Intergalactic Therapeutics to Present Preclinical Data Highlighting Potential of Its Non-Viral Gene Therapy Platform at the American Society of Gene and Cell Therapy 26th Annual Meeting

PR Newswire · 05/11/2023 11:00

Lead program, IG-002 targeting ABCA4 retinopathies demonstrated long-term, persistent protein expression over twelve months with a single administration

Newly developed in vitro efficacy model highlights ability of non-viral gene therapy platform to deliver large genetic payloads

Data support plan to advance IG-002 into the clinic in 2024

CAMBRIDGE, Mass., May 11, 2023 /PRNewswire/ -- Intergalactic Therapeutics, a company focused on transforming medicine through non-viral gene therapy, announced today that it will present two posters highlighting preclinical data for IG-002, Intergalactic's lead program addressing all forms of ABCA4-related retinopathies, at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held in Los Angeles, CA, from May 16-20, 2023.

Featured presentations will highlight data assessing a newly developed in vitro efficacy model demonstrating the potential of Intergalactic's integrated platform for delivery of full-length ABCA4 to retinal cells in vivo. The Company will also present preclinical in vivo results demonstrating, for the first time, that a single subretinal administration of a non-viral DNA payload encoding the complete human ABCA4 coding sequence resulted in durable, 12-month expression of human ABCA4 protein in adult porcine retinas. The expression levels achieved suggest potential therapeutic benefit in individuals living with ABCA4-related retinopathies.

In both studies, full-length ABCA4 DNA encoded into Intergalactic's proprietary C3DNA (covalently closed and circular DNA) cargo platform was delivered using COMET, Intergalactic's proprietary in vivo electro-transfer based system for targeted delivery of C3DNA.

"We are excited to share encouraging preclinical data from our lead program, IG-002, as well as our non-viral gene therapy platform with the cell and gene therapy community at ASGCT," said Theresa G.H. Heah, M.D., M.B.A., Chief Executive Officer of Intergalactic Therapeutics. "Together, these findings highlight the potential of our proprietary non-viral C3DNA + COMET platform to deliver large genetic payloads and enable persistent and durable gene therapies to address previously intractable disorders. We are working diligently to advance IG-002 towards an IND filing in the first half of 2024 as we seek to revolutionize treatment for patients with few or no therapeutic options."

Details for the presentations are as follows:

Poster Title: COMET-mediated delivery of C3DNA in human cell-based in vitro models of retinal pigment epithelium 

Presenter: Rachelle Prantil-Baun, Ph.D., Director, Head of Tissue Targeting at Intergalactic Therapeutics 

Abstract Presentation Number: 714 

Session: Wednesday Poster Session 

Date and Time: Wednesday, May 17, 2023 at 12:00 p.m. PDT 

Location: Los Angeles Convention Center, Los Angeles, CA

Poster Title: Non-viral delivery of ABCA4 to photoreceptors 

Presenter: Gayathri Ramaswamy, Ph.D., Vice President, Drug Discovery and Disease Biology at Intergalactic Therapeutics 

Abstract Presentation Number: 1512 

Session: Friday Poster Session 

Date and Time: Friday, May 19, 2023 at 12:00 p.m. PDT 

Location: Los Angeles Convention Center, Los Angeles, CA

About ABCA4-Related Retinopathies

ABCA4-related retinopathies, including Stargardt disease, cone-rod dystrophy, and retinitis pigmentosa, lead to degeneration of rods and cones and cause vision loss that often starts in childhood or adolescence, ultimately leading to blindness. These diseases are associated with mutations in the ABCA4 gene, more than 900 of which have been described. There are currently no approved treatments for ABCA4-related retinopathies and, while replacement with wild-type ABCA4 protein has the potential to slow or stop disease progression, standard AAV gene therapy platforms are not appropriate for addressing these diseases due to the large size of the gene and the growing safety concerns associated with viral vectors. Intergalactic's novel platform bypasses these drawbacks and enables local delivery of non-viral gene therapy, representing a potentially transformative approach for addressing these blinding diseases. 

About Intergalactic Therapeutics

Intergalactic Therapeutics, an Apple Tree Partners (ATP) company, is transforming medicine through non-viral gene therapy. Its proprietary integrated platform comprises three core pillars: versatile C3DNA technology (covalently closed and circular DNA); groundbreaking precise and tunable approaches to local therapeutic delivery using the COMET electro-transfer system; and a rapid, scalable, and cost-effective manufacturing process to make gene therapy safer and more accessible. With a diversified portfolio of potential new treatments for eye diseases and beyond, Intergalactic is dedicated to helping patients around the world by bringing non-viral gene therapies into reality. For more information, visit

Cision View original content to download multimedia:

SOURCE Intergalactic Therapeutics