Arcturus is developing safe and effective RNA medicines to treat cystic fibrosis. Cystic fibrosis is a life-threatening disease caused by over 1800 different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein moves ions across cell membranes and is an important part of making normal mucus.
When the CFTR protein malfunctions, the body produces mucus that is too thick and clogs ducts in many organs including the lungs, pancreas, intestine, liver and kidneys. Bacteria can grow in airways clogged by thick mucus leading to persistent infections. Respiratory failure is the most common cause of death in cystic fibrosis patients. Thick mucus can also clog ducts in the pancreas, preventing digestive enzymes from being released into the gut. This causes severe digestive problems if untreated.
Cystic fibrosis affects approximately 70 000 patients worldwide and there is currently no cure for this disease. Patients with this disease only live to be about 40 years old. All approved cystic fibrosis therapeutics only treat the symptoms of the disease not the genetic cause, or only treat a single mutation that affects 4-5% of patients.
RNA medicines can be tailored to treat any mutation in the CFTR gene and so can be used to treat all cystic fibrosis patients. Arcturus’ patented Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR™) technology can safely deliver RNA medicines to affected organs.
Arcturus is developing novel RNA medicines to treat the rare disease transthyretin-mediated amyloidosis or ATTR. ATTR is caused by over 125 different mutations in the transthyretin (TTR) gene. If the TTR gene has a mutation, the TTR protein can aggregate together into clumps both inside and outside cells. These clumps are called amyloid and cause tissues to break down and organs to malfunction. Patients with ATTR can have severe heart and neurological problems including heart failure, muscle weakness and wasting, and weight loss. Specific symptoms and symptom severity are different for each mutation that causes this disease.
ATTR patients are currently treated by liver and heart transplants and by supportive treatments that alleviate symptoms. Liver transplants act as a form of gene therapy because donor livers produce normal TTR. There is no known cure for this disease.
Arcturus is using our proprietary unlocked nucleomonomer agent (UNA) Oligomer™ technology to develop RNA medicines that treat the genetic cause of ATTR and stop the body from producing mutant TTR proteins. RNA medicines can target any mutation in the TTR gene and can be safely delivered to affected organs using our patented Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR™) delivery platform.
Arcturus is harnessing the power of RNA medicines to develop new treatments for a rare disease called ornithine transcarbamylase, or OTC, deficiency.
OTC is an enzyme made by the liver that helps convert ammonia into urea. Our bodies make ammonia when we break down the proteins in our food. Too much ammonia is toxic to the brain, so it is normally converted into urea, which is removed from the blood by the kidneys.
Mutations in the OTC gene can mean the protein doesn’t function properly. When this happens, too much ammonia accumulates in the body. This can cause severe symptoms including vomiting, headaches, coma and death. OTC deficiency is an inherited disease that can cause developmental problems, seizures and death in newborn babies.
There is no cure for this life-threatening disease. Patients are put on low-protein diets and treated with drugs to try and stop their bodies from accumulating ammonia. Patients with severe symptoms may be offered a liver transplant because donor livers can make normal OTC.
Arcturus is developing RNA medicines that help the liver of any patient make healthy functional OTC enzyme. Our patented Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR™) technology safely and effectively delivers RNA medicines to liver cells.
Arcturus is developing RNA medicines to treat a severe genetic liver disease that affects infants and children. This disease has no cure and can be fatal without a liver transplant. Patients with this liver disease cannot absorb fats and vitamins properly and so fail to thrive. They also suffer from jaundice and intense itchiness.
Current treatment options can improve symptoms, but there is no available drug to treat the genetic cause of this disease. Arcturus is using our proprietary unlocked nucleomonomer agent (UNA) Oligomer™ chemistry and Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR™) delivery platform to develop RNA medicines to treat the genetic source of this fatal disease.