Pipeline

Adverum is advancing a pipeline of gene therapy product candidates targeting unmet medical need in ocular and rare diseases.

Programs

Partnered Programs

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

Directed Evolution

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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.

Manufacturing

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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.

Collaborations

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. Under an agreement with Editas we are leveraging our AAV-vectors for use with Editas’ leading Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based genome editing technologies to treat up to five inherited retinal diseases. 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.