Vision is our most dominant sense, often taken for granted by those who have it. Those without it understand its importance, as its absence complicates learning, reading, walking, and living life to its fullest.
The impairment of vision occurs when an eye condition affects the function of the visual system, which has serious consequences throughout one’s life.
An individual, if he lives long enough, tends to experience at least one eye condition in their lifetime. Globally, about 2.2 billion people have some form of vision impairment. In about half of them, damaged vision can either be prevented or is yet to be addressed. Here, timely and quality care is important as it can mitigate many of the consequences of eye conditions.
Vision impairment is a financial burden not only for the individual but also for the broader economy, with an estimated annual global cost of productivity of $411 billion. To reduce this economic burden, a team of researchers used anti-inflammatory drugs in hydrogel to deliver the drugs effectively to the inflamed area and reduce inflammation in the retina.
Researchers injected mice suffering from retinal degeneration with the drug-loaded inflammation-responsive hydrogel and noted that the retina’s inflammatory factors reduced to 6.1%.
A protective effect was also found on photoreceptor cells, which get destroyed by retinal degeneration. Notably, the hyaluronic acid-based hydrogel, by allowing for different rates of degradation in each patient, minimizes the need for repeated injections.
The team plans to digitize the amount of drug and hydrogel used and treatment period as per disease progression, for future commercialization. Other areas to be assessed include the long-term stability of the drug delivery system and whether it would also work on other retinal diseases, noted Prof. Seung Ja Oh from Kyung Hee University.
Creating Extremely Small Implants with Long-Term Viability
To treat blindness, vision implants have emerged as a spectacular technology that enables people with advanced vision loss to regain some sense of vision.
When a person is blind, their visual cortex still functions, awaiting input. This is where brain stimulation comes into the picture, involving the sending of electrical impulses through an implant to the brain’s visual cortex. To achieve this, thousands of electrodes—each representing a pixel—are required to provide enough information to create an image.
According to Maria Asplund, a Professor of Bioelectronics at Chalmers:
“The more electrodes that ‘feed’ into it, the better the image would be. (The image created) Would be like the matrix board on a highway, a dark space, and some spots that would light up depending on the information you are given.”
While this technology has already been in existence for many decades now, it needs to be improved upon due to its bulky size, which also causes scarring in the brain. Then there’s the matter of implant material, which can be too rigid and corrode over time.
So, a team of researchers from the University of Freiburg, the Netherlands Institute for Neuroscience, and Chalmers University of Technology in Sweden came together to develop a really small implant. This implant is like a ‘thread’ with all these electrodes, the size of a neuron, placed in a row.
Having a remarkably small electrode means one implant can fit lots of electrodes, allowing for a more detailed image.
“Miniaturization of vision implant components is essential. Especially the electrodes, as they need to be small enough to be able to resolve stimulation to large numbers of spots in the ‘brain visual areas.”
– Study lead Asplund
The thing is, creating a really small implant, when combined with the complexity of a human body, comes with challenges, such as having the small electrode last in a moist, humid environment for a long time.
The electrical implant in this research is 40 mm wide and 10 mm thick, with its metal parts only a few hundred nanometers thick. Given its tiny size, it can’t afford to corrode or stop working. To prevent this, the team used a conducting polymer to protect the metal from corrosion and to transduce the electrical stimulation.
“The conducting polymer-metal combination we have implemented is revolutionary for vision implants as it would mean they hopefully could remain functional for the entire implant lifetime.”
– Asplund
The study has achieved the first step of making a small electrode the size of a nerve cell and having it effectively working in the brain for a very long time, the next step is to now “create an implant that can have connections for 1000s of electrodes,” which Asplund is already exploring in project Neuraviper.
Treating RPE Cells Shows Great Potential for AMD
Vision loss can affect anyone regardless of age, although most people with vision impairment and blindness are above the age of 50 years. Age-related macular degeneration (AMD) is actually among the leading causes of blindness in both the developed and developing world, but in different ways.
In the developed world, an accumulation of drusen (lipid deposits) and the subsequent degeneration of parts of the RPE, the choriocapillaris, and the outer retina cause sight to deteriorate. Meanwhile, in developing nations, the growth of abnormal blood vessels in the choroid (the middle layer between the retina and sclera) and their successive movement into the RPE cells leads to hemorrhaging or retinal detachment, causing AMD.
To help with the pressing issue, researchers from Anglia Ruskin University (ARU) reported a new way of treating a common cause of blindness. In this new way, researchers led by Professor Barbara Pierscionek used nanotechnology to build a 3D “scaffold,” which was used to grow retina cells.
Published in Materials & Design, the study showcased the successful growth of retinal pigment epithelial (RPE) cells that remained healthy and viable for up to 150 days. RPE cells, a single layer of post-mitotic cells, sit just outside the retina’s neural part. When these cells are damaged, vision can deteriorate.
The technology, called ‘electrospinning,’ was used for the first time to create a scaffold on which the RPE cells could grow. When the scaffold was treated with fluocinolone acetonide, a steroid that protects against inflammation, the cells’ resilience increased.
By promoting the growth of eye cells, the study showcases huge potential in the future development of ocular tissue for transplantation into the patient’s eye. This could also revolutionize treatment for one of AMD.
The number of AMD cases is actually expected to increase significantly in the coming years. According to recent research, an estimated 77 million people in Europe will have some form of age-related macular degeneration by 2050. These increasing numbers mean there would be a considerable need for “additional healthcare service and resource allocation.”
As a result, there has been a focus on finding efficient ways to transplant these cells into the eye, and the latest study demonstrates that nanofiber scaffolds treated with the anti-inflammatory substance can actually enhance RPE cells’ functionality and growth. According to Pierscionek:
“This system shows great potential for development as a substitute for Bruch’s membrane, providing a synthetic, non-toxic, biostable support for transplantation of the retinal pigment epithelial cells. Pathological changes in this membrane have been identified as a cause of eye diseases such as AMD, making this an exciting breakthrough that could potentially help millions of people worldwide.”
Using H2 Gas to Prevent Vision Loss from ROP
While blindness is severely affecting old people, childhood blindness is just as much of a pressing matter. Children who are visually impaired usually have delayed language, motor, emotional, social, and cognitive development, putting a significant burden on families and society.
In children, blindness can result from several causes, including vitamin A deficiency, congenital conditions, measles, rubella, glaucoma, childhood cataracts, conjunctivitis in the newborn, and retinopathy of prematurity (ROP).
Retinopathy of prematurity is a significant problem in developed and rapidly developing regions of the world. Vision loss from ROP is on the rise due to the increased survival of low birth weight and low gestational age infants.
This eye disease happens in babies who are born early or who weigh less than 3 pounds at birth. ROP occurs when abnormal blood vessels grow in the retina. This is because the retina’s blood vessels start developing in the fourth month of pregnancy and continue on until around the due date. So, when a baby is born prematurely, these blood vessels may stop developing normally, and the retina then develops abnormal ones, which can grow in the wrong direction. When they grow this way too far, these abnormal blood vessels can pull the retina up off the back of the eye.
There are no visible signs of ROP that you look for or detect, but in advanced cases, the retina may pull away from its normal position, which is called retinal detachment and can cause vision loss and blindness. If a baby had ROP and it caused damage, it may later result in the baby’s eyes wandering, not following objects, pupils looking white, and having trouble recognizing faces.
While some babies have mild cases of ROP and get better without treatment, some babies need treatment such as injections, eye surgery, and laser treatment to protect their vision and prevent blindness.
Today, anti-VEGF and retinal photocoagulation are used to treat this proliferative retinal vascular disease causing childhood blindness. However, they are known to develop several complications.
Laser therapy has the disadvantages of permanent loss of peripheral vision and a high chance of myopia. Meanwhile, anti-vascular endothelial growth factor agent (anti-VEGF) therapy in premature infants is associated with a high recurrence rate and potential systemic side effects.
Against this backdrop, the latest study published in BMC went on to find new and better therapeutic strategies to prevent or treat ROP. For this, researchers from Tianjin University looked into the viability of Hydrogen (H2) as a treatment by investigating its effects on retinal angiogenesis, neovascularization, and neuroglial dysfunction in the retinas of oxygen-induced retinopathy (OIR) mice.
The researchers focused on Hydrogen because this element is widely considered a useful antioxidative and neuroprotective method for treating hypoxic-ischemic disease (a type of brain damage caused by a lack of oxygen) without toxic effects.
Molecular hydrogen (H2) has been recognized as a novel therapeutic medical gas due to its unique properties, such as its quick diffusion into tissues, selectively scavenging highly toxic ROS, and penetrating through blood-retinal barriers. It also possesses anti-apoptotic, cytoprotective, and anti-inflammatory properties.
So, the study exposed seven-day-old mice to 75% oxygen for five days before returning to normal air conditions. On administering the different stages of hydrogen gas (H2) inhalation, the study found that 3-4% H2 does not disturb mice’s retinal physiological angiogenesis but improves vaso-obliteration and neovascularization.
Furthermore, it was found that H2 promotes vascular health by aiding in regenerating blood vessels and reducing abnormal vessel growth in the retina. H2 has also been found to protect nerve cells (astrocytes) and prevent inflammation caused by microglia.
The critical pathways involved are Nrf2-notch axis modulation and Hif-1α/VEGF, which play roles in healthy blood vessel formation and reducing harmful neovascularization. The study also showed that inhaling H2 does not negatively impact normal retinal development in healthy animals.
Unlike the current mainstream treatments like laser therapy and anti-VEGF injections, which have side effects and only target later stages of the disease, H2 was found to prevent early-stage retinal damage and promote healthy vessel growth without side effects.
These results make H2 a promising, non-invasive treatment option addressing the very source of retinal diseases. As such, the study suggests that H2 could be a safer, more effective alternative for conditions like retinopathy of prematurity (ROP), diabetic retinopathy (PDR), and other retinal vascular diseases, or H2 can be used in addition to current treatments. According to the study:
“H2 can be established as a treatment strategy that focuses on the true recovery of the injured organ, rather than merely delaying disease progression.”
However, the study has some limitations. For instance, the team couldn’t monitor hydrogen levels in the plasma and retina in real-time, and other significant signaling pathways may also contribute to H2’s protective effects against retinal neovascularization. On top of that, the OIR model, while used frequently to study experimental neovascularization, may not even fully represent human neovascular eye conditions.
Companies Involved in Restoring Sight
Now, let’s take a look at some prominent companies that are actively working on advanced therapies and technologies to treat blindness and severe visual impairments.
#1. Editas Medicine
The leading gene-editing company utilizes CRISPR technology to develop therapies for various genetic disorders, including those causing blindness, like leber congenital amaurosis (LCA).
In 2020, Editas Medicine announced the dosing of the first patient in the clinical trial of CRISPR Medicine AGN-151587 (EDIT-101), delivered via subretinal injection, for the treatment of LCA10, an inherited form of blindness caused by mutations in the CEP290 gene. While the company reported “clinically meaningful outcomes” for this BRILLIANCE trial, they’re pausing its enrollment and seeking a partner due to a small population being affected. In March, clinical trial results indicated that experimental treatment for LCA was safe and efficacious.
Now, if we look at the company’s financials, it has a market cap of $329.9 million as the shares trade at $4, down 60.51% YTD. It has an EPS (TTM) of -2.36 and a P/E (TTM) of -1.70.
For Q2 of 2024, the company reported collaboration and other research and development revenues decreasing to $0.5mln, research and development expenses increasing to $54.2mln, and general and administrative expenses increasing to $18.2mln. Meanwhile, as of June 30, 2024, Editas reported $318.3mln in cash, cash equivalents, and marketable securities.
“I am proud of the Editas team’s work and our advancement in the first half of 2024 as we move closer to becoming a commercial-stage company and continue developing clinically differentiated, transformational medicines for people living with serious, previously untreatable diseases.”
– Gilmore O’Neill, CEO of Editas Medicine
Click here to learn all about investing in Editas Medicine.
#2. Spark Therapeutics
Previously a standalone US-based public company, Spark was acquired by Swiss multinational healthcare company Roche in 2019 in a $4.8 bln deal. With a market cap of $261.48 million, the shares of Roche are currently trading at $40.39, up 11.48% YTD. It has a P/E (TTM) of 19.01 and an EPS (TTM) of 2.12 and pays a dividend yield of 3.34%.
Roche Group CEO Thomas Schienecke reported “a very strong growth in the first half year” during the company’s 2Q24 earnings call. This was driven by an 8% increase in sales. The company’s prescription medicine, Sauvimo, is approved in the US for treating people with wet AMD.
Spark, meanwhile, is a gene therapy company that has developed Luxturna, the first FDA-approved gene therapy for the inherited retinal disease LCA. Late last year, the company collaborated with SpliceBio for an undisclosed genetic retinal disease. As per the deal, SpliceBio, a genetic medicines company utilizing Protein Splicing to develop gene therapies, received $216 million plus royalties in payments.
To conclude, there are many breakthroughs and technological advancements being made in the medical world to help people of varying age levels regain their vision and lead self-sufficient and healthy lives, which points to a brighter future all around.
Click here for a list of top CRISPR companies to invest in.
