Generation Bio Achieves Two Milestones in Non-Viral Gene Therapy for the Treatment Of Hemophilia A

Feb, 2021 - by CMI


Generation Bio Co., a genetic medicines company focused on R&D of a new class of gene therapy for the treatment of several rare and prevalent diseases, announced data from a study related to the use of the company’s cell-targeted lipid nanoparticle (ctLNP) system for the treatment of hemophilia A.  

Hemophilia A is an inherited, hereditary bleeding condition caused by a deficiency of a particular blood clotting protein, factor VIII. It is the most common type of hemophilia and accounts for about one percent of all hemophilia cases. Hemophilia A, unlike hemophilia B, does not result from damage or injury to the clotting system. Instead, hemophilia A occurs when the body's autoimmune system mistakenly attacks healthy blood clotting proteins. People with hemophilia A are usually free of bleeding but occasionally minor cuts, bruises, scars, or similar injuries can cause minor, temporary blood clots that break off and travel through the blood flow and into the tissues.

The prevalence of hemophilia A at birth was 25 per 100,000 males, according to according to a study spearheaded by World Federation of Hemophilia. Treatment for hemophilia A involves drugs that reduce the activity of the factor VIII enzyme and increase the activity of factor V, making both proteins more effective in clotting. One of the most commonly used medications for hemophilia A consists of heparin, an anti-clot agent that was the first anticoagulant used in humans. Since then, a number of different heparin inhibitors have been introduced to the market. These inhibitors decrease the amount of time that active bleeding takes and reduce the risk of bleed outs. Some of these inhibitors also prevent the accumulation of cholesterol and reduce the risk of heart attack and stroke.

The recent study of Generation Bio Co. focused on assessing the tolerability and targeted factor VIII expression levels in hemophilia A mice with a single dose of closed-ended DNA (ceDNA) delivered via ctLNP system. The company also announced data from studies conducted with a ceDNA research construct delivered via ctLNP. Recently, the company, on its trading debut, reached 23% after the IPO was upsized and priced at US$ 19 a share.