Visgenx, Inc. has released positive data from a non-human primate translation study assessing the gene therapy VGX-0111 to slow or halt dry age-related macular degeneration (AMD) progression.
Refresh me on this company.
Based in San Diego, California, Visgenx is a biotechnology company developing gene-based therapeutics for degenerative retinal diseases that are based on increasing expression of the ELOVL2 gene, which has been shown to regulate aging in the retina and other tissues.
Tell me more.
The company’s science stems from research (now exclusively licensed by Visgenx) out of the Shiley Eye Institute at the University of California, San Diego (UCSD), which found that the ELOVL2 transgene expression plays a key role in maintaining the function and survival of retinal and other tissues’ cells.
Recent research has discovered that a potential decline in the biosynthesis of long chain and very long chain polyunsaturated fatty acids (LC and VLC PUFA; lipids) are instrumental in AMD development.
To note: During the aging process, the ELOVL2 gene expression’s decline can cause a reduction in LC and VLC PUFAs, which are key for vision.
Now talk about Visgenx’s strategy.
Visgenx’s therapeutic approach involves slowing cellular senescence in macular degeneration (among other age-related disorders) by using a ELOVL2 gene therapy called VGX-0111 to “turn back the clock” to a more youthful level of ELOVL2 expression, according to the company.
Now tell me about VGX-0111.
As an experimental gene therapy candidate, VGX-0111 is designed to increase (and restore) expression of ELOVL2, leading to an increase in lipid levels within the retina and, as a result, potentially slowing / halting dry AMD progression.
Now talk about this trial.
The translational study assessed the use of a single subretinal injection of VGX-0111—carrying an ELOVL2 transgene— to determine if the gene therapy expresses in the target tissues and causes LC and VLC PUFAs to increase at a dose that is well-tolerated.
Findings?
Per Visgenx, VGX-0111 was well-tolerated and successfully demonstrated its ability to appropriately express in the target tissue areas of the retina as well as cause a meaningful increase in LC and VLC PUFAs.
Significance?
According to Visgenx Co-Founder and CSO Martin Emanuele, PhD, the data supports the possibility for LC and VLC PUFAs to be increased, “with a well-tolerated treatment in a species closely related to humans.”
What’s next?
With these study results, the company plans to move its ELOVL2 development program forward toward conducting a human proof-of-concept study on VGX-011.