NEW YORK – Kamau Therapeutics has launched as a new biopharmaceutical company focused on treating genetic diseases with its gene-correction technology.
The San Francisco-based firm, which emerged from stealth on Thursday, is launching with assets from gene-editing company Graphite Bio. Under the terms of a deal with Graphite, Kamau has an option to acquire all of Graphite's genome editing assets, including a DNA repair platform that uses homology directed repair (HDR) and CRISPR-Cas9 technologies.
Genetics medicine firm Graphite launched in 2020, having raised $45 million in a Series A funding round. However, in February, the firm announced it would reduce its workforce by 50 percent and discontinue further development of nulabeglogene autogedtemcel (nula-cel) as a treatment for sickle cell disease. This decision came a month after the gene editing-based, autologous hematopoietic stem cell therapy caused a serious adverse event in the first patient dosed in the Phase I/II CEDAR trial. Graphite voluntarily paused the trial to investigate the adverse event, but ultimately decided it would pursue strategic alternatives, including external development of the drug.
Kamau has acquired Graphite's nula-cel as its lead program. The new firm will retain control of all intellectual property related to the HDR-based CRISPR gene correction technology and nula-cel's investigational new drug application.
Nula-cel is a hematopoietic stem cell therapy that's engineered to transform the sickle cell gene into a non-pathogenic one, correcting the mutation that causes hemoglobin to sickle. Kamau will present proof-of-concept data from the first sickle cell disease patient treated with nula-cel within the CEDAR trial during a poster session at the American Society of Hematology's annual meeting on Monday.
Kamau said it will continue to enroll more patients with sickle cell disease into the Phase I/II trial over the next 18 months.
The company's scientific leadership comprises cofounders Matthew Porteus and Maria Grazia Roncarolo, who developed the HDR platform technology. Porteus also was a scientific cofounder of CRISPR Therapeutics and academic cofounder of Graphite. Jane Grogan, former CSO at Graphite, and Jerry Cacia, former chief technical officer at Graphite, will serve as strategic advisers to Kamau.
Graphite in November announced it would merge with LENZ Therapeutics in an all-stock merger and develop treatments for presbyopia, a condition that causes vision loss. In connection with the merger, Graphite entered into a subscription agreement to raise $53.5 million through a private investment in public equity financing.