A pipeline with the potential to cure
Aiming to develop cures for diseases previously thought to be incurable
We’re reimagining what’s possible in gene editing tomorrow by creating it today—all with the goal of helping people live longer, healthier lives. By harnessing the power of our UltraHDR™ platform to fulfill the promise of gene editing, we’re getting closer to achieving our mission of making ‘one dose, one cure’ a reality.
Our groundbreaking work has led to promising advancements toward potential cures for serious genetic diseases, like sickle cell disease and beta-thalassemia.
The importance of our work is embodied in the progress we’re making, as we get closer to helping patients write the next chapter in their life story.
Validation
Enabling
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Nula-cel, formerly known as GPH101, is our investigational, next-generation, gene-edited autologous stem cell-based therapy that is currently in development for sickle cell disease (SCD). A serious, life-threatening inherited blood disorder, SCD affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. With nula-cel, we aim to directly correct the underlying mutation that causes SCD to decrease the production of sickle hemoglobin and restore the expression of adult hemoglobin, thereby potentially curing the disease. The U.S. Food and Drug Administration (FDA) granted Fast Track and Orphan Drug designations to nula-cel for the treatment of SCD.
Nula-cel is being studied in the CEDAR trial, a Phase 1/2 multicenter, open-label clinical trial designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.
Validation
Enabling
2
GPH102 is our research program for the treatment of beta-thalassemia, one of the most common autosomal recessive disorders, with approximately 68,000 people worldwide born with the disease each year. Beta-thalassemia is a genetic blood disorder characterized by reduced production of beta-globin, a protein that forms oxygen-carrying hemoglobin with alpha-globin. Individuals with the most severe form of beta-thalassemia fail to produce functional beta-globin, which results in severe anemia and transfusion dependency. Using our gene replacement approach, GPH102 is designed to replace the mutated beta-globin gene with a functional gene and restore adult hemoglobin (HbA) expression to levels similar to individuals who do not have the disease.
Validation
Enabling
2
We have an early-stage research program for the treatment of alpha-1 antitrypsin (AAT) deficiency, a severe inherited genetic disorder that can cause progressive lung and liver disease, and affects approximately 60,000 people in the United States. The program leverages the company’s targeted gene insertion approach to permanently increase AAT protein production, offering a new way to potentially treat the disease.
Validation
Enabling
2
We have ongoing discovery research efforts to develop potential best-in-class non-genotoxic HSC targeted conditioning (NGTC) regimens, which could significantly broaden the number of diseases and patients who can be treated with the company’s precision-engineered, one-time treatments and cures. The company also intends to leverage industry advances to accelerate its efforts in this area.
Join our mission
to cure
At Graphite Bio, we rise to the occasion with energy and courage to deliver for patients. As a part of our team, you’ll share that innate sense of responsibility to improve the greater good, using your expertise and passion to meaningfully improve lives—because you know you can.