Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the conference’s virtual status, the conference will maintain its purposes: to inform ideas on drug development, research, treatment, and diagnostic information related to cystic fibrosis. At the start of October, clinical-stage mRNA therapeutics company Translate Bio announced that the company would have both an oral and poster presentation at the NACFC to discuss unique mRNA treatments.
The Translate Bio Therapies
According to the company website, Translate Bio is a clinical-stage mRNA therapeutics company creating treatments that address protein or gene mutations or defects:
Using our proprietary mRNA therapeutic platform (MRTTM), we create mRNA that encodes functional proteins. Our mRNA is delivered to the target cell where the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease.
One of Translate Bio’s lead therapeutic candidates, MRT5005, is currently being explored in the Phase 1/2 RESTORE-CF trial. This study, enrolling 40 patients with cystic fibrosis, will examine the safety, efficacy, and tolerability of MRT5005. Administered through a nebulizer, MRT5005 uses mRNA to deliver a functioning CFTR protein to patients. According to the Cystic Fibrosis Foundation (CFF), CFTR:
helps to maintain the balance of salt and water on many surfaces in the body, such as the surface of the lung.
Through this treatment, patients hopefully receive functioning protein, allowing for cellular hydration and symptom reduction. Currently, Translate Bio received Fast Track, Rare Pediatric Disease, and Orphan Drug designations for MRT5005.
During the NACFC, Translate Bio will have both an oral and poster presentation. In the oral presentation, which will take place on October 21, team members will discuss mRNA and its promising ability to treat cystic fibrosis. As a whole, attendees will talk about nucleic acid therapies. During this presentation, members of Translate Bio will also share information on MRT5005 and the RESTORE-CF study.
Next, the poster presentation covers preclinical research associated with mRNA therapies. During this, researchers will cover:
- Benefits of CFTR mRNA
- Treatment advances
- Preclinical data
- Changing dosing requirements, particularly using CFTR mRNA
As a recessive genetic disorder, patients with cystic fibrosis must inherit defective genes from both parents. These genetic mutations impact salt movement throughout the body. As a result, patients with cystic fibrosis have thick, sticky mucus which accumulates in their respiratory and digestive systems. This prevents nutrient absorption, traps bacteria, and causes lung damage. Generally, cystic fibrosis occurs most often in Caucasians than those of other ethnicities. Over 70,000 patients globally have cystic fibrosis. Symptoms include:
- Infertility (males)
- Constipation or diarrhea
- Frequent lung infections
- Difficulty with exercise
- Salty skin
- Poor weight gain
- Abdominal pain
- Nasal polyps
- Difficulty breathing
- Stuffy nose
Learn more about cystic fibrosis.