SQZ Biotech and AskBio collaborate to overcome AAV immunogenicity


Patients’ immune systems generating neutralising antibodies toward therapeutic AAVs is one of gene therapy’s biggest challenges and SQZ's proprietary technology proposes tolerising antigen carriers

Image as seen on SQZ Biotechnology's website showing its proposed process

SQZ Biotechnologies and Asklepios BioPharmaceutical (AskBio) have formed a research collaboration to create tolerising antigen carriers (TACs) containing AAV (adeno-associated virus) components to solve one of gene therapy’s biggest challenges – the barrier to treatment posed by patients’ immune systems generating neutralising antibodies toward therapeutic AAVs.

SQZ and AskBio will combine their proprietary cell and gene therapy platform technologies to address this issue.

Gene therapies utilising AAV vectors can be transformative for patients with genetic diseases, but neutralising antibodies can prevent large populations of patients from benefitting from AAV gene therapies.

Patients’ immune systems develop neutralising antibodies after receiving their first dose of AAV, or they can be pre-existing. This collaboration will strive to give these patients access to novel therapeutics and enable them to take multiple or repetitive doses to gain the full, durable benefit these treatments can provide.

The technology

The collaboration will combine the proprietary cell and gene therapy platform technologies of both companies and will evaluate the administration of SQZ TACs and AskBio’s gene therapies to potentially address AAV immunogenicity. SQZ is a pioneer in cell therapy, and the company’s knowledge and expertise, as well as their capabilities in manufacturing, are critical to this collaboration’s approach to synergising cell and gene therapies.

Preclinical data from SQZ has demonstrated that SQZ TACs specifically inhibit undesired immune responses in multiple contexts, including AAV models. As a leader in the AAV field, AskBio brings expertise in AAV technology, capsid design, clinical processes and manufacturing that would allow for application of these novel methods to overcome immunogenicity. The two companies have a shared goal to increase world-wide access of transformative therapeutics. Sign up for your free email newsletter

R Jude Samulski, Chief Scientific Officer and co-founder of AskBio, said: “Addressing AAV immunogenicity is essential to the future of gene therapy as it is one of the most significant limiting factors plaguing the gene therapy space today.”