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Kamau Therapeutics Reveals Clinical-Stage Gene Correction Technology for Life-Threatening Genetic Diseases After Emerging from Stealth Mode

Friday, December 08, 2023

Kamau Therapeutics, a company in the clinical-stage of gene correction, has recently emerged from stealth mode following a strategic deal with Graphite Bio. In this arrangement, Kamau gained an option to acquire all genome editing assets, encompassing a platform technology utilizing precision DNA repair through homology-directed repair (HDR) and CRISPR/Cas9.

Developed by gene-editing experts Drs. Matthew Porteus and Maria Grazia Roncarolo at Stanford, the HDR platform received FDA clearance for the Investigational New Drug (IND) application of nulabeglogene autogedtemcel (nula-cel), now forming the foundation for Kamau's lead program. This program, nula-cel, is designed to potentially cure sickle cell disease by precisely correcting the mutation causing it.

Nula-cel, described as an investigational hematopoietic stem-cell therapy, aims to restore normal adult hemoglobin by precisely addressing the genetic mutation responsible for sickle hemoglobin. The therapy has demonstrated proof of concept in the initial patient treated for sickle cell disease, with comprehensive data set to be presented at the 2023 Annual Meeting of the American Society of Hematology (ASH).

Kamau's HDR gene correction technology holds the promise to redefine genetic disease treatment by offering precision, template-based gene correction with minimal off-target effects. Co-founders Dr. Porteus and Dr. Roncarolo are leading the scientific efforts to expand the technology's application beyond sickle cell disease to other genetic diseases.

The one-year follow-up on the first patient treated with nula-cel will be presented at the ASH Annual Meeting, outlining outcomes and insights from the Phase 1/2 clinical trial. Early results show promise, and although challenges were encountered, the treatment demonstrated positive impacts on reducing sickle hemoglobin and increasing healthier hemoglobin types.

In a significant move, Kamau has entered into a Contract Development and Manufacturing Organization (CDMO) agreement to enhance manufacturing capabilities. This strategic initiative aims to optimize production efficiency, increase cell yield, and improve overall cell health for a robust and sustainable manufacturing framework.

Sickle Cell Disease affects over 500,000 people worldwide annually, with more than 100,000 individuals in the U.S. having the disease. Kamau's gene-editing technology holds significant promise in providing a cure for this life-threatening genetic condition, marking a substantial advancement in genetic medicine.



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