Hereditary retinal disease is the most common cause of serious vision impairment among people of working age. There are currently around 5,000 people in Sweden with this type of disease and worldwide, approximately 1 out of every 4,000 suffers from some form of hereditary retinal disease. To date, very few cures or medications have been available to treat hereditary retinal disease. But now, the pharmaceutical company, Mireca, is hoping to change that. With support from RISE, the company is developing new substances for slowing down retinal degeneration (the progressive death of cells) and thereby curing many forms of hereditary retinal disease.
Slows down retinal degeneration
The new drug that Mireca is developing is based on modified nucleotides, which are the DNA building blocks that slow down the progressive death of nerve cells in the eye. It thereby cures patients suffering from diseases that could cause them to go blind. The company started out as an EU project supported by RISE and several others.
“Initially, our focus was on pharmaceutical development of the new substances,” explains Nicolaas Schipper, Section Manager at RISE. “Right now, we’re helping Mireca both optimize and scale up the manufacturing processes. We’ve gone from manufacturing quantities in milligrams to kilograms, while simultaneously adhering to the GMP requirements and quality levels such that the substances can be used in clinical studies on humans,” he says.
Slow release and fewer injections
Mireca is developing a new drug that is injected into the eye and one important component of the process focuses on slow release so that patients will not need injections on a daily basis.
Retinitis Pigmentosa, Leber’s congenital amaurosis and Stargardt disease are just some of the most common types of retinal disease. They are all forms of retinal degeneration, which means that the delicate nerve layer of light-sensing cells lining the back of the eye malfunction and disappear. It’s a rare condition and symptoms typically first occur when a person is around 10-11 years old. Those diagnosed with the disease risk becoming completely blind.
“At present, there are no treatments available for retinal degeneration,” says Barbara Brunnhuber, CEO of Mireca. “Gene therapy can help some, but we hope that the new drug we’re developing can be used on a much wider scale, curing many types of diseases that cause these types of cell changes,” she says.
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Slows down retinal degeneration
The new drug that Mireca is developing is based on modified nucleotides, which are the DNA building blocks that slow down the progressive death of nerve cells in the eye. It thereby cures patients suffering from diseases that could cause them to go blind. The company started out as an EU project supported by RISE and several others.
“Initially, our focus was on pharmaceutical development of the new substances,” explains Nicolaas Schipper, Section Manager at RISE. “Right now, we’re helping Mireca both optimize and scale up the manufacturing processes. We’ve gone from manufacturing quantities in milligrams to kilograms, while simultaneously adhering to the GMP requirements and quality levels such that the substances can be used in clinical studies on humans,” he says.
Slow release and fewer injections
Mireca is developing a new drug that is injected into the eye and one important component of the process focuses on slow release so that patients will not need injections on a daily basis.
“We’ve yet to find the best solution for slow release of the drug, but we’re certainly working on it,” says Nicolaas Schipper. “Now though, thanks to a new EU project that has received funding from Marie Skłodowska-Curie Actions (MSCA), we’ve been able to involve two PhD candidates who are focusing on this aspect specifically,” he says.
Help on a wide scale
Mireca is hoping to gain a better understanding of just how widespread application of the new drug could be. Conservative estimates are that it will be possible to use the drug in treating between 35 and 80 percent of those suffering from retinal degeneration. The company feels that its collaboration with RISE has been very successful.
“RISE continues to impress us with its chemical expertise,” says Barbara Brunnhuber. “They’ve helped us strengthen our portfolio of intellectual property and we’ve already obtained Notice of Allowance from the United States Patent and Trademark Office (USPTO), which will give us patent protection on our key substance,” she says.
