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.
Bromsar celldöd i ögat
De nya läkemedel som Mireca utvecklar baseras på modifierade nukleotider, det vill säga DNA-byggstenar som bromsar celldöden hos nervceller i ögat, och på det viset kan bota patienter som annars riskerar att bli blinda. Företaget startade som resultatet av ett EU-projekt där bland andra RISE deltog.
– Från början arbetade vi med den farmaceutiska utvecklingen av de nya substanserna, berättar Nicolaas Schipper, biträdande enhetschef på RISE. Just nu hjälper vi Mireca med att optimera och skala upp tillverkningsprocesserna. Vi har gått från tillverkning i storleksordningen milligram till att nu kunna tillverka substanser i kilogram, samtidigt som vi följer regulatoriska GMP-krav och håller en kvalitet som gör att substanserna kan användas i kliniska studier på människor.
Långsam frisättning ska minimera injiceringar
Formuleringen som Mireca tillverkar injiceras i ögat, och en viktig del i arbetet mot ett nytt läkemedel handlar om att hitta metoder för långsam frisättning, så att patienterna inte behöver injicera dagligen.
– Vi har inte nått fram till en tillräckligt bra lösning för fördröjd frisättning av läkemedlet än, säger Nicolaas Schipper. Men tack vara ett nytt EU-projekt finansierat med anslag från Marie Skłodowska-Curie Actions (MSCA) har vi kunnat ta in två doktorander som arbetar med just detta.
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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.
