Adverum Biotechnologies Introduces an Intravitreal Gene Therapy Program for Geographic Atrophy and Presents Data on Its Ocular Gene Therapy Platform
2023年5月18日 - 10:00PM
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 BiotechnologiesAdverum
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.
Forward-looking StatementsStatements 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
nonclinical data on an intravitreal gene therapy for the treatment
of geographic atrophy secondary to dry age-related macular
degeneration via expression of Complement Factor I, research
pipeline data supporting the utility of proprietary
adeno-associated virus vector platform in ocular gene therapy in
optogenetics and chemistry, manufacturing and controls strategies
for AAV-mediated gene therapies in highly prevalent diseases.
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
intravitreal gene therapies. 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 Quarterly Report on Form
10-Q for the quarter ended March 31, 2023 filed with the SEC on May
11, 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 ReddiVice President, Head of Corporate Strategy, External
Affairs and EngagementAdverum Biotechnologies, Inc.T:
650-649-1358E: areddi@adverum.com
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