It’s time to change the clinical paradigm

We are driven by our mission to accelerate breakthroughs in gene therapy for patients suffering from severe genetic diseases.

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We’re here to transform and accelerate

By accelerating breakthroughs in gene therapy, we strive to provide treatments that improve the lives of patients who have always been told, “there’s nothing that can be done.” 

We are committed to advocating for new care guidelines, including easily accessible newborn screening for severe genetic diseases, at a time when we can alter the trajectories of the diseases for good. We know that when you have the opportunity to treat early with a genetic disease, you offer patients, caregivers and their families more than hope – you give them options and a path toward a better life.

Leveraging innovative adeno-associated virus technologies

Our goal is to improve the quality of life for patients with severe genetic diseases through the development of gene therapy treatments that confront diseases head-on. Patients with genetic diseases often have a nonworking or missing gene. Gene therapy involves introducing healthy copies of the dysfunctional or missing gene back into the body to restore the production of a protein and the natural function of the cells. Our initial pre-clinical pipeline utilizes the proven and well-characterized AAV9 vector to target diseases with significant unmet need.

The replacement gene istransferred to the target cellvia the AAV vector. vector binds to cell vector packagedinto capsid vesicle breaksdown releasingvector nucleus cytoplasm viral vector new gene new gene injectedinto nucleus

The AAV-based gene therapy potential

Gene therapy has the potential to transform the lives of patients suffering from severe genetic diseases. By introducing healthy copies of the dysfunctional or missing gene back into the body, the gene therapy targets the root cause of a genetic disease. AAV-based gene therapy may provide a lifetime of benefit from a one-time administration.

Driven by speed-to-clinic and speed-to-market

Our team leverages a proven management team with the expertise to accelerate the development, manufacturing and commercialization of novel gene therapy treatments for patients suffering from severe genetic diseases.

  • Rapidly developing and implementing a scalable process from early stage development to commercialization
  • Decreasing overall timelines to product filing and launch
  • Optimizing product impurity profiles to improve patient safety
  • Driving innovative clinical development, and collaborative regulatory and quality strategies fundamental to success
  • Leveraging our deep commercial expertise to proactively work with payers, policymakers and providers to ensure patient access and clinical value are captured in all development strategies

Time is everything

We are focused on getting gene therapy solutions from bench to bedside as safely and quickly as possible.