Adverum has developed a broad and diverse pipeline of proprietary and partnered programs in gene therapy.
- Product Candidate, Indication
ADVM-022 and ADVM-032 are next-generation gene therapy product candidates optimized for intravitreal injection, which are being evaluated as treatments for age-related macular degeneration (wAMD) as well as other retinal conditions associated with VEGF over-expression. wAMD is the leading cause of visual deterioration and legal blindness in patients over 60 years of age, and affects ~1.2 million people in the US alone.1 Standard of care treatment involves injections of anti-VEGF proteins into the vitreal space, as often as every 4-8 weeks.
ADVM-022 and ADVM-032 utilize a novel vector allowing for intravitreal delivery of anti-VEGF cDNA to potentially treat wAMD as well as other retinal conditions associated with VEGF over-expression.
1Arch Ophthalmol. 2004;122(4):564-572. doi:10.1001/archopht.122.4.564
- ADVM-043 (Rare Disease)
ADVM-043 is our lead gene therapy product candidate designed to deliver alpha-1 antitrypsin (A1AT) protein in patients with alpha-1 antitrypsin deficiency. Compared to 25 other serotypes, AAVrh10 showed the highest level of hA1A1T expression in mice following intrapleural administration1. This form of administration also showed stable, long-term expression of hA1AT mRNA in primates2.
- ADVM-053 (Rare Disease)
ADVM-053 is our gene therapy product candidate designed for the treatment of patients with hereditary angioedema (HAE). ADVM-053 is an AAV based gene transfer vector that aims to provide persistent levels of human C1-esterase inhibitor (C1-INH) to potentially correct the deficiency state that can lead to life threatening attacks.
- Product Candidate, Indication
- AVA-311 (Ocular Disease)¹
AVA-311 is being developed for the treatment of Juvenile X-linked Retinoschisis (XLRS), an inherited retinal disease caused by mutations in the RS1 gene located on the X chromosome, therefore occurring almost exclusively in males. AVA-311 is comprised of an optimized AAV vector to deliver the RS1 gene into the eye via intravitreal injection.
In May 2014, Adverum signed a collaboration agreement with Regeneron that includes the development of AVA-311.
- Up to 5 Undisclosed Targets²
IndicationInherited Retinal Disease
¹ Collaboration agreement with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products (AVA-311 and up to 3 undisclosed targets) for ophthalmic diseases
² Collaboration agreement with Editas Medicines to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases
Through directed evolution, we generate a diverse library of millions of AAV variants and subsequently screen the variants in multiple in vitro and in vivo tests to identify the optimal variant for a specific disease. Our directed evolution technology allows us to create proprietary vectors and optimize them to target cells in tissues such as different layers of the retina. Each of these cell layers constitutes a potential therapeutic target for currently unmet medical needs, providing us with multiple opportunities to apply our directed evolution technology.
Our seasoned pharmaceutical development team has decades of gene therapy manufacturing experience. Adverum’s industrialized manufacturing process—based on our proprietary baculovirus expression system—is highly efficient and scalable, with production yields up to one hundred times greater than those obtained using conventional AAV production systems.
Our process enables us to manufacture commercial grade material for diseases with large patient populations. We have performed multiple single use bioreactor production / purification runs and have the ability to make highly concentrated purified vector.
Adverum collaborates with leading academic institutions and pharmaceutical companies who can augment our industry-leading expertise in gene therapy.
Please contact us for more information.
- High Levels of Persistent Expression of A1-Antitrypsin Mediated by the Nonhuman Primate Serotype rh.10 Adeno-associated Virus Despite Preexisting Immunity to Common Human Adeno-associated Viruses, Mol Ther Vol. 13, No. 1, January 2006,De et al.
- Intrapleural Administration of an AAVrh.10 Vector Coding for Human a1-Antitrypsin for the Treatment of a1-Antitrypsin Deficiency, HUMAN GENE THERAPY CLINICAL DEVELOPMENT 24:161–173 (December 2013), Chiuchiolo et al.
- Perdomini, M., (2014) Prevention and Reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s ataxia Nature Medicine Volume 20 (Number 5, May) 542-549
- Sharpe LT, Stockman A, Jagle H, Nathans J. Opsin genes, photopigments, color vision and color blindness. In: Gegenfurtner KR, Sharpe LT (eds.) Color Vision. Cambridge UP: Cambridge, 1999.
- Perdomini et al. Nature Med, April 2014 Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s Ataxia