Graphite Bio Achieves Major Victory Against Sickle Cell Disease, Reduces Workforce by Half

Graphite Bio has pulled the plug on its much-anticipated gene therapy for sickle cell disease (SCD), nulabeglogene autogedtemcel (nula-cel). The end of the development of this potential breakthrough treatment has left many SCD sufferers and researchers disappointed.

Graphite, a Bay Area biotech, is undergoing a major corporate restructuring that will result in the loss of 50% of its workforce, as well as other cost-saving initiatives. With $283.5 million in cash, cash equivalents, and marketable securities at the end of 2022, the company is hoping to reduce cash burn and become more efficient.

Nula-cel is a revolutionary gene-editing therapy that seeks to correct the mutation in the beta-globin gene that causes sickle cell disease (SCD). This innovative autologous hematopoietic stem cell treatment has the potential to dramatically reduce the production of sickle hemoglobin, while simultaneously restoring levels of healthy adult hemoglobin. It is an exciting advancement in the medical world, offering hope to those living with SCD.

Graphite was at the forefront of research in nula-cel in the Phase I/II, open-label CEDAR trial, which saw 15 participants testing the candidate’s safety, engraftment success, gene correction rate and effect on total hemoglobin levels. In August 2022, Graphite marked a milestone by dosing their first patient for the CEDAR trial.

Several months after commencing a clinical trial, Graphite decided to temporarily suspend it after a volunteer had a prolonged period of low blood cell counts, known as pancytopenia. Even though this event did not meet the criteria for halting the trial, Graphite judged it was likely caused by the study medication, and so decided to err on the side of caution.

The patient was successful in achieving neutrophil engraftment as indicated by CEDAR and has since had no signs of myelodysplasia. However, during this period, the patient required transfusions and growth factor support to manage episodes of pancytopenia.

At the time, Graphite’s Vice President of Communications and Investor Relations, Stephanie Yao, revealed that the company was diligently working to reduce the risk factors associated with its nula-cel manufacturing process. According to Yao, Graphite was evaluating whether modifications to the process could improve key drug product characteristics.

In a shocking announcement on Thursday, Josh Lehrer, CEO of nula-cel, declared that it was no longer feasible to continue the development of the company’s product due to the “time and resources needed to resume the CEDAR study and the evolving treatment landscape” for Sickle Cell Disease.

Despite the challenges, the company remains committed to its gene-correction approach as a potential treatment for sickle cell disease (SCD). With a determined spirit, they are exploring external development opportunities for their revolutionary therapy, nula-cel, with the hope of one day making a real difference in the lives of those affected by SCD.

Graphite is determined to expand the accessibility of its gene therapies to a wider range of diseases and patients, and is actively pursuing research activities for its non-genotoxic conditioning program for hematopoietic stem cell transplantation. This groundbreaking program could revolutionize the way we treat certain illnesses and open up possibilities for even more effective treatments.

Graphite is set to revolutionize the medical field with its breakthrough gene therapies. In particular, its beta-thalassemia gene replacement therapy GPH-102 and its targeted gene insertion candidate for alpha-1 antitrypsin deficiency are both showing promising results in early development. With these treatments, Graphite has the potential to revolutionize the way we approach treating genetic disorders, creating a brighter future for those suffering from these diseases.

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