Sunday, April 6, 2025

Gene Therapy as Gene Editing using Viruses is now being implemented but is not "sustainable"!!: Razib Khan

 Richter cited Gilead Sciences’

treatments for hepatitis C, which achieved cure rates of more than 90 percent. The company’s U.S. sales for these hepatitis C treatments peaked at $12.5 billion in 2015, but have been falling ever since. Goldman estimates the U.S. sales for these treatments will be less than $4 billion this year, according to a table in the report.

“GILD is a case in point, where the success of its hepatitis C franchise has gradually exhausted the available pool of treatable patients,” the analyst wrote. “In the case of infectious diseases such as hepatitis C, curing existing patients also decreases the number of carriers able to transmit the virus to new patients, thus the incident pool also declines … Where an incident pool remains stable (eg, in cancer) the potential for a cure poses less risk to the sustainability of a franchise.”

Creepy admission for sure - a confession!!  lung gene disease - cystic fibrosis - is already being treated with gene editing of adults by taking a virus that goes through the whole body.   

"Before we started using the CFTR modulators to treat patients, there were typically 200-300 people with CF on the transplant list at any given time. Since these drugs became available, this number has been reduced by more than 75%....The CFTR modulators work by partially restoring function to the defective CFTR protein. For example, some of the mutations in the CFTR gene still make a protein, but the protein gets stuck inside the cell and can't get to where it needs to go, explained Marrah Lachowicz-Scroggins, Ph.D., chief of the lung development and pediatric diseases branch in NHLBI's Division of Lung Diseases. Yet, while the modulators have greatly improved patient care, they aren't perfect. People on them are required to take them twice per day, and some estimates suggest they can cost upwards of $300,000 per patient per year.


Cystic fibrosis (CF) is a genetic disorder that causes thick, sticky mucus to build up in the lungs, digestive tract, and other organs, leading to breathing and digestive problems
 

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