring vision loss in this patient population, OpCT-001’s unique degenerative tissue-replacing cell therapy could prove to be a much-needed game changer.