Ionis Inhaled Antisense Drug Shows Potential In CF
Promising In COPD And Asthma Too
The Phase I trial of IONIS-ENAC-2.5Rx for cystic fibrosis represents the first time an antisense medicine delivered directly to the lung has shown a significant reduction in ENaC messenger RNA levels.
You may also be interested in...
Vertex has become a biotech investor favorite amid the tumult of 2020, but the potential of its pipeline is now under question.
While Vertex appears to have the cystic fibrosis market covered with its CFTR modulators, Enterprise has been exploring another channel, TMEM16A, in the hope of developing compounds that could possibly be used by everybody with the lethal genetic disease.
After buying BlueRock Therapeutics outright last year, the Leverkusen-headquartered group has put down $2bn up front to acquire another cell and gene therapy specialist in Asklepios BioPharmaceutical.