Adverum is advancing a pipeline of gene therapy product candidates targeting unmet medical need in ocular and rare diseases.
- Product Candidate, Indication
For wet AMD, we are advancing our clinical gene therapy candidate ADVM-022 (AAV.7m8-aflibercept). ADVM-022 uses a proprietary capsid (AAV.7m8) to deliver a proprietary expression cassette which expresses aflibercept. ADVM-022 is administered as a single intravitreal injection and is designed to minimize the treatment burden of repeated anti-VEGF injections, which is the current standard of care for the treatment of wet AMD.
In preclinical non-human primate (NHP) studies, a single intravitreal administration of ADVM-022 provided sustained expression of aflibercept for at least two years at levels comparable to those experienced three to four weeks post-injection of aflibercept protein. Further, in a laser-induced choroidal neovascularization model in NHPs, the industry standard for testing new wet AMD therapies model, a single intravitreal injection of ADVM-022 13 months before lasering provided the same level of protection from clinically relevant lesions as an intravitreal bolus of aflibercept at the time of lesioning, the current standard of care.
This therapy utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is designed to provide sustained therapeutic levels of aflibercept, minimize the burden of frequent anti-VEGF injections, and improve real-world vision outcomes for patients.
- Rare Disease
- 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.
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
To create next-generation vectors, we use a multi-step process known as directed evolution. Our directed evolution technology uses a library of engineered AAV capsid genes, which exhibit different properties and capabilities than naturally occurring AAVs. Once we have created an initial pool of millions of different AAVs, we screen the AAVs in the pool for novel properties, e.g., specific transduction of a particular cell type, or the capability to evade pre-existing neutralizing immune response. Once capsids with desirable properties are identified, those capsids are screened to create a smaller pool of optimized vectors which are further screened until we have identified a select number of engineered AAVs with the characteristics we seek.
Our AAV vector manufacturing process is based on the Baculovirus Expression Vector System (BEVS), which has been used in a number of FDA- and EMA-approved products. This approach is well suited for the production of large quantities of AAVs, as it takes advantage of the efficiency of viral infection coupled with the high density and scalability of insect cells grown in serum-free suspension cultures. Compared to the mammalian cell-based approaches commonly used in the field, our manufacturing process is designed to produce higher yields of vectors per manufacturing campaign in a cost-effective manner.
Our AAV manufacturing method is industrialized, highly scalable and ready for adaptation for commercial stage.
Adverum collaborates with leading biopharmaceutical companies and academic institutions to advance the potential of gene therapy for treating patients.
Our partnered programs include vectors we are developing under collaboration agreements. Our agreement with Regeneron provides for development of up to eight distinct ocular therapeutic targets, which includes AVA-311 for the treatment of juvenile X-Linked Retinoschisis (XLRS).
Our current collaborators include:
We are open to exploring additional collaboration opportunities and encourage you to contact us for more information.