David Kirn, 4D Molecular Therapeutics CEO (via website)

4DMT's in­hal­able gene ther­a­py for cys­tic fi­bro­sis shows ear­ly pos­i­tive signs in tiny group of pa­tients

4D Mol­e­c­u­lar Ther­a­peu­tics an­nounced Wednes­day that its in­hal­able gene ther­a­py for cys­tic fi­bro­sis showed signs of im­proved qual­i­ty of life in an ear­ly look from its Phase I dose ex­plo­ration study.

The drug, 4D-710, is de­liv­ered via neb­u­liz­er and is de­signed to achieve ex­pres­sion of a pro­tein called CFTR in lung air­way ep­ithe­lial cells. CFTR helps keep the bal­ance be­tween salt and wa­ter on sur­faces in­clud­ing the lung, but a mal­func­tion of the pro­tein can lead to the ac­cu­mu­la­tion of a thick mu­cus that caus­es some of the symp­toms be­hind cys­tic fi­bro­sis.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.