Today our ability to treat many diseases is limited only by our ability to effectively deliver genetic medicines.
Patients treated with genetic medicines receive nucleic acids, either DNA or RNA, to fix, replace or fine tune the expression of certain genes. By supplying patient cells with new functional copies of genes or fixing mutated genes, genetic medicines provide the biological “source code” to not only treat, but to cure disease. These programmable medicines have the potential to address unmet needs for millions of patients, including those with rare genetic diseases for which few effective treatments are available.
With 8 FDA approved therapies and 120 more in at least phase II trials, viral vectors are currently the standard delivery system for genetic medicines, of which, the adeno-associated virus (AAV) is the most common. Viral-delivered gene therapies have demonstrated clinical benefit across a range of genetic diseases; however, their ability to unlock the full potential of genetic medicine faces significant limitations: