Adverum Biotechnologies Presents Research Pipeline Data Supporting Utility of its Proprietary Platform and AAV.7m8 Capsid in Ocular Gene Therapy
Adverum Biotechnologies (Nasdaq: ADVM) presented promising data on its gene therapy pipeline at the 2022 ASGCT Annual Meeting. Highlights include advancements in ADVM-062 for blue cone monochromacy, which has achieved Orphan Drug Designation, and progress towards a Phase 2 trial of ADVM-022 for wet AMD. New proprietary AAV vectors, including LSV1, were showcased for their efficiency in transducing ocular cells. The company is focused on enhancing its AAV manufacturing processes and expanding its research into ocular gene therapy options.
- ADVM-062 has received Orphan Drug Designation from the FDA.
- ADVM-062 demonstrated good tolerance at all tested doses.
- LSV1 shows high efficiency in transducing NHP retina, indicating strong potential for ocular applications.
- None.
REDWOOD CITY, Calif., May 19, 2022 (GLOBE NEWSWIRE) -- 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 Reddi
Vice President, Head of Corporate Strategy and External Affairs & Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358
Investors
Laurence Watts
Gilmartin Group
T: 619-916-7620
E: laurence@gilmartinir.com
Media
Megan Talon
Associate Director, Corporate Communications
Adverum Biotechnologies, Inc.
T: 650-649-1006
E: mtalon@adverum.com
FAQ
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