Graphite Bio develops next-generation gene editing therapies that harness targeted gene integration and homology-directed repair (HDR) to repair genetic defects, deliver genetic cargo precisely, and engineer new cellular effector functions. The company is initially focused on ex vivo engineering of hematopoietic stem cells and advancing a portfolio of programs with curative potential. Its lead candidate, GPH101, has received IND clearance from the FDA to initiate a Phase 1/2 clinical trial in sickle cell disease. Graphite is completing IND-enabling studies for GPH201 (XSCID/IL2RG deficiency) and GPH301 (Gaucher disease, Types 1 and 3). The company says it will use newly raised funds to expand and advance its pipeline into the clinic and to support initial clinical milestones. Graphite Bio was co-founded by academic pioneers including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD. Graphite Bio develops a next-generation gene editing platform that enables high-efficiency, site-specific DNA integration to precisely insert or replace genetic payloads. The platform combines complementary technologies and builds on academic work from Dr. Danny Dever and Dr. Matt Porteus that increased integration efficiency from under 1% to greater than 50% across diverse lesions and cell types. Its approach aims to correct defective genes (including single-nucleotide mutations), replace malfunctioning genes while preserving normal regulation, or insert therapeutic cargo into precise genomic loci with durable expression and reduced off-target toxicity. The company’s lead program corrects the sickle β‑globin point mutation and Graphite has shown restoration of healthy hemoglobin in patient cells. Graphite expects an upcoming IND and to commence Phase 1 studies of its first candidate in early 2021, with several other programs advancing toward the clinic in the next 12–18 months. The company was co-founded by academic pioneers including Matthew Porteus and Maria Grazia Roncarolo and is led by CEO Josh Lehrer; it is backed by Versant Ventures and Samsara BioCapital following a $45 million Series A.

Total raised

$195M

Funding rounds

2

Latest round

Series B

Latest activity

Mar 2021