Driving the Next Wave of Gene Therapy Innovation
The first wave of gene therapy innovation brought forth gene therapies that used naturally occurring AAVs, leading to two FDA-approved gene therapies. AAV remains the preferred delivery vector for genetic payloads – however, naturally occurring AAVs utilizing strong, ubiquitous promoters face limitations in biodistribution, off-target gene expression, pre-existing immunity, and manufacturability that limit their impact to a narrow set of diseases.
To overcome these limitations, we are transforming the current random trial-and-error approach into a data-driven process that guides simultaneous optimization of multiple characteristics. By addressing the current limitations in gene delivery and expression, we aim to unlock new indications for gene therapy. Our capabilities in engineering AAV capsids, regulatory elements, and payloads uniquely position us to develop best-in-class genetic medicines.
We are committed to making gene therapy a possibility for all patients with limited options for treating their disorder. This ambitious undertaking requires strong collaborations and partnerships to be successful. To accelerate our impact, we strive to work closely with biopharma and academic partners, patients, and disease foundations. Let’s build a future where the gene therapy modality is a standard in the armamentarium for many diseases.