SalioGen Therapeutics has raised $20m to support preclinical validation of its platform, Exact DNA Integration Technology (EDIT), for developing non-viral vector gene therapies using the enzyme saliogase, which SalioGen has engineered to aid with the delivery of in vivo gene therapies. The company is initially targeting the diseases familial hypercholesterolemia and inherited macular degeneration, but envisions the platform to deliver therapies for multiple rare diseases. SalioGen aims for its technology to overcome the limitations of viral vector–associated gene therapy and allow gene therapies to become accessible to larger populations of patients. GlobalData predicts that with the increased rate of development in the gene therapy pipeline, non-viral vector–produced gene therapies, using platforms such as that developed by SalioGen, will provide access to therapies for patients with no existing, or very limited, treatment options, such as those with rare diseases.

Currently, many gene therapies are based on viral vectors, most notably the adeno-associated viral vector (AAV); however, these have some significant limitations, including limited cloning capacity and inducing the production of neutralizing antibodies. These limitations are restricting prophylaxis towards a variety of diseases that require viral vector-based gene therapies. Non-viral gene therapy will potentially provide rare disease markets with highly efficacious therapies that can be administered to a wider patient population, leading to an improved cost-to-benefit ratio for healthcare providers and patients.

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There are several challenges to the delivery of novel gene therapies to market, which non-viral gene therapy technology may potentially address, particularly on drug efficacy. 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. Some early clinical data have demonstrated that non-viral gene therapies can potentially have greater efficacy than their viral gene therapy counterparts, as they are less likely to elicit an immunogenic response. Their initial targets are rare diseases, a market that has significant potential, with multiple monogenic metabolic disorders in need of therapies that provide improved efficacy and curative potential for patients.

Currently, AAVs are being widely used to develop gene therapies for lysosomal storage disorders, which are monogenic disorders resulting in the build-up of toxic metabolites, such as with Fabry disease and Pompe disease. Key opinion leaders (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, for gene therapies, trial data that demonstrate high efficacy and possibly curative potential. As such, SalioGen Therapeutics is in a strategic position to lead in the space with its own non-viral gene therapies, having been an early innovator.

Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.

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