Several gene therapies recently have been approved to treat a variety of disorders: a rare eye disease called Leber congenital amaurosis, spinal muscular atrophy in infants, and hemophilia B in adults.
In gene therapy, genetic material is delivered into patients’ bodies with the objective of replacing or correcting faulty DNA. Several gene therapies recently have been approved to treat a variety of disorders: a rare eye disease called Leber congenital amaurosis, spinal muscular atrophy in infants, and hemophilia B in adults. One limitation of current gene therapies is their delivery system. They are typically delivered to patients via a viral vector, a harmless virus that carries its genetic cargo into the body, but this approach typically only affects cells in the liver. Delivering precision genetic medicine to organs and cells beyond the liver represents one of the next frontiers in gene therapy. ReCode Therapeutics, a Leaps portfolio company, is on the forefront of that effort.