beyond-baby-kj:-next-steps-in-manufacturing-genome-editing-cures
Beyond Baby KJ: Next Steps in Manufacturing Genome Editing Cures

Beyond Baby KJ: Next Steps in Manufacturing Genome Editing Cures

Panelists:

Image of Sandy Ottensmann

Sandy Ottensmann

VP/GM, Gene Writing & Editing Business Unit, IDT

Panelist

Image of Sandy Ottensmann

Sandy Ottensmann

Sandy Ottensmann has spent the last decade within Danaher’s life sciences and diagnostics business, having led IDT’s Gene Writing and Editing and Core/PCR Business Units since January 2023. She holds an MBA from Harvard Business School, a Master of Science in Chemistry from Stanford University, and a Bachelor of Science with honor in Chemistry from the California Institute of Technology.

Image of Mark Wetzel

Mark Wetzel

VP/GM mRNA CDMO Services, Aldevron

Panelist

Image of Mark Wetzel

Mark Wetzel

Mark Wetzel is VP/GM mRNA CDMO Services at Aldevron .

Image of Sadik Kassim, PhD

Sadik Kassim, PhD

CSO/CTO (Genomic Medicines), Danaher Corporation

Panelist

Image of Sadik Kassim, PhD

Sadik Kassim, PhD

Sadik Kassim is a scientist at Genomic Medicines for the Life Sciences Companies at Danaher and executive with extensive experience in the biotechnology industry with a specific focus on cell and gene therapy bioprocessing and translational research. Currently, he serves as CSO and CTO at Danaher, with a focus on Genomic Medicines.

Broadcast Date: 

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Last month, for the first time, a baby with a rare genetic disorder known as “Baby KJ” was treated with a gene-editing therapy tailored to his rare mutation. The milestone made headlines—and marked a win for the genome editing field, offering new promise for the future of genomic medicine.

But there is another layer to the story: how the treatment was manufactured. Developing a single, personalized therapy required a coordinated sprint from teams at IDT, Aldevron, and Danaher, working under intense pressure to produce the gene-editing payload and its delivery system. Now, the real challenge lies ahead—delivering treatments like this to more patients. On this GEN Live, we take a close look at the work behind this successful treatment and perhaps more importantly, we explore how this can be done on a larger scale in the future. We will discuss the challenges that lie ahead, including scale and cost, and how some companies are thinking about solving those.

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