Graphite Bio Raises $150M to Advance GPH101, Other Gene-editing Therapies

Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative treatment for sickle cell disease (SCD).

In addition to GPH101, which recently entered clinical testing, Graphite Bio is developing two other gene-editing candidates that are still in a preclinical stage of development. These candidates are GPH301 for Gaucher disease types 1 and 3, and GPH201 for X-linked severe combined immune deficiency (XSCID).

“We are grateful for the confidence of our investors, which will support our clinical trial in sickle cell disease and advance our XSCID and Gaucher programs toward the clinic,” Josh Lehrer, MD, CEO of Graphite Bio, said in a press release.

The gene-editing company’s lead clinical candidate, GPH101, directly targets the HBB gene that encodes the beta-globin protein, a critical component of the oxygen-carrying hemoglobin protein.

In patients with SCD, a mutation in HBB causes hemoglobin to stick together, leading to abnormally shaped, fragile red blood cells known as sickle cells. GPH101 uses a combination of natural DNA repair mechanisms along with the CRISPR-Cas9 gene-editing tool to correct the disease-causing mutation in HBB and restore normal hemoglobin function.

Earlier this year, Graphite Bio received authorization from the U.S. Food and Drug Administration to launch CEDAR, the first-in-human Phase 1/2 trial to assess the safety, pharmacological properties, and preliminary efficacy of GPH101 in adults and adolescents with severe SCD.

With that decision, Graphite Bio became the first company to lead a potentially curative therapy for SCD to clinical testing. CEDAR is expected to start later this year.

“In a short time, the Graphite Bio leadership team has demonstrated impressive execution of its clinical development strategy, advancing GPH101 into a Phase 1/2 clinical trial for sickle cell disease,” said Dave Gardner at Rock Springs Capital, one of the investment companies that led the financing round.

“Rock Springs is confident in Graphite Bio’s ability to leverage its differentiated gene editing approach to positively impact patients across a broad range of diseases,” Gardner said.

In addition to Rock Springs Capital, the financing round was led by RA Capital Management. These companies were joined by multiple new investors, to include Cormorant Asset Management, Deerfield Management Co., Federated Hermes Kaufmann Funds, Fidelity Management & Research Co., Janus Henderson Investors, and Logos Capital. Other new investors include OrbiMed, Perceptive Advisors, Surveyor Capital, and Venrock Healthcare Capital Partners.

It also was supported by the existing investor Samsara BioCapital and founding investor Versant Ventures.

“RA Capital believes that Graphite Bio’s targeted integration approach represents the next generation of gene editing, and we are excited about the significant potential that this platform holds for a wide variety of diseases,” said Jake Simson, partner at RA Capital Management.

“The progression of Graphite’s programs, broad therapeutic potential of the platform, and the leadership team’s demonstrated track record in driving programs to patients motivated us to fund the advancement of Graphite Bio’s pipeline,” Simson added.