Adverum Biotechnologies Introduces an Intravitreal Gene Therapy Program for Geographic Atrophy and Presents Data on Its Ocular Gene Therapy Platform

2023-05-19

基因疗法

- Geographic atrophy (GA) program introduced with complement Factor I (CFI), a key component of the complement cascade, as a payload and utilizing two intravitreal (IVT) capsids, 7m8 and LSV1 -

- IVT administration of AAV-CFI using either the 7m8 or LSV1 capsid resulted in high expression levels and was well tolerated in non-human primates (NHPs) -

- Presented data on an optogenetics program utilizing an engineered melanopsin mutant that could serve as a therapeutic transgene for optogenetic vision restoration in indications with photoreceptor loss such as advanced GA, among others -

- Presented manufacturing strategies for AAV-mediated gene therapies in highly prevalent diseases -

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 presented new nonclinical data on an IVT gene therapy for the treatment of GA secondary to dry age-related macular degeneration (dry AMD) via expression of CFI at the American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting, in Los Angeles, California. Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.

“The ability to deliver both CFI and an engineered melanopsin leveraging our proprietary capsids via IVT administration demonstrates the strength of our platform at Adverum,” commented Brigit Riley, Ph.D., chief scientific officer of Adverum Biotechnologies. “Similar to wet AMD, patients with GA are forced either to receive frequent, often monthly, injections or to experience faster lesion growth and vision deterioration. We’re in the early stages of development for a GA therapeutic and are excited by the potential for a paradigm shift for dry AMD patients worldwide with a single, in-office, IVT injection creating CFI biofactories in the retina.”

Geographic Atrophy Poster Highlights

GA is a highly prevalent disease characterized by retinal pigment epithelium (RPE) and photoreceptor death. The inhibition of components of the complement pathway has been shown to meaningfully reduce GA lesion growth.

CFI, a rate-limiting enzyme within the complement cascade, naturally blocks the activity of proteins involved in complement overactivation.

Continuous expression of CFI in ocular tissue holds the possibility to inhibit complement overactivation, thereby halting GA lesion growth and preserving vision for dry AMD patients.

7m8 and LSV1 capsids packaged with AAV-CFIco, yielded robust intraocular human CFI levels in NHPs.

IVT administration of AAV-CFIco via both proprietary capsids was well tolerated, with no anti-inflammatory steroids used at any timepoint in the nonclinical study.

Administering an AAV-mediated therapy to express CFI in patients via IVT delivery, a routine in-office procedure, could be an ideal treatment profile for a widely adoptable treatment for GA.

Optogenetics Poster Highlights

In a poster presentation exploring an optogenetic approach to vision restoration, Adverum presented data on an engineered melanopsin that demonstrated improved kinetics, including speed and light sensitivity, and that may have utility as a therapeutic transgene for optogenetic vision restoration. Melanopsin through its unique ability to regenerate chromophore has the potential to be an effective light sensor candidate by generating pseudo-photoreceptors for optogenetic vision restoration.

Chemistry, Manufacturing and Controls (CMC) Poster Highlights

In a poster presentation examining CMC strategies for AAV gene therapies, Adverum presented data demonstrating that improved bacmid purity enables lower passage number and higher gene of interest stability, and that manufacturing process changes, such as adding Sf-RVN cells with optimal media (rather than Sf9 cells) or the use of low MOI for rAAV production, can consistently yield higher titer rAAV.

The ASGCT poster and oral presentations will be made available on the Publications page of the Adverum website.

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 www.adverum.com.

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