Research & Development
Graphite Bio Announces US FDA Fast Track Designation Granted to GPH101 for the Treatment of Sickle Cell Disease
3 May 2022 - - The US Food and Drug Administration has granted Fast Track Designation to GPH101 for the treatment of sickle cell disease, US-based gene editing company Graphite Bio, Inc. (NASDAQ: GRPH) said.

GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes 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.

GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes sickle cell disease.

SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide.

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 production and restore adult hemoglobin 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.

Graphite Bio is a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases.

Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine's most elusive goals: to precisely "find & replace" any gene in the genome.

Graphite Bio's UltraHDR gene editing platform is designed to precisely correct genetic mutations, replace entire disease-causing genes with functional genes or insert new genes into predetermined, safe locations.

The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.
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