Our Treatment

Focus Area

Age-related macular degeneration (AMD) resulting in Geographic Atrophy (GA) is the leading cause of global vision impairment in the Western World, impacting one out of every five individuals aged 75 and above. Deterioration of the retina macula, brought about by aging, genetic elements, toxicity, and inflammation, results in distorted, wavering vision which can progress to complete loss of central vision.

Luxa Biotechnology's clinical plan is to prevent vision loss by transplanting new retinal pigment epithelial (RPE) cells under the retina. By enhancing the health of the RPE layer, the goal is to improve vision for patients with dry AMD and GA.

What is Age-related Macular Degeneration?

The back of the eye is lined by a layer called the retina, a thin layer of nerve tissue that acts like the film inside a camera. In the middle of the retina is the macula, a concentration of photoreceptor cells that detect light and color, which plays the most important role in vision.

Age-related Macular Degeneration is an eye disease caused by degeneration in the macula.

Luxa’s Disease Modifying Approach

Dry AMD patients experience progressive vision loss and do not have an available vision-improving treatment. The disease involves early loss of the retinal pigment epithelial (RPE) cells that support the overlying photoreceptor cells in the central part of the retina. Our strategy is to replace the RPE cells that have been lost due to disease, and thereby improve vision. We discovered a unique adult stem cell source of RPE cells- a resident stem cell present in the native human RPE layer. These adult RPE stem cells (RPESC) produce millions of new healthy RPE cells in tissue culture. These RPE cells exhibit critical functions that support retinal health, including phagocytosis of the photoreceptor outer segments and the secretion of beneficial growth factors.

Subretinal injection of the cells is performed in the operating room under anesthesia.

Development Status

Summary

  • Technology : RPESC-RPE-4W - A superior and patented RPE Cell Replacement Therapy

  • Indication : Dry Age-related Macular Degeneration (AMD) and Geographic Atrophy (GA)

  • Clinical Trial Phase : 1/2a

  • ClinicalTrials.gov ID : NCT04627428

Ongoing Clinical Trials

  • The FDA allowed the Phase I/IIa clinical trial, which initiated in 2022 at the Kellogg Eye Center in Michigan, USA.

  • Two additional clinical sites are scheduled to open in Fall of 2024.

  • The clinical grade RPESC-RPE-4W cells are injected subretinally in patients with AMD / GA who have enrolled in the clinical trial.

  • Clinical data publication is planned in 2025.

Completed Preclincal Studies

  • Optimization of growth conditions to create the RPESC-RPE-4W product.

  • In vitro engraftment studies of RPESC-RPE-4W demonstrating extensive engraftment on human RPE monolayers.

  • Inability of RPESC-RPE-4W cells to form tumors

  • In vivo engraftment studies of RPESC-RPE-4W in preclinical models demonstrating extensive in vivo engraftment.

  • In vivo Transplantation of RPESC-RPE-4W in two animal models of retinal degeneration demonstrating durable vision rescue.

  • GMP manufacturing and cryo-preservation of RPESC-RPE-4W cells established at Cedars Sinai GMP facility, Los Angeles, USA.

  • Pre-IND Safety and Efficacy studies demonstrating no safety concerns in animal models.

The types of Age-related Macular Degeneration 

Dry AMD: Dry AMD is caused by RPE degeneration and is characterized by abnormal deposits called drusen that form under the retina. Drusen are accumulated waste products of the retina, which can grow in size and contribute to the dysfunction and death of the RPE cells. This in turn causes impairment and death of the photoreceptors and other retinal cells, leading to vision blurring and loss.

Wet AMD: Wet AMD is caused by the formation of abnormal blood vessels growing under and through the macula, causing fluid leakage and bleeding. This can damage the macula and cause a rapid deterioration of central vision.

The symptoms of Age-related Macular Degeneration

Early-stage dry AMD doesn't cause any symptoms and can go unnoticed unless signs are detected by a retinal examination. In intermediate dry AMD, some people notice mild symptoms, such as mild blurriness in their central vision or trouble seeing in low lighting.

Late AMD is accompanied by a blurry area in the center of vision. Over time, this blurry area enlarges and deepens to result in legal blindness.

Age-related macular degeneration consists of two clinical types: wet AMD and dry AMD.
Dry AMD accounts for approximately 90% of all AMD cases.
AMD is the leading cause of vision loss in people over the age of 65.
According to the global market research agency Market Research Future (MRF), the economic impact of macular degeneration is expected to reach $15.3 billion in 2027 from $9.6 billion in 2021.

As the aging population progresses, the impact on patients and their families and the economic impact will increase.