CRISPR Therapeutics, a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Casebia Therapeutics, a joint-venture established by CRISPR Therapeutics and Bayer for developing CRISPR-based therapeutics in select disease areas, have signed a collaboration agreement with StrideBio, a U.S.-based company developing novel AAV vectors for in vivo gene delivery applications.

Under the terms of the agreement, StrideBio will use its proprietary platform to develop AAV vectors with improved properties such as tissue specificity and reduced susceptibility to immune responses. CRISPR Therapeutics and Casebia will have an option to exclusively license AAV vectors with desired properties for use in their in vivo gene-editing programs. StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

“Beyond our ex vivo lead programs, we see tremendous potential to apply our CRISPR/Cas9 platform to diseases requiring in vivo delivery. StrideBio has a unique platform that can identify next-generation AAV vectors which are much more effective than those currently in use. This partnership positions us at the forefront of viral delivery technology for our in vivo programs,” said Samarth Kulkarni, Chief Business Officer of CRISPR Therapeutics.

StrideBio’s AAV vector development platform makes use of structure-guided evolution to discover novel capsids with improved properties for in vivo gene delivery applications. The underlying technology is based on the work of Dr. Aravind Asokan at the University of North Carolina at Chapel Hill and Dr. Mavis Agbandje-McKenna at the University of Florida. 

“This collaboration is another example of how CRISPR Therapeutics and Casebia can partner to improve our shared gene editing platform. Casebia has a focus on in vivo gene editing in the liver, eye and heart where AAV vectors are a promising delivery modality, and where StrideBio’s improved vectors can advance our ability to develop effective therapeutics,” said Jim Burns, president and CEO of Casebia.

“We founded StrideBio to meet the challenge of pre-existing immunity in patients. Building on our deep understanding of how neutralizing antibodies interact with different AAV serotypes, we have established a unique technology platform that will allow us to evolve advanced AAV vectors that are both resistant to pre-existing immunity and have improved tissue specificity. This collaboration with CRISPR Therapeutics and Casebia will allow us to greatly accelerate our progress in that area,” said Aravind Asokan, co-founder of StrideBio.