It’s time to change the clinical paradigm

We are driven by the urgent mission to rapidly advance the development and delivery of life-changing gene therapies to patients and families in need.

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We’re here to transform and accelerate

Our mission is to transform the practice of medicine, providing clinicians with treatments that offer remarkable results for diseases in which patients 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 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 treatment, you offer patients, their loved ones and their clinicians more than hope – you give them options and a path toward a better life.

Leveraging innovative Adeno-Associated Virus technologies

Through the development and application of innovative gene therapies, our goal is to confront severe and life-threatening genetic diseases head-on, targeting faulty genes and introducing functional genetic material to restore function. Our team is leveraging the latest Adeno-Associated Virus (AAV) technologies as our targeted method of delivery.

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 and families suffering from severe and life-threatening genetic diseases.

It is estimated that between 8,000 and 10,000 diseases have a gene deficiency root cause and are ideal candidates for gene therapy treatment.

AAV-mediated gene therapy may provide a lifetime of benefit from a one-time administration.

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

Our team is focused on driving transformational outcomes in gene therapy powered by internal company capabilities and an experienced team focused on winning for patients. We are committed to:

  • 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.