SQZ Biotechnologies, and Asklepios BioPharmaceutical (AskBio), announced a research collaboration to create tolerizing 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 neutralizing antibodies toward therapeutic AAVs. SQZ and AskBio will combine their proprietary cell and gene therapy platform technologies to open the door to new treatment paradigms with potential impact across many genetic diseases.
Gene therapies utilizing AAV vectors can be transformative for patients with genetic diseases, but neutralizing antibodies can prevent large populations of patients from benefitting from AAV gene therapies. Patients' immune systems develop neutralizing 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. Expanding patient eligibility and allowing repeat treatment could change the future of how products are developed and significantly impact the long-term health of millions in need.
"This is a tremendous opportunity to bring together the power of both cell and gene therapy for patients. AskBio has been an innovative leader in gene therapy and shares our patient-centric philosophy. By working together and leveraging the potential of both our platforms, we hope to bring more effective, more durable treatments to patients suffering from devastating rare genetic disorders," said Armon Sharei, PhD, founder and chief executive officer of SQZ Biotech.
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The collaboration between SQZ and AskBio will evaluate the administration of SQZ TACs and AskBio's gene therapies to potentially address AAV immunogenicity. 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.
"AskBio is firmly committed to improving the lives of underserved patients, such as those suffering from Pompe, Huntington's and various neuromuscular and central nervous system diseases,” R. Jude Samulski, PhD, 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. SQZ's pioneering approach to tolerance could offer a solution to this problem."
"Our collaboration with SQZ is exemplary of our goal to broadly explore potential redosing of AAV gene therapies," said Sheila Mikhail, chief executive officer and co-founder of AskBio. "We are thrilled to be working with SQZ and are hopeful that this initial research collaboration utilizing two of the most promising therapeutic modalities currently available, cell and gene therapy, will ultimately provide options to improve patients' immune response to gene therapy."