Oculogenex, Inc.’s novel gene therapy for the treatment of intermediate age-related macular degeneration (AMD) will take flight this week as part of the SpaceX 30th Commercial Resupply Services (CRS) mission to the International Space Station (ISS) with NASA.
Let’s start with this company.
Oculogenex is a La Habra, California-based biotechnology company specializing in developing gene therapy solutions for retinal disorders, including AMD.
Of interest: Oculogenex was founded by Hema Ramkumar, MD, an ophthalmic retinal surgeon and current CEO.
And this gene therapy?
In a nutshell, the investigational ocular gene therapy is designed to be delivered as a one-time intraocular injection into the eye, conducted in-office.
- How it works: By making retinal cells more resistant to stress, the therapy works to repair cell damage and increases their lifespan.
- Its potential: To activate dormant stem cells within the retina in order to replace lost tissue, thereby targeting the root causes of dry AMD.
What makes it unique?
Per Dr. Ramkumar, the investigational therapy’s mechanism of action (MOA) is conducted via epigenetic reprogramming, including:
- Renewing retinal progenitor cells
- Inhibiting cellular senescence
- Encourages retinal epithelium cell (RPE) division
- Repairing DNA damage
- Increasing antioxidant and anti-apoptotic gene expression
- Increasing the lifespan of cells by increasing telomere length
Any clinical data on it?
Yes! But not with humans.
The gene therapy was reportedly shown to be effective against retinal damage in multiple in vitro models of dry AMD—signifying its potential to prevent advanced AMD progression.
Per the data, Dr. Ramkumar stated that 80% of treated RPE cells were shown to have more viability than young and aged RPE cells after 8 hours of oxidative stress. Further, strong protein expression was observed following injection.
Watch below.
What’s the issue with these models?
As noted by the ISS, “these models do not emulate intermediate AMD’s milder, chronic oxidative stress.”
Which brings us to using a new testing environment: space.
I’m listening …
Oculogenex is participating in the ISS National Laboratory-sponsored investigation as part of the ISS’s sponsored programs, which provide organizations with a unique research platform and the flexibility to explore key variables for improving quality of life on Earth by using the space environment as a tool.
And in this case?
Essentially, the company is seeking to confirm if spaceflight can be a novel biologic model of intermediate AMD.
Oculogenex will evaluate its investigational therapy aboard the ISS with the ultimate goal of determining whether gene therapy can prevent spaceflight-induced retinal dysfunction and degeneration, according to the ISS.
And key to this is the presence of microgravity.
Microgravity, you say?
Microgravity is a condition where gravity appears very small; it’s often used synonymously with the terms “zero gravity” and “weightlessness.”
Most notably: For astronauts who have undergone long-duration space flight (LDSF) missions in microgravity environments, microgravity has also been found to play a role in the development of spaceflight-associated neuro-ocular syndrome (SANS).
Gotcha. So what kind of testing will be conducted?
According to the ISS, experimentation will be conducted on female rodents, as the development of macular degeneration has been found to be more common in women than men.
The space environment will be the gene therapy’s disease model for AMD, with oxidative stress caused by space flight used to replicate the disease developing in patients on Earth.
And when will this flight launch?
The ISS reports that NASA’s SpaceX SRS-3 mission is scheduled to launch no earlier than Thursday, March 21, at 4:55 pm EST from the Cape Canaveral Space Force Station.
Included on this flight will be 40+ ISS National Lab-sponsored payloads.
See here for a list of other research projects in the program’s pipeline.
And click here for launch updates.