Adverum Biotechnologies Presents Research Pipeline Data Supporting Utility of its Proprietary Platform and AAV.7m8 Capsid in Ocular Gene Therapy
May 19 2022 - 8:00AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today will announce new research pipeline data supporting
the utility of its proprietary adeno-associated virus (AAV) vector
platform in ocular gene therapy. These new data will be featured in
oral presentations during the American Society of Gene and Cell
Therapy (ASGCT) 2022 Annual Meeting in Washington, D.C. and
virtually.
“Adverum is an industry leader in the development of
adeno-associated virus ocular gene therapy, including cassette
engineering and vectorizing therapeutic proteins, and we are
pleased to have multiple presentations highlighting our platform at
ASGCT. As we continue to prepare for the initiation of a Phase 2
trial of ADVM-022 for wet AMD in the third quarter of 2022, we are
also advancing other research programs toward the clinic and
expanding our pipeline in ocular gene therapy by building on the
potential of a single in-office intravitreal injection with our
proprietary AAV.7m8 capsid,” said Brigit Riley, Ph.D., chief
scientific officer at Adverum Biotechnologies. “We are excited to
present non-clinical data on ADVM-062 (AAV.7m8-L-opsin) for blue
cone monochromacy, which received Orphan Drug Designation by the
U.S. Food and Drug Administration in January 2022, and continue to
advance this program toward an investigational new drug application
submission. Adverum continues the technical advances of our
in-house adeno-associated virus manufacturing processes. Finally,
we are maturing a portfolio of proprietary vectors with specific
ocular cell tropism and are excited to showcase our innovative work
on LSV1, a novel capsid for ocular gene therapy.”
ADVM-062 for Blue Cone
Monochromacy (BCM) Data Highlights
- ADVM-062 was well tolerated at all doses tested
- No observed adverse effect level for the study was 3x10^11
vg/eye dose in non-human primate (NHP)
LSV1 Data Highlights
- Identified LSV1 from a library
screen in NHP
- LSV1 has a unique 3D structure at
the 3-fold axis imparting new structural characteristics from
parental backbone
- LSV1 efficiently transduces NHP
retina from the vitreous
- High expression in the fovea and
periphery, as well as retinal pigment epithelium expression
About Blue Cone Monochromacy
BCM is an X-linked recessive hereditary condition caused by the
absence of function in the L and the M opsin genes and can manifest
in loss of visual acuity, photosensitivity, myopia and infantile
nystagmus that can persist into adulthood. Consequently,
individuals with BCM have visual impairments to important aspects
of daily living such as facial recognition, learning, reading, and
daylight vision. Currently, BCM affects approximately 1 to 9 in
100,000 males, worldwide and there is no cure for BCM.
About ADVM-062 Gene Therapy
ADVM-062 (AAV.7m8-L-opsin) is a novel gene therapy product
candidate being developed to deliver a functional copy of the
OPN1LW gene to the foveal cones of patients suffering from blue
cone monochromacy (BCM) via a single IVT injection. ADVM-062
utilizes Adverum’s propriety vector capsid, AAV.7m8. In January
2022, the FDA granted Orphan Drug Designation to ADVM-062.
About Adverum Biotechnologies
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene
therapy company targeting unmet medical needs in serious ocular and
rare diseases. Adverum is evaluating its novel gene therapy
candidate, ADVM-022, as a one-time, intravitreal injection for the
treatment of patients with neovascular or wet age-related macular
degeneration. For more information, please visit
www.adverum.com.
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. 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; 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 ADVM-022. 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, 2022 filed with
the SEC on May 12, 2022. 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.
Inquiries
Anand ReddiVice President, Head of Corporate Strategy and
External Affairs & EngagementAdverum Biotechnologies, Inc.T:
650-649-1358
Investors
Laurence WattsGilmartin GroupT: 619-916-7620E:
laurence@gilmartinir.com
Media
Megan TalonAssociate Director, Corporate CommunicationsAdverum
Biotechnologies, Inc.T: 650-649-1006E: mtalon@adverum.com
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