San Francisco-based Kamau Therapeutics, a clinical-stage gene correction company, has emerged from stealth mode, following a strategic transaction with the struggling Graphite Bio.
Under the terms of the agreement, Kamau will acquire all Graphite's genome editing assets, including a platform technology that integrates precision DNA repair, using homology directed repair (HDR) and CRISPR/Cas9.
According to Kamau, the HDR platform technology will form the basis for its lead program, nulabeglogene autogedtemcel (nula-cel), previously developed by Graphite. Nula-cel is an investigational hematopoietic stem-cell therapy engineered to restore normal adult hemoglobin by precisely correcting the mutation that causes sickle hemoglobin — potentially providing a cure for patients with the life-threatening genetic disease.
Graphite had received FDA clearance for the IND application of nula-cel back in
December 2020. Then, in February 2023, following a serious adverse event in an early trial, Graphite decided to discontinue further development of the treatment for sickle cell disease, cutting the company’s workforce in half in the process.
Now, Graphite is fresh off a reverse merger deal with California biotech Lenz Therapeutics that formed a new publicly-traded combined company focused on presbyopia, the inevitable loss of near vision in people over the age of 45.
Kamau still believes in the tech, and aims to enroll additional patients with sickle cell disease in the phase 1/2 clinical trial within the next 18 months. "Kamau aims to achieve the gold standard in genome engineering: directly correcting mutations that cause diseases, and we are making strides toward this ambitious goal," said Kamau co-founder Dr. Matthew Porteus.
