Graphite Bio, Inc.

Based in California, US, and founded in 2020, Graphite Bio, Inc. is a gene-editing company that provides gene-replacement therapies intended for patients suffering from genetic diseases such as sickle cell disease (SCD) and severe combined immune deficiency (SCID). The company was co-founded by Matthew Porteus and Maria Grazia Roncarolo. As of 2020, the company is led by Dr.

Josh Lehrer as the CEO. In 2021, Graphite Bio, Inc. raised Series B funding of USD 150 million from RA Capital and Rock Springs Capital. The company launched its IPO of 14 million shares for USD 17 per share.

The company began trading on the Nasdaq Global Market under the ticker symbol, GRPH. Graphite’s pipeline of products consists of GPH101 – an HSC product candidate for therapy for sickle cell disease (SCD), GPH201 - hematopoietic stem cell treatment for patients with severe combined immune deficiency (SCID) with IL2RG deficiency, known as x-linked SCID (XSCID), and GPH301 – treatment for Gaucher disease. The company generates revenue by selling developed therapies to pharmaceutical companies. A pharmaceutical company will approach Graphite Bio, Inc.

to observe therapies under clinical trial. Once therapy was approved, the company will be paid for its developed cure of a certain genetic disease, depending on the agreement. Following the funding in 2021, Graphite Bio, Inc. plans to use the investment for product development and expansion.

Graphite Bio intends to develop GPH301 for the treatment of both Type 1 and Type 3 Gaucher disease and explore targeted conditioning regimens.