Our Purpose

Boldly Innovating to Unlock the Full Potential of Gene Therapy for Countless Patients

GUIDING PRINCIPLES

• Dare to Cure – Patients are waiting. Create big dreams for patients and take calculated risks to achieve them
• Break Boundaries – Question the status quo and innovate beyond conventional approaches
• Beyond Yourself – It’s about patients, their families, our team, our families. We strengthen each other.
• Prepare and Execute Relentlessly – Start with the end in mind, over-prepare and make contingency plans to your contingency plans

Our Strategy

To achieve our goal of unlocking the full potential of gene therapy for many patient populations, we strive to achieve the following:

• Develop transformative precision gene therapies using the power of directed evolution and our “disease first” approach.
• Design, develop and commercialize a diverse portfolio of product candidates in a broad range of therapeutic areas for both rare and large market diseases.
• Continue to expand our cGMP manufacturing capabilities.
• Protect, defend and expand our intellectual property portfolio of proprietary AAV vectors discovered through directed evolution or Therapeutic Vector Evolution.
• Selectively partner with leading pharmaceutical companies to harness the full potential of our platform while retaining product rights in key markets, and work closely with patient advocacy groups to inform our programs.