In this article, we take a look at five biotech companies developing potentially transformative medicines to treat cystic ...

The CFTR protein reaches the cell membrane but the ... chloride from week 16 to week 40 VX-770: 2.2 mEq/L ± 12.2 CF = cystic fibrosis; CFQ-R = Cystic Fibrosis Questionnaire-Revised; FEV 1 ...

Cystic fibrosis is caused by faults in a gene called CFTR, which gives the body “instructions” to produce a protein that regulates salt and water in the body. “If that gene is missing or ...

The drug is expected to reach the more than 5,000 patients who don’t make any cystic fibrosis transmembrane conductance regulator (CFTR) protein, adding to the more than 65,000 people treated ...

Arcturus Therapeutics Holdings Inc. (the "Company", "Arcturus", Nasdaq: ARCT), a commercial messenger RNA medicines company ...

A gene is a specific sequence of DNA that carries the instructions for making a protein. In non-integrating gene therapy, a piece of DNA with a correct copy of the CFTR gene is delivered to an ...

Joseph Payne; President, Chief Executive Officer, Director; Arcturus Therapeutics Holdings Inc Andrew Sassine; Chief Financial Officer, Director; Arcturus Therapeutics Holdings Inc Padmanabh Chivukula ...

Good day and welcome to the Vertex Pharmaceuticals third-quarter 2024 earnings call. (Operator Instructions) Please note this event is being recorded. I would now like to turn the conference over to ...

When you think about the benefits of protein, building and maintaining muscle are likely what come to mind. But this powerhouse nutrient has many other benefits as well—from increasing energy ...