Cell and Gene Therapy

The DNA donor molecule is the genetic blueprint for gene knock-ins in combination with CRISPR (clustered short interspaced palindromic repeats)-Cas9 or other nucleases. While commercial products for small gene-size knock-ins (2 kilobases or less) are available, efficient and scalable solutions for the integration of larger payloads carrying several genes or entire genetic circuits are currently lacking. Stellate DNA commercializes GatalystsTM (genome catalysts), a novel class of DNA donors that are based on our patented innovation to use circular single stranded DNA in gene editing. GatalystsTM enable large genome integrations (up to 20 kilobases) to be precisely targeted and minimize cytotoxicity in cell transfections. In addition, Stellate’s proprietary production platform allows scalable GatalystsTM production with milligram to gram yields and up to 100-times cost reduction when compared to competing DNA donor products. This cost reduction allows cell therapies like CAR-T immunotherapy to be delivered to patients faster and cheaper than with current state-of-the-art viral vehicles and without safety issues inherent to viruses and payload restriction by capsid sizes. The combined benefit of both production and use of GatalystsTM enables ultra-large DNA payload integrations in a precise manner which is truly transformational in gene and cell engineering.