Our approach

Our company is purpose built to treat the underlying cause of genetic disease. The key elements of our strategy to achieve this vision are

  • Focus our gene therapy candidates in areas of high unmet need and with substantial potential for societal impact and commercial opportunity, such as genetically defined cardiovascular diseases and APOE4- associated Alzheimer’s disease.
  • Advance a deep and diverse pipeline that includes candidates that are designed to address both larger rare and more prevalent patient populations, prioritizing conditions most likely to benefit from our therapies.
  • Pursue a staged, capital-efficient approach for advancing programs through clinical development and regulatory approval.
  • Utilize a unified, high-quality manufacturing platform that can quickly respond at scale to high impact opportunities.
  • Pursue next-generation genetic medicine technologies that can enhance our capabilities and expand our impact on patients.
  • Leverage and expand upon our partnerships and exclusive licenses with world class academic institutions.
  • Build a fully integrated genetic medicine company and selectively evaluate strategic opportunities to maximize the impact of our pipeline.