Case Study
Investing in Your Success - Kamau Therapeutics Case Study
Abstract: Investing in Your Success Upfront
Kamau Therapeutics is a clinical-stage gene correction company advancing cell therapy development with high-efficiency targeted gene integration. Their platform, leveraging HDR editing and CRISPR-Cas9 technology, not only cuts DNA but also repairs it, building on Dr. Matthew Porteus’ research at Stanford Medicine.
Kamau Therapeutics partners with MaxCyte to optimize cell engineering processes. Using MaxCyte’s GMP platform, they have advanced their lead program, nula-cel, currently in clinical development for Sickle Cell Disease. Since 2021, the partnership has focused on process development, culminating in a clinical collaboration announced in 2024.
Kamau Therapeutics emerged out of stealth to continue clinical development for nula-cel in Sickle Cell Disease.
MaxCyte provided continuous support for Kamau Therapeutics to design, optimize, and troubleshoot their CD34+ HSC engineering process.
Together MaxCyte & Kamau Therapeutics optimized and qualified a scalable, reproducible, closed CMC process using an ExPERT GTx® instrument.
MaxCyte and Kamau Therapeutics announced a clinical collaboration to use the optimized cell engineering process to continue GMP manufacturing for their lead asset, nula-cel for Sickle Cell Disease.
“Through our collaboration with MaxCyte and the use of their proven non-viral platform, our goal is to treat or cure a range of serious genetic diseases with unmet medical needs using homology-directed repair.”
Matt Porteus, Co-Founder, Kamau Therapeutics, Inc.
Summary
- Kamau Therapeutics emerges out of stealth to continue clinical development for nula-cel in Sickle Cell Disease.
- MaxCyte provided continuous support for Kamau Therapeutics to design, optimize, and troubleshoot their CD34+ HSC engineering process.
- Together MaxCyte & Kamau Therapeutics optimized and qualified a scalable, reproducible, closed CMC process using ExPERT GTx® process.
- Development costs for early-stage companies can exceed $1M/month, and cutting process development timelines can significantly reduce cash burn.
- MaxCyte and Kamau Therapeutics announce clinical collaboration to use the optimized cell engineering process to continue GMP manufacturing for their lead asset, nula-cel for Sickle Cell Disease.
