In March 2021, SalioGen therapeutics raised $20m to support the preclinical validation of its non-viral gene therapy platform, exact DNA integration technology. The development of non-viral gene therapy manufacturing platforms will likely increase patient access to gene therapy and improve the cost-to-benefit ratio, says GlobalData, a leading data and analytics company.

SalioGen has engineered a mammalian derived enzyme, saliogase, to aid with the delivery of these gene therapies in vivo. The company is aiming for its technology to overcome the limitations of viral vector-associated gene therapy, enabling these therapies to be accessed by larger populations of patients. Initially they are targeting the rare diseases, familial hypercholesterolemia and inherited macular degeneration.

Healthcare payers interviewed by GlobalData argue that a gene therapy must have significant efficacy in comparison to the existing marketed therapies for rare genetic diseases, such as enzyme replacement therapy, that receive insurance coverage or reimbursement, to justify high prices.

The adeno-associated viral vector (AAV) is a vector that many gene therapies are currently based on, however, it has significant limitations that SalioGen’s technology can potentially overcome. These limitations, including a limited cloning capacity and inducing the production of neutralizing antibodies, are restricting the prophylaxis towards a variety of diseases that require viral vector-based therapies.

Akash Patel, Healthcare Analyst at GlobalData, comments: “AAVs are currently widely used to develop gene therapies for lysosomal storage disorders, which are rare monogenic disorders that result in the build-up of toxic metabolites such as with Fabry disease and Pompe disease. KOLs interviewed by GlobalData have previously stated the need for improved efficacy of therapies in this space, a lower cost to improve patient access to therapies, and trial data for gene therapies to demonstrate high efficacy and possibly curative potential.

“Rare disease markets can directly benefit from the production of non-viral gene therapy which, from improved gene transduction, will provide highly efficacious therapies that can be administered to a wider patient population. This will directly lead to an improved cost-to-benefit ratio for healthcare providers and patients for these high-cost therapies.”