Lead program, IG-002 targeting ABCA4 retinopathies, demonstrates durable 12-month expression of human ABCA4 protein using a single subretinal dose of DNA payload
CAMBRIDGE, Mass., April 20, 2023 /PRNewswire/ -- Intergalactic Therapeutics, a company focused on transforming medicine through non-viral gene therapy, announced today that Gayathri Ramaswamy, Ph.D., Vice President, Drug Discovery and Disease Biology, will present at the Annual Retinal Cell and Gene Therapy Innovation Summit on April 21, 2023 at the Marriott New Orleans Warehouse Arts District in New Orleans, Louisiana.
"We are excited to share encouraging preclinical data from our lead program, IG-002 targeting ABCA4-associated retinopathies. Data to be presented demonstrate for the first time that a non-viral gene therapy is able to achieve long-term, persistent protein expression with a single administration," said Theresa G.H. Heah, M.D., M.B.A., Chief Executive Officer of Intergalactic Therapeutics. "We believe that our C3DNA platform holds the potential to make non-viral gene therapies a reality, and with these data in hand, we are working to rapidly advance IG-002 through IND-enabling studies and into the clinic."
Presentation details are as follows:
Presentation Title: A novel, non-viral approach to delivering full length ABCA4 to photoreceptors
Date and Time: Friday, April 21, 2023 at 10:45 a.m. to 11:00 a.m. CT
Presenter: Gayathri Ramaswamy, Ph.D., Vice President, Drug Discovery and Disease Biology at Intergalactic Therapeutics
Dr. Ramaswamy will share preclinical results from Intergalactic's lead program IG-002 addressing all forms of ABCA4-related retinopathies. The data demonstrate for the first time that a single subretinal administration of a DNA payload encoding the human ABCA4 gene 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 the study, full-length ABCA4 DNA was packaged into Intergalactic's proprietary C3DNA (covalently closed and circular DNA) cargo platform, and was delivered using COMET, Intergalactic's proprietary electro-transfer based system for targeted delivery of C3DNA.
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 www.intergalactictx.com.
SOURCE Intergalactic Therapeutics
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