Our Focus

We are driven above all by the unprecedented opportunity to transform the lives of patients and families affected by a wide array of genetic diseases. Learn about how we are harnessing the power of gene editing to create fundamental breakthroughs for patients and families.

Development Programs

The Potential Benefits of CRISPR/Cas9

Pipeline at-a-Glance

Pipeline at-a-Glance

Pipeline at-a-Glance

Gene editing: the future

Casebia is dedicated to leading the gene editing and genetic engineering revolution today, and over the long term, advancing the CRISPR technologies of tomorrow. Together with our partners, we are working to optimize gene editing across multiple disciplines and create the therapies that will unlock the full potential of gene editing for patients:

  • Cas9 protein: Modification of multiple characteristics of the Cas9 protein to improve specificity, efficiency, and ease of deliverability
  • guide RNA selection: Combining bioinformatics and experimental assays to identify guide RNAs with high efficiency and no off-target cutting
  • Delivery of CRISPR/Cas9 to target cells: Developing next-generation delivery technologies to increase delivery efficiency and access additional organ systems
  • Efficiency of correction: Achieving high efficiency of correction across all cell types and enable new therapeutic strategies
  • Cellular engineering: Improving the ex vivo cell collection, manipulation and administration process for a variety of stem and hematopoietic cell types

Statement on Heritable Genome Editing

Casebia is focused on using gene editing technologies to develop treatments for diseases by the editing of somatic cells where there is a clearly favorable benefit-risk ratio to ethically justify the treatment. We support the position of the National Academies of Sciences, Engineering and Medicine, which last year put out a report entitled “Human Genome Editing: Science, Ethics and Governance.” The Academies recommended that “clinical trials using heritable genome editing should be permitted only within a robust and effective regulatory framework,” and goes on to list 10 specific conditions necessary to meet that threshold.