The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies. [1] The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years:
An independent study [1] cites using HemaCare-sourced human cord blood, bone marrow, and mobilized peripheral blood as the starting material in a new strategy for more efficient genetic modification of human stem cells.
The study is based at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Researchers there have been looking into ways to make gene transduction, which is used to introduce an edited copy of a human gene into stem cells, more efficient. The new strategy is part of their goal to improve the potency and long-term engraftment of stem cells used for gene therapy in the clinic.
Sickle cell disease (SCD) is a genetic disorder that affects red blood cells. This disease affects people of color more commonly and is due to the effect of abnormally shaped red blood cells (crescent or sickle-shaped instead of disc shaped). The misshapen red blood cells contain a form of hemoglobin, hemoglobin S, resulting from mutation of the beta-globin gene. Hemoglobin S does not hold oxygen efficiently, and the abnormal shape of the red blood cells prevents normal flow through the blood vessels, leading to vessel blockage. This leads to the pain and tissue damage seen in patients with SCD.
Scientists corrected the defective genes of Duchenne muscular dystrophy disease by genome editing
Duchenne muscular dystrophy (DMD) is a devastating progressive disease that usually starts in early childhood. DMD occurs because of a mutated gene which fails to produce dystrophin, a protein which is important for the normal structure and function of muscle. Currently, no definitive treatments are available except for palliative therapy, which can only delay the symptoms of the disease. Moreover, available treatments are not completely effective, as they treat just one aspect of the disease, and they may have side effects in the long run.
Immunosuppressive properties of novel FoxA1 expressing Treg cells can be exploited for multiple sclerosis treatment