March 15, 2021 -- Graphite Bio has closed $150 million in financing to expand and advance its pipeline of targeted gene integration therapies.
The company received an investigational new drug (IND) clearance from the U.S. Food and Drug Administration (FDA) to initiate its first phase I/II clinical trial evaluating GPH101, the company's candidate in sickle cell disease. Graphite is also completing IND-enabling studies for its GPH201 candidate in severe combined immune deficiency (SCID) with IL2RG deficiency, known as X-linked SCID (XSCID) and GPH301 for Gaucher disease (types 1 and 3).
Graphite Bio's targeted gene integration platform harnesses the cellular process of homology-directed repair to efficiently repair genetic defects at their source, deliver genetic cargo with precision, and engineer new cellular effector functions. The company is focused on developing ex vivo engineered hematopoietic stem cells.
The financing round was led by RA Capital Management and Rock Springs Capital.
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