Leniolisib for previously untreated activated phosphoinositide 3-kinase delta syndrome

Leniolisib is in clinical development for patients with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). APDS is an inherited disorder where the patient is unable to fight infections because the immune system (the body's natural defences) does not work properly. APDS is caused by defects in the genes that control the production of a protein called phosphoinositide 3-kinase delta 􀅐􀀞􀀑􀆒􀀔􀃕􀅑􀄺 Beginning in childhood, people with APDS develop recurrent infections, particularly in the lungs, sinuses, and ears. Over time, recurrent respiratory tract infections can lead to a condition called bronchiectasis, which damages the passages leading from the windpipe to the lungs (bronchi) and can cause breathing problems. Currently, treatment of APDS is limited to supportive therapies, with no therapy approved for the treatment of the disease.