ELOVL2

Charles River will manufacture for Visgenx's investigational gene therapy treatment for dry Age-Related Macular Degeneration SAN DIEGO, Dec. 7, 2023 /PRNewswire/ -- Visgenx, Inc., a biotechnology company focused on developing gene therapies for degenerative retinal diseases, announced an extension of their contract development and manufacturing organization (CDMO) collaboration with Charles River Laboratories International, Inc., for VGX-0111, a gene therapy being developed by Visgenx for dry age-related macular degeneration (AMD). Continue Reading "Manufacturing of gene therapies is highly complex and product quality is of paramount importance," said William Pedranti, CEO, Visgenx. "We have the utmost confidence in Charles River to manufacture for VGX-0111 as we work toward our goal to bring treatment for those suffering from dry-AMD." We have the utmost confidence in Charles River to manufacture for VGX-0111 Post this Following Visgenx's Type B pre-IND meeting with the FDA in the summer of 2023, Charles River will begin manufacturing materials for VGX-0111 with the goal of initiating a clinical proof-of-concept in 2025. The material manufactured by Charles River will support a good laboratory practice (GLP) pre-clinical toxicology study and follows a prior adeno-associated virus (AAV) production collaboration. "We are excited to expand our partnership with Visgenx on this gene therapy," said Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River. "The collaboration will tap into our premier gene therapy CDMO capabilities, and we are thrilled that our expertise will help to bring VGX-0111 one step closer to the clinic." What is Dry-AMD? Dry-AMD is a progressive eye disease that affects over 11 million people in the United States and is the leading cause of severe vision loss or blindness in adults over age 50. There is no known cure for dry-AMD and only limited treatments are available. Dysregulation of very long-chain fatty acid biosynthesis resulting from declining expression of a gene known as ELOVL2 is believed to be a contributor to dry-AMD. Numerous studies have elucidated the importance of ELOVL2 in maintaining the function and promoting survival of retinal cells and other tissues. The downregulation of ELOVL2 may lead to accelerated cellular aging and potentially form the foundational basis of dry-AMD. Visgenx's lead product, VGX-0111, a candidate gene therapy based on the ELOVL2 gene, is intended to restore a physiologically healthy level of ELOVL2 expression, thereby potentially slowing or halting vision loss resulting from dry-AMD. About Visgenx Visgenx, Inc. is a biotechnology company focused on developing gene-based therapeutics for degenerative retinal diseases. Visgenx's initial product is VGX-0111, an ELOVL2 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. For more information on Visgenx, visit . Forward Looking Statements This press release contains forward-looking statements related to Visgenx, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995, that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the potential for VGX-0111 as a treatment for dry-AMD. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements include that the therapy may not be effective at treating dry-AMD. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law. @ 2023 Visgenx, Inc. All Rights Reserved. CONTACT: William Pedranti, william@visgenx.com SOURCE Visgenx

Presentation will address the Role of Long Chain and Very Long Chain Polyunsaturated Fatty Acids in Vision to Better Inform Dry AMD Therapeutic Development SAN DIEGO, Nov. 14, 2023 /PRNewswire/ -- Visgenx, Inc., a biotechnology company focused on developing gene therapies for degenerative retinal diseases, today announced that the Company's Chief Science Officer and Co-Founder, Marty Emanuele, Ph.D., will present at the 4th Annual Dry AMD Therapeutic Development Summit on November 16th in Boston, Massachusetts. Continue Reading "It is rewarding to have been selected to present at this industry event focused on the latest developments in the investigational treatment of dry AMD," stated Dr. Emanuele. "I look forward to discussing the important role that fatty acids play in retinal cell health and how the decrease in these lipids may be contributing to the cause of dry AMD." "I look forward to discussing the important role that fatty acids play in retinal cell health...." Post this The Company's lead product VGX-0111, is a candidate gene therapy being developed for the treatment of dry AMD. VGX-0111 is based on the ELOVL2 gene, which is required for the biosynthesis of long chain and very long chain fatty acids necessary for the function and survival of retinal cells. ELVOL2 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. 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. ELVOL2 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 . Forward Looking Statements This press release contains forward-looking statements related to Visgenx, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995, that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the potential for VGX-0111 as a treatment for Dry AMD. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements include that the therapy may not be effective at treating Dry AMD. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law. @ 2023 Visgenx, Inc. All Rights Reserved. Contact: William Pedranti (949) 413-2672 SOURCE Visgenx

This issuance provides patent protection in the U.S. for therapeutics incorporating the optimized ELOVL2 transgene until 2041The patent is being prosecuted in the European Union and other countries around the globe SAN DIEGO, Oct. 25, 2023 /PRNewswire/ -- Visgenx, Inc., a biotechnology company focused on developing gene therapies for degenerative retinal diseases, today announced that the United States Patent and Trademark Office has issued U.S. Patent No. 11,793,890, entitled "ELOVL2 Constructs for Human Gene Therapy". The patent, which is exclusively owned by Visgenx, has an expiration date of October 19, 2041. Continue Reading "The ELOVL2 gene is important in that its expression is believed to be necessary for the survival of cells in the retina and other organ systems," stated Marty Emanuele, Ph.D., Co-Founder and Chief Science Officer at Visgenx. "However, it is also uniquely susceptible to aging related methylation of its promotor and down regulation. Thus, decline in its expression may play an underlying role in multiple aging related disorders including age-related macular degeneration. Accordingly, we believe that ELOVL2 gene therapy is an attractive strategy for treating these conditions." This issuance provides patent protection in the U.S. for therapeutics incorporating the optimized ELOVL2 transgene Post this "The issued patent covers an optimized ELOVL2 transgene that can be used in expression vectors targeting multiple tissues," said Chris Chavez, Ph.D., Vice President of Research and Development at Visgenx and primary inventor on the patent. "Therefore, we believe the issued patent adds substantial value not only to our lead program in dry AMD, but also provides the basis for a platform of ELOVL2 based therapeutics to treat aging related disorders in other tissues where it is highly expressed including the brain, liver and pancreas." 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, an ELOVL2 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. For more information on Visgenx, visit . Forward Looking Statements This press release contains forward-looking statements related to Visgenx, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995, that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the potential for VGX-0111 as a treatment for dry AMD and other conditions. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements include that the therapy may not be effective at treating dry AMD or other conditions. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law. © 2023 Visgenx, Inc. All Rights Reserved. Contact: William Pedranti (949) 413-2672 SOURCE Visgenx