Casebia Advances Genetic Engineering with New Tools and Techniques

“Our scientific team and collaborators have made extraordinary progress in the two years since our labs in Cambridge and San Francisco became fully functional,” said Jim Burns, Ph.D., CEO of Casebia. “These advances put new tools in the toolbox of our scientists as we work to create life-altering treatments for a variety of genetic diseases.”

In particular, Casebia and its collaborators will be highlighting the following advances during the 2019 ASGCT Annual Meeting:

• A new way of treating retinitis pigmentosa with an adeno-associated virus delivery system that has the ability to self-inactivate after delivering its payload, reducing potential immunogenicity and off-target effects.
• A suite of synthetic RNA-guided nucleases designated Cas-sRGNs™ (pronounced “Cas-surgeons”). The new nucleases exhibit properties that are anticipated to facilitate in vivo genome editing applications.
• An effective method for delivery of Cas-sRGNs™ into liver tissue using lipid nanoparticles (LNPs) that potentially enables better treatment of liver-based diseases via genetic engineering.
• A novel approach for transforming effector T cells, which promote autoimmune responses, into regulatory T cells that suppress inflammation and disease. These gene-edited, regulatory-like T cells (edTreg) will be evaluated as a therapy for IPEX syndrome and other autoimmune disorders linked to deficient regulatory T-cell function.

“Each of these advances have the potential to improve treatments currently under development for patients suffering from genetic diseases for which there are no adequate treatments,” said Andrew Scharenberg, M.D., Chief Scientific Officer of Casebia. “The research that will be presented at ASGCT underscores our scientific capabilities and our unique approach as we move towards clinical trials of our own product candidates.”

In total, the ten abstracts accepted at the conference each contain a significant advance in the company’s proprietary science. The abstracts can be viewed as part of the 2019 ASGCT Abstract Book or at https://casebia.com/news/#presentations.

About Casebia Therapeutics
Casebia Therapeutics is a novel joint venture between Bayer and CRISPR Therapeutics, formed in 2016 to advance CRISPR/Cas9 gene-editing therapeutics to discover and develop curative treatments for patients suffering from genetic diseases of the eye, ear and blood, as well as autoimmune, metabolic and cardiovascular conditions. The Casebia technology platform is moving genetic medicine forward by enabling new genetic engineering tools and revolutionary delivery methods for gene editing that target treatments precisely to specific areas of the body affected by disease. Casebia’s headquarters are in Cambridge, MA, with a research center in San Francisco, CA. For more information, please visit www.casebia.com or follow us at on Twitter @casebiatx.

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