Ray Therapeutics (RayTx) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for RTx-015, its lead investigational candidate for retinitis pigmentosa (RP).
Refresh me on RayTx.
This late-stage preclinical biotechnology company is developing potential first-in-class novel, genotype-independent, optogenetic gene therapies for severe retinal diseases.
Its science: Is based on this optogenetic approach that delivers a bioengineered, highly light-sensitive protein to targeted retinal cells—leading to visual function improvement, regardless of the underlying genetic mutation.
- In prior news: Click here to learn about the company securing an oversubscribed $100 million in Series A financing as well as, more recently, an $8 million grant from the California Institute for Regenerative Medicine (CIRM).
And how does this approach compare to other retinal disease treatments?
The general goal for retinal disease treatment has been to slow the rate of vision loss, not necessarily restore lost vision.
- Examples of this include anti-vascular endothelial growth factor (VEGF) injections and new complement inhibitors such as SYFOVRE (pegcetacoplan injection) and IZERVAY (avacincaptad pegol intravitreal solution).
According to RayTx, this approach is largely “ineffective in the later stages of disease, where photoreceptor damage is significant.”
Explain RayTx’s science.
The intent is for a single treatment of an optogenetic therapy (delivered via the same method as a gene therapy) to offer patients “lifelong benefits.”
More specifically: In lieu of treating just one genetic cause of a disease, optogenetics utilizes a precise-targeting method that doesn’t require specific gene intervention—and can bypass dysfunctional photoreceptors.
See here for a more in-depth explanation of this process.
Got it. Now to the company’s pipeline.
Two candidates are under clinical development and evaluation:
- RTx-015 (our topic of discussion) for RP and other inherited retinal diseases (IRDs), including choroideremia
- RTx-021 for Stargardt’s disease and geographic atrophy (GA)
Let’s take a closer look at RTx-015.
Administered via an in-office intravitreal (IVT) injection, this lead asset is designed to repurpose retinal neurons that survive during retinal diseases into light-sensitive photoreceptors
- These retinal ganglion cells (RGCs) then directly relay visual signals to the brain for interpretation as vision.
Its potential: To improve the quality of life for patients with retinal degenerative diseases (with RP the current focus). See below for a visual of its process.
Next up: This FDA designation.
This is reserved for regenerative medicine therapies (RMT, such as RTx-015) that use the body’s own cells to promote healing and repair tissues or organs.
Notably: It’s one of several designations in the FDA program exclusively designed for RMATs to speed up the development and review process of investigational drug candidates intended to treat, modify, reverse, or cure a serious or life-threatening condition (such as RP).
- And looking at RMAT specifically: This is based on preliminary clinical evidence indicating a specific therapy could potentially address an unmet need for the condition.
What’s the advantage of this for RTx-015?
With RMAT, sponsors (such as RayTx) are provided with “intensive FDA guidance on efficient drug development” as well as key insights into how to achieve accelerated regulatory approval, such as:
- Meeting post-approval requirements
- Potential Priority Review of a Biologics License Application (BLA)
Nice! So what’s the latest on the clinical trial front for this therapeutic?
The company is evaluating RTx-015 in the ENVISION study, an open-label, non-randomized, and multicenter phase 1 trial (NCT06460844).
The participants: An estimated 15 patients (aged 18+) with either RP or choroideremia.
The setup: Includes four varying doses of RTx-015 (low, middle, high, and higher) administered as single IVT injections.
The primary outcome: Is the development of treatment-emergent adverse events (TEAEs) among patients in each cohort over a 12-month period.
- See here for secondary outcomes.
And when might data be available from that study?
Per Clinical Trials, the study is expected to conclude in October 2030.