- A recently completed non-human primate study of VGX -0111 demonstrated good tolerability, provided strong transgene expression in the targeted region of the retina, and increased production of the lipids whose decline is associated with macular degeneration
- The Company is moving the development program forward with the intention of conducting a human proof of concept study
SAN DIEGO, CA, USA I June 27, 2023 I Visgenx, Inc., a biotechnology company focused on developing gene therapies for degenerative retinal diseases, today announced positive data from a non-human primate study. The important translational study used a single subretinal injection to deliver VGX-0111 (carrying an ELOVL2 transgene) to investigate whether VGX-0111 expresses in the target tissues and causes an increase in certain very long chain polyunsaturated fatty acids at a dose that is well tolerated. VGX-0111 was able to successfully demonstrate all three of these important parameters.
Dry age-related macular degeneration (AMD) is a leading cause of blindness. The underlying cause of dry AMD remains unknown; however, recent studies suggest that a decline in the biosynthesis of certain long chain and very long chain polyunsaturated fatty acids (LC and VLC PUFA) plays an important underlying role. The ELOVL2 enzyme plays a central role in the biosynthesis of LC and VLC PUFAs. During aging, expression of the ELOVL2 gene declines leading to a reduction in the LC and VLC PUFAs necessary for vision. VGX-0111 is an experimental gene therapy intended to increase expression of ELOVL2. It is thought that restoring physiological ELOVL2 expression will increase levels of LC and VLC PUFAs in the retina and slow or halt the progression of dry AMD.
The findings demonstrate that it is possible to increase the fatty acids whose decline is associated with dry AMD with a well-tolerated treatment in a species closely related to humans.
Studies in natural aging models of dry AMD in rodents have demonstrated that a single treatment with VGX-0111 increased ELOVL2 expression and protected against photoreceptor loss. The recently completed study investigated VGX-0111 in non-human primates as the next step toward human clinical trials. The studies showed VGX -0111 was well tolerated and expressed appropriately in the targeted areas of the retina. Most importantly, the study demonstrated a meaningful increase in the LC and VLC PUFAs whose deficit is associated with dry AMD.
“A robust body of scientific literature supports that long chain and very long chain polyunsaturated fatty acids play essential roles in both the vision transduction process and supporting the bioenergetics of retinal function,” stated Martin Emanuele, PhD, Chief Science Officer of Visgenx. “The findings demonstrate that it is possible to increase the fatty acids whose decline is associated with dry AMD with a well-tolerated treatment in a species closely related to humans. This is very encouraging and bodes well for further development.”
ABOUT VISGENX, INC.
Visgenx, Inc. is a biotechnology company focused on developing gene-based therapeutics for degenerative retinal diseases. Visgenx’ initial product is VGX-0111, a gene therapy candidate being developed for the treatment of dry Age-related Macular Degeneration (AMD). Close to 200 million people suffer from dry AMD globally; it is a leading cause of blindness. VGX-0111 is based on the ELOVL2 gene, which is required for the biosynthesis of lipids necessary for the function and survival of retinal cells. ELOVL2 expression declines with aging which may be an underlying pathology of dry AMD. VGX-0111 is intended to restore a normal level of ELOVL2 expression thereby slowing or halting the vision loss resulting from dry AMD. For more information on Visgenx, visit www.visgenx.com.
SOURCE: Visgenx
Post Views: 229
- A recently completed non-human primate study of VGX -0111 demonstrated good tolerability, provided strong transgene expression in the targeted region of the retina, and increased production of the lipids whose decline is associated with macular degeneration
- The Company is moving the development program forward with the intention of conducting a human proof of concept study
SAN DIEGO, CA, USA I June 27, 2023 I Visgenx, Inc., a biotechnology company focused on developing gene therapies for degenerative retinal diseases, today announced positive data from a non-human primate study. The important translational study used a single subretinal injection to deliver VGX-0111 (carrying an ELOVL2 transgene) to investigate whether VGX-0111 expresses in the target tissues and causes an increase in certain very long chain polyunsaturated fatty acids at a dose that is well tolerated. VGX-0111 was able to successfully demonstrate all three of these important parameters.
Dry age-related macular degeneration (AMD) is a leading cause of blindness. The underlying cause of dry AMD remains unknown; however, recent studies suggest that a decline in the biosynthesis of certain long chain and very long chain polyunsaturated fatty acids (LC and VLC PUFA) plays an important underlying role. The ELOVL2 enzyme plays a central role in the biosynthesis of LC and VLC PUFAs. During aging, expression of the ELOVL2 gene declines leading to a reduction in the LC and VLC PUFAs necessary for vision. VGX-0111 is an experimental gene therapy intended to increase expression of ELOVL2. It is thought that restoring physiological ELOVL2 expression will increase levels of LC and VLC PUFAs in the retina and slow or halt the progression of dry AMD.
The findings demonstrate that it is possible to increase the fatty acids whose decline is associated with dry AMD with a well-tolerated treatment in a species closely related to humans.
Studies in natural aging models of dry AMD in rodents have demonstrated that a single treatment with VGX-0111 increased ELOVL2 expression and protected against photoreceptor loss. The recently completed study investigated VGX-0111 in non-human primates as the next step toward human clinical trials. The studies showed VGX -0111 was well tolerated and expressed appropriately in the targeted areas of the retina. Most importantly, the study demonstrated a meaningful increase in the LC and VLC PUFAs whose deficit is associated with dry AMD.
“A robust body of scientific literature supports that long chain and very long chain polyunsaturated fatty acids play essential roles in both the vision transduction process and supporting the bioenergetics of retinal function,” stated Martin Emanuele, PhD, Chief Science Officer of Visgenx. “The findings demonstrate that it is possible to increase the fatty acids whose decline is associated with dry AMD with a well-tolerated treatment in a species closely related to humans. This is very encouraging and bodes well for further development.”
ABOUT VISGENX, INC.
Visgenx, Inc. is a biotechnology company focused on developing gene-based therapeutics for degenerative retinal diseases. Visgenx’ initial product is VGX-0111, a gene therapy candidate being developed for the treatment of dry Age-related Macular Degeneration (AMD). Close to 200 million people suffer from dry AMD globally; it is a leading cause of blindness. VGX-0111 is based on the ELOVL2 gene, which is required for the biosynthesis of lipids necessary for the function and survival of retinal cells. ELOVL2 expression declines with aging which may be an underlying pathology of dry AMD. VGX-0111 is intended to restore a normal level of ELOVL2 expression thereby slowing or halting the vision loss resulting from dry AMD. For more information on Visgenx, visit www.visgenx.com.
SOURCE: Visgenx
Post Views: 229
