Gene editing as the standard of care for the treatment of human disease

We believe that use of gene editing systems will become the standard of care for numerous diseases in the near future. Towards that end, the Innovative Genomics Foundation supports IGI’s mission to develop new gene editing systems, new delivery systems to target those gene editors to the exact tissues in the body where they are needed, and to identify new and ground breaking approaches for man of the diseases that effect our human family.

Examples of the IGI’s therapeutic approaches

Sickle Cell Disease

In 2019, doctors treated a young woman from Mississippi, Victoria Gray, with cells derived from her own bone marrow that had been enigneered using CRISPR/Cas gene editing to turn back on the production of a type of hemoglobin made normally in human fetuses in utero that is shut off upon birth. This fetal hemoglobin can help take the place of the adult hemoglobin and resume delivering oxygen where needed. Ms. Gray was the first person in the United States to be treated therapeutically with cells corrected using a CRISPR editing system. In the winter of 2023, Ms. Gray was reported to be completely free of symptoms from her sickle cell disease- free of the debilitating fatigue and pain attacks that had been a part of her whole life.

CRISPR Cures Initiative

In January of 2024, the Innovative Genomics Institute announced a partnership with Danaher Corporation to accelerate the development of the CRISPR Cures Program. The Beacon for CRISPR Cures research team, led by IGI member Dr. Fyodor Urnov along with IGI’s Founder Dr. Jennifer Doudna and IGI Executive Director Dr. Brad Ringeisen, will initially focus on developing gene-editing therapies for two rare genetic “inborn errors of immunity” (IEIs), familial hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (ART-SCID), and taking these therapies to initial regulatory submission. These diseases are typical of many rare diseases in that they have small patient populations, making drug development challenging and cost-prohibitive, so they are often neglected by the pharmaceutical industry. These projects are being worked on by IGI members in collaboration with our colleagues at UCSF and UCLA.

Sickle cell disease is a genetic disease that affects nearly 100,000 Americans and millions more globally, disproportionately affecting people of African descent. People with sickle cell disease share a single-letter DNA mutation that causes red blood cells to become crescent-shaped, or "sickled." These distorted cells can block blood vessels, causing immense pain and poor oxygen transport through the body. In addition to chronic pain, complications include organ damage, strokes, and anemia, and it can be fatal.

Sickle cell disease is caused by a single mutation in the gene encoding hemoglobin, found in red blood cells that helps to carry oxygen to tissues of the body.