Freeline Therapeutics has advanced a new gene therapy that can potentially replace the need for lifelong factor IX injections in patients with Hemophilia B. This could emerge as a game changer in the management of Hemophilia B, a hereditary bleeding disorder. The absence of factor IX which is a naturally produced protein known for its blood clotting function causes this disorder. The absence or inadequate presence of factor IX protein would result in uncontrolled bleeding. Hemophilia B is also referred as the Christmas disease which is now a well-known rare disease. But, it is easily diagnosed when compared
to many other rare diseases. Factor IX injections belong to the therapeutic class of antihemophilic agents and are administered through IV path. This has been the standard of care, which can be potentially replaced by the gene therapy by eventually eliminating the need for lifelong use of injections.
Understanding Hemophilia B from standard of care perspective and its limitations
Hemophilia B is diagnosed in approximately one in 25000 male births. Severe Hemophilia B cases are diagnosed within in one or two years of birth while moderate symptoms are expressed up to five or six years of age. The causes of this disorder are well known and hereditary has a significant role. Spontaneous mutations or undesirable genetic changes are also responsible for one third of the cases. It is also sex linked, it is carried though X Chromosomes. Both mother and father can pass on the disorder to the child as the X Chromosomes are found in both men and women. Hemophilia B is characterized by longer bleeding in the affected patients. This pattern of bleeding could be both internal or external. Recombinant FIX product is the available medication for Hemophilia B which is given through IV injections. In some high-risk patients these injections are also given prophylactically with a preventive intent. If a novel gene therapy can become a bankable alternative to the lifelong dependency of factor IX injections, it will potentially change the treatment paradigm and eventually become the new standard of care.
Freeline Therapeutics Gene Therapy innovations powered by AAV platform
technology
AAV stands for Adeno-associated virus vectors and is now considered as one of the leading gene delivery platforms. Freeline Therapeutics has been working on the possibilities of developing innovative therapies based on their proprietary AAV platform. Advancing gene therapy that can effectively target wide range of diseases enabled by its novel AAV capsid is a landmark accomplishment worth mentioning. Freeline Therapeutics Gene Therapy prospects for Haemophilia B based on the AAV platform is now backed by the positive clinical outcomes. A single gene therapy administered in ten patients has resulted in desirable outcomes in 90% of the patients. These outcomes are measured based on the durable normal factor IX levels in the patients who received gene therapy and did not require weekly replacement therapy further. These ground-breaking outcomes can form the basis for the novel Hemophilia B gene therapy’s regulatory approval.
