Genomics
Leveraging knowledge of the genome to potentially address rare diseases with a genetic cause.
Exploring Opportunities to Target the Genetic Drivers
Recent advances in genomics have transformed drug discovery and clinical research towards precision medicine. This has created the opportunity to target the genetic drivers of rare and chronic disease to alter the course of disease and enable long-lasting treatment.
Genomic medicine is an emerging discipline that uses an individual’s genetic information to address disease at the genetic level. As pioneers in rare disease R&D, we are uniquely positioned to accelerate the promise of genomic medicine for people living with rare diseases.
Understanding the Underlying Genetic Cause of Disease
The treatments used in genomic medicine come from a variety of strategies, including gene therapy and gene editing. Gene therapy is designed to add, alter or inactivate the disease-associated gene. Gene editing is designed to repair the faulty gene by directly changing the genetic material (e.g., cell’s DNA). By directly addressing the underlying genetic cause of disease, these strategies have the potential to treat many rare diseases, 80% of which are believed to have a genetic cause.
Advancing Potential Genomic Medicine Treatments
We have the capabilities to deliver an industry-leading suite of genomic medicine platforms to advance our gene therapy and gene editing programmes. Our platforms are like a robust array of building blocks, creating the potential for us to operate with versatility and adapt our scientific approach to develop precision treatments for a variety of rare diseases. We are pairing these building blocks with our manufacturing proficiency to potentially target complex diseases. And we aim to drive our programmes into the clinic rapidly to bring promising genomic medicine treatments to people living with rare diseases as quickly as possible.
Establishing an Ecosystem and Fostering Access
We are committed to establishing an ecosystem that fosters access to genomic medicines. With our history of listening to and learning from patients and caregivers, working with regulatory agencies and payers and designing, and executing clinical trials, we aim to drive our programmes into the clinic rapidly to bring promising genomic medicine treatments to people living with rare diseases as quickly as possible.
