Graphite Bio Secures $150 Million Series B Financing to Advance Pipeline of Next-Generation Gene Editing Therapies

  • Total of $195 million raised to date to support expansion and clinical advancement of Graphite Bio pipeline of targeted gene integration therapies with curative potential
  • The financing, led by RA Capital Management and Rock Springs Capital, included participation by other prominent new and existing investors

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Graphite Bio, a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced the close of an oversubscribed $150 million Series B financing. The company will use the funds to expand and advance into the clinic its pipeline of investigational high efficiency, targeted gene integration therapies with curative potential for serious diseases.

RA Capital Management and Rock Springs Capital led the financing with significant participation from new investors Cormorant Asset Management, Deerfield Management Company, Federated Hermes Kaufmann Funds, Fidelity Management & Research Company, Janus Henderson Investors, Logos Capital, OrbiMed, Perceptive Advisors, Surveyor Capital (a Citadel company), and Venrock Healthcare Capital Partners, joining existing investor Samsara BioCapital and founding investor Versant Ventures.

“We are gratified to receive the tremendous support from this group of world-class investors who share in our ambitious vision. This milestone provides further validation for our highly differentiated gene integration platform and enables us to advance our pipeline through initial clinical milestones,” said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. “To date, we have delivered on our commitment to advance our pipeline with urgency, always with an intent focus on delivering truly innovative therapies to patients. 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.”

Graphite Bio has received IND clearance from the U.S. Food & Drug Administration (FDA) to initiate its first Phase 1/2 clinical trial evaluating investigational candidate GPH101 in sickle cell disease. The company is also completing IND-enabling studies to advance investigational therapies GPH201 and GPH301 in severe combined immune deficiency with IL2RG deficiency, known as x-linked SCID (XSCID), and Gaucher Disease (Types 1 and 3), respectively.

“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.”

“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. “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.”

Cowen acted as a financial advisor for the financing.

About Graphite Bio
Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The company’s targeted gene integration platform harnesses the cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients’ lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD. For more information, please visit

Christy Curran
Sam Brown, Inc.

Source: Graphite Bio