Adverum Biotechnologies Announces Upcoming Data Presentations at ASGCT 2023 including Data on a New Intravitreal (IVT) Program for Geographic Atrophy Secondary to Dry AMD

May 2, 2023

REDWOOD CITY, Calif., May 02, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced new data will be presented during the American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting being held May 16-20, 2023, in Los Angeles, California.

At ASGCT 2023, Adverum will unveil new nonclinical data on the development of an IVT gene therapy for the treatment of geographic atrophy through the overexpression of Complement Factor I (CFI). CFI is a rate-limiting enzyme in the complement cascade, naturally inhibiting the activity of proteins involved in complement overactivation. Continuous overexpression of CFI in ocular tissue inhibits the complement cascade in the eye and has the potential to halt lesion growth and preserve vision in patients with geographic atrophy secondary to age-related macular degeneration. Adverum designed and evaluated an optimized human CFI cassette using its two proprietary capsids 7m8 and LSV1 enabling IVT delivery. Both capsids demonstrated potent CFI levels in nonhuman primates (NHP), suggesting potential for continuous therapeutic levels of CFI in humans with a potential one-and-done outpatient IVT injection.  

Adverum will also deliver data presentations on Ixo-vec (ixoberogene soroparvovec, formerly ADVM-022) currently being evaluated in the Phase 2 LUNA trial in wet age-related macular degeneration (wet AMD). The first of two oral presentations will feature nonclinical data in NHP that support staggered bilateral administration of Ixo-vec in the treatment of wet AMD. Bilateral treatment is an area of unmet need where incidence of neovascularization (nAMD) in the second eye is up to 42% in the first two to three years following diagnosis in the primary eye. The second oral presentation will focus on identification of dose-dependent inflammation pathways involved in innate and adaptive immune response that are commonly observed in gene therapy. The data support the 2x10^11 vg/eye (2E11) and 6x10^10 vg/eye (6E10) doses in combination with enhanced corticosteroid prophylactics regimens in the ongoing Phase 2 LUNA trial. Additionally, a poster presentation will focus on Ixo-vec’s evaluation of per cell vector genome biodistribution and mRNA expression in NHP.

Adverum will also present new data in an Optogenetics program, as well as Adverum’s adeno-associated virus (AAV) advanced chemistry, manufacturing, and controls (CMC) process development platform for highly prevalent ocular diseases.

Adverum abstracts being presented at ASGCT are as follows:

Oral Presentation Title: Bilateral Ixo-vec NHP Tolerability and Efficacy Following a Staggered Dosing Interval between Eyes - Gene Therapy nAMD
Abstract Number: 76
Session: AAV Vectors: Clinical and Non-Human Primate Studies
Date & Time: Wednesday, May 17, 2023, 4:15 pm - 4:30 pm PDT
Presenter: Kris Poulsen

Oral Presentation: Identification of Dose-Dependent Immune Landscape Signatures Following Administration of Ixo-vec in Non-Human Primates
Abstract Number: 139
Session: Understanding and Modeling Immune Responses to Gene Therapy and Vaccines
Date & Time: Thursday, May 18, 2023, 2:00 pm - 2:15 pm PDT
Presenter: Julian Ramos

Poster Presentation: CMC Strategies for AAV Gene Therapies for Highly Prevalent Diseases
Abstract Number: 423
Session: Wednesday Poster Session
Date & Time: Wednesday, May 17, 2023, 12:00 pm PDT
Presenter: Ali Aghajanirefah

Poster Presentation: Engineered Melanopsin Mutants for Optogenetic Vision Restoration
Abstract Number: 711
Session: Wednesday Poster Session
Date & Time: Wednesday, May 17, 2023, 12:00 pm PDT
Presenter: Cameron Baker

Poster Presentation: Development of an Intravitreal (IVT) Gene Therapy for Geographic Atrophy (GA) by Overexpressing Complement Factor I (CFI) to Inhibit the Complement Cascade
Abstract Number: 976
Session: Thursday Poster Session
Date & Time: Thursday, May 18, 2023, 12:00 pm PDT
Presenter: Gustavo de Alencastro

Poster Presentation Title: Intravitreal Delivery of Ixo-vec for Neovascular Age-Related Macular Degeneration Results in Widespread Expression of Aflibercept in Cynomolgus Monkey Eyes as Confirmed Using In-Situ Hybridization
Abstract Number: 1557
Session: Friday Poster Session
Date & Time: Friday, May 19, 2023, 12:00 pm PDT
Presenter: Julio Nieves

About Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding the potential benefits for staggered, bilateral administration of Ixo-vec in the treatment of wet AMD. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of Ixo-vec. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on March 30, 2023. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Corporate, Investor and Media Inquiries

Anand Reddi
Vice President, Head of Corporate Strategy, External Affairs and Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358

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Source: Adverum Biotechnologies, Inc.