FDA fast tracks unique next-generation gene-edited therapy
for sickle cell disease patients

Graphite Bio is a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to treat or cure serious diseases. The FDA has just granted Fast Track Designation to GPH101 for the treatment of sickle cell disease (SCD).

GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes SCD. GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore adult hemoglobin (HbA) expression, thereby potentially curing SCD.

Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.

The FDA’s Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. GPH101 was previously granted orphan drug designation by the FDA.

“The FDA’s decision to grant Fast Track Designation to GPH101 supports the ongoing development of our unique gene correction approach that we believe could offer a definitive cure for sickle cell patients,” said Josh Lehrer, M.D., M.Phil., chief executive officer of Graphite Bio.

Source – BusinessWire