Our Approach
Our Approach 2018-02-22T08:58:51+00:00

Our vision is to be a global leader in AAV-based genetic medicine for rare diseases.

Focused Selection Criteria

We use a focused set of criteria to select product candidates that we believe have the best chance of success. We believe the application of our selection criteria enables the efficient, cost-effective and successful development of our product candidates. These criteria include:

  • Serious, life-threatening rare diseases;
  • Monogenic diseases with well-understood biology;
  • Disease characteristics well-suited for treatment with AAV gene therapy technology;
  • High potential for meaningful clinical benefit;
  • Compelling preclinical data;
  • Clear measures for evaluation in clinical trials; and
  • Opportunities for expedited development through established regulatory pathways.

Proprietary Manufacturing

We believe the quality, reliability and scalability of our gene therapy manufacturing approach is critical to our long-term success.

Our proprietary manufacturing capabilities provide better control over the cost and timelines of developing our product candidates, superior protection of novel inventions and intellectual property, and expanded possibilities for new programs and partnerships.

See Our Manufacturing

Broad Collaborative Network

We believe our strong relationships with key opinion leaders, leading academic institutions, other rare disease companies and patient advocacy groups will support our product development efforts and our potential for future commercial success.

Leveraging our collaborations with these parties allows us to better understand the diseases we target and optimize our research, clinical development and commercial plans.