RARE Daily

Kamau Therapeutics Emerges from Stealth with Graphite Bio’s Gene-Editing Portfolio

December 8, 2023

Rare Daily Staff

Kamau Therapeutics has emerged from stealth mode, following a strategic transaction with Graphite Bio in which Kamau received an option to acquire all genome editing assets, including a platform technology that integrates precision DNA repair, using homology directed repair (HDR) and CRISPR/Cas9.

Graphite Bio, in February, discontinued the development of its experimental gene-edited therapeutic for sickle cell disease, cut half its workforce, and began a process to explore and review a range of strategic alternatives focused on maximizing stockholder value. The company completed a reverse merger in November with Lenz Therapeutics, a company with a lead program targeting presbyopia.

The HDR platform technology received U.S. Food and Drug Administration clearance for the Investigational New Drug application of nula-cel and is now the basis for Kamau’s lead program, nula-cel, previously developed by Graphite. The newly established company will retain control of all intellectual property related to HDR-based CRISPR gene correction as well as the IND application.

Nula-cel is an experimental, potentially first-in-class hematopoietic stem-cell therapy engineered to restore normal adult hemoglobin (HgbA) by precisely correcting the mutation that causes sickle hemoglobin (HgbS), which may provide a cure for patients with this life-threatening genetic disease. The therapy has established proof of concept in the first patient treated for sickle cell disease. The data will be presented during a poster session on Monday, December 11, at the 2023 Annual Meeting of the American Society of Hematology.

Kamau’s HDR gene correction technology holds immense promise in enhancing human health outcomes through curative cell therapies. The versatile and robust platform corrects disease-causing DNA mutations, offering unmatched precision, template-based gene correction, and minimal off-target effects with the capacity to replenish the bloodstream with healthy red blood cells.

Kamau co-founders Matthew Porteus, previously a scientific co-founder of CRISPR Therapeutics and academic co-founder of Graphite Bio, and Maria Roncarolo, will also serve as the company’s scientific leadership to expand the use of the platform HDR technology in addressable genetic diseases beyond sickle cell disease. Jane Grogan, previously the chief scientific officer at Graphite, and Jerry Cacia, previously the chief technical officer at Graphite, will serve as strategic advisors to Kamau.

“Our HDR technology brings to life the concept of genome editing. Kamau’s approach surpasses first-generation technologies, which often introduce new errors in the genome. Instead, our technology can correct, add, replace, and rearrange disease-causing genes with functional or new ones,” said Matthew Porteus. “In our lead program, nula-cel, the technology transforms the sickle cell gene into a non-pathogenic one, addressing the root cause of the illness at the genomic level. This results in a significant reduction of pathogenic sickle hemoglobin and the production of non-pathogenic adult hemoglobin. 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.”

Sickle Cell Disease (SCD) is a common genetic condition affecting more than 500,000 people worldwide every year. and more than 100,000 individuals in the United States, with around 20 percent having a severe form of the disease. SCD occurs when a gene called HBB undergoes a specific change, which changes one substance in its genetic code to another. This genetic alteration leads to the creation of a variant known as HbS. The irregular shape of red blood cells resulting from this mutation causes serious health issues, including anemia, blockages in blood flow, severe pain, an increased risk of stroke, damage to multiple organs, and ultimately a shorter lifespan, representing a significant medical need.

Kamau is advancing its manufacturing processes. Kamau recently entered into a Contract Development and Manufacturing Organization (CDMO) agreement to advance its manufacturing processes, and is focused on elevating the HDR technology, increasing cell yield, and improving overall cell health.

The company plans to enroll additional patients with SCD in the phase 1/2 clinical trial of nula-cel, for which it has received both fast track and orphan drug designations from the FDA.

Photo: Matthew Porteus of Kamau

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