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Axentis Pharma Gains Patents from BioDevelops Pharma to Further Cystic Fibrosis Program

Transaction covers technique that allows proteins marked for destruction to escape enzymatic digestion. Axentis Pharma has obtained patents from BioDevelops Pharma related to a cystic fibrosis treatment. The acquired therapeutic approach enables proteins, whic

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Transaction covers technique that allows proteins marked for destruction to escape enzymatic digestion.

Axentis Pharma has obtained patents from BioDevelops Pharma related to a cystic fibrosis treatment. The acquired therapeutic approach enables proteins, which get destroyed in spite of have some functionality, to escape.

In cystic fibrosis, proteins that are designed to regulate the concentration of salt in the epithelial cells of the lungs and other organs have mutations due to a genetic defect. The body thus destroys these mutated proteins, even though they are sufficiently functional.

“In patients with cystic fibrosis,” Gergely Lukacs, M.D., Ph.D., who guided the experimental setup in his labs at the Sick Kids Hospital in Toronto explains, “the protein CFTR (cystic fibrosis transmembrane conductance regulator) with mutated structure is marked with ubiquitin. These ubiquitin-marked proteins are degraded by multiple mechanisms prematurely. If this marker is missing, the protein evades destruction and can regulate the concentration of water and salt secretion in the lung despite its minor structural defect.”

The agreement between Axentis Pharma and BioDevelops Pharma includes two patent families with a series of international patent applications. They cover the use of molecules for the enzymatic removal of ubiquitin markers in incorrectly folded proteins. The inventions also protect the use of Velcade in cystic fibrosis.

Swiss company Axentis Pharma formed less than a year ago with patent-pending technology to develop drugs for diseases caused by incorrect protein folding in the endoplasmic reticulum, such as cystic fibrosis. “Just six months ago, we reached agreement with aRigen Pharmaceuticals, Japan, on terms and conditions regarding an exclusive license for developing and marketing a patented system for the liposomal encapsulation of an active ingredient designed to combat lung infections in patients with cystic fibrosis,” remarks Joerg Zielasek, president of Axentis Pharma. The company is preparing to initiate a Phase II trial in Germany using the acquired technology, tobramycin liposome formulation, against lung infections in cystic fibrosis patients.

“By acquiring the rights to BioDevelops’ technology platform, Axentis Pharma is both enhancing its development pipeline and offering both patients and investors extensive prospects for the treatment of this disease.”

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01What Is Cystic Fibrosis?

Cystic fibrosis (CF) is a genetic disorder, which means you get it from your parents at birth. It affects your lungs, pancreas, and other organs. CF changes the way chloride (salt) moves through the cells of your body. This causes the mucus (which should be thin and slippery) in various organs to become thick and sticky. Over time, this thick mucus builds up inside your airways, making it hard to breathe. The mucus traps germs and leads to infections and inflammation. It can also cause severe, long-term damage to the lungs and lead to respiratory failure (inability to breathe normally) and death. In the pancreas, the thick mucus caused by CF prevents the release of digestive enzymes when you eat. This leads to malnutrition and poor growth. CF can also cause liver disease, reproductive problems, and cystic fibrosis-related diabetes (CFRD). More than 40,000 people in the U.S. live with CF. Doctors diagnose about 1,000 new cases each year. Today, more than half of the CF population is aged 18 or older, and new treatments have expanded the life expectancy by decades.

Source: www.webmd.com ↗
02What is cystic fibrosis? A Mayo Clinic expert explains

Learn more from pulmonologist Sarah Chalmers, M.D. Cystic fibrosis (CF) is a condition passed down in families that causes damage to the lungs, digestive system and other organs in the body. CF affects the cells that make mucus, sweat and digestive juices. These fluids, also called secretions, are usually thin and slippery to protect the body's internal tubes and ducts and make them smooth pathways. But in people with CF, a changed gene causes the secretions to become sticky and thick. The secretions plug up pathways, especially in the lungs and pancreas. CF gets worse over time and needs daily care, but people with CF usually can attend school and work. They often have a better quality of life than people with CF had in past decades. Better screening and treatments mean that people with CF now may live into their mid- to late 50s or longer, and some are being diagnosed later in life.

Source: www.mayoclinic.org ↗
03What you can do

You might want to take a friend or family member with you to the appointment to help you remember information. Before your appointment, make a list of: Symptoms and when they started. Include anything that makes symptoms worse or better. All medicines, vitamins, herbs and supplements that you or your child take. Include the doses. Family history, such as whether anyone in your family has cystic fibrosis. Treatment you or your child have had for CF, if any. Include what the treatment was and if it helped. Any other medical conditions and their treatments. Questions to ask your healthcare professional. Questions to ask may include: What is likely causing these symptoms? What kinds of tests are needed? What treatment do you recommend? I or my child have other health conditions. How will cystic fibrosis affect them? Are there any limits needed? Feel free to ask other questions during your appointment.

Source: www.mayoclinic.org ↗
04Who Was Included in These Studies?

Overall, Alyftrek for cystic fibrosis treatment has been studied in a variety of people. Adults Adolescents Males and females Asian, Black, and White people

Source: www.webmd.com ↗
05What is it used for?

A sweat test is used to diagnose cystic fibrosis (CF).

Source: medlineplus.gov ↗
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Research and Statistics: Who Has Cystic Fibrosis?

About 40,000 people are living with cystic fibrosis in the United States, and there are approximately 105,000 people with CF worldwide. (3) More than 75 percent of people with the disease are diagnosed by age 2, and more than half of all people living with cystic fibrosis are 18 or older. CF occurs predominantly in white populations, at a rate of 1 in 2,500 births. Between 2 and 5 percent of white people are carriers of the CFTR gene variant but have no overt clinical signs of disease. The disease is less common among African Americans, occurring at the much lower frequency of approximately 1 out of 17,000 births. (15) CF gene variants are most prevalent in persons of northern and central European ancestries or of Ashkenazi Jewish descent. They are rarely found in Native Americans, Asians, or native Africans. (16) CF is equally common among men and women, but women patients fare significantly worse than male patients with the disease. The median survival age for female CF patients is about three years younger than it is for men, but the reasons for the poorer survival rates among women are not completely understood. (17)

Source: everydayhealth.com ↗
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