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.

Expanded access policy

Discovery Validation
IND Enabling
Phase 1
Phase 2
Phase 3
Discovery
Validation
IND
Enabling
1
Phase
2
3
GPH101
Sickle cell disease (SCD)
Gene correction

GPH101 is our investigational, next-generation, gene-edited autologous stem cell-based therapy that is currently in development for sickle cell disease (SCD). SCD is a group of rare inherited blood disorders and among the most prevalent monogenic blood diseases. It is estimated that there are 100,000 people living with SCD in the United States and millions worldwide. With GPH101, 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.

GPH101 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.

Learn more about the Phase 1/2 CEDAR clinical trial here.

Discovery
Validation
IND
Enabling
1
Phase
2
3
GPH102
Beta-thalassemia
Gene replacement

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.

Discovery
Validation
IND
Enabling
1
Phase
2
3
Therapeutic protein production (alpha-globin)
Alpha-1 antitrypsin (AAT) deficiency
Targeted gene insertion

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.

Discovery
Validation
IND
Enabling
1
Phase
2
3
Non-genotoxic conditioning (NGTC)
Undisclosed
Engraftment

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.

We are driven to help patients live their best lives

Our team works tirelessly to change the lives of millions of people around the world through collaboration, bold thinking, and a culture that believes we are smarter when we work together.

Careers