Gene Therapy

Since we are able to define the genetic subpopulations of patients with dry AMD and linked diseases at the DNA level, gene therapy provides Gemini with a potentially transformative modality.

Because gene therapy restores the activity of a defective gene with a correct copy of the gene, it is able to address the root cause of diseases and serve as a long-term therapeutic solution. For genetically defined subpopulations within those affected by dry AMD and linked diseases, gene therapy may have the potential to slow or even prevent the disease from occurring.

Gemini Therapeutics uses adeno-associated virus (AAV) to introduce into the retina a copy of the gene of interest. AAVs are naturally occurring, nontoxic viruses carrying a specific DNA sequence allowing retinal cells to make a correct copy of the gene that is defective in specific patients. The AAV viral vectors deliver the corrected gene to cells of interest and direct those cells to make a protein that restores the function to the retina of treated patients. This approach could allow physicians to treat genetically defined subpopulations of patients with dry AMD before they have lost sight and prevent the disease from progressing, allowing patients to remain independent while carrying out their daily activities.

Gene therapy expands the range of patients that Gemini can treat and offers a longer-term potential once a particular target and method of treatment have been defined.