Exploring Genome Editing Treatments Beyond Infant KJ: Advanced Stages in their Creation
Headline: Panel Discussion Highlights Challenges and Solutions in Scaling Gene-Editing Therapies
In a recent discussion on GEN Live, industry experts Sandy Ottensmann, Mark Wetzel, and Sadik Kassim came together to examine the work behind a successful gene-editing therapy treatment.
Sandy Ottensmann, the Vice President and General Manager of the Gene Writing & Editing Business Unit at IDT, was joined by Mark Wetzel, the Vice President and General Manager of mRNA CDMO Services at Aldevron. Sadik Kassim, who holds a PhD, completed the panel.
Last month, a significant milestone was reached as a baby with a rare genetic disorder named "Baby KJ" was treated with a gene-editing therapy tailored to his specific mutation. This treatment was a collaborative effort between coordinated teams at IDT, Aldevron, and Danaher Corporation, another company associated with the panelists.
The gene-editing payload and its delivery system were produced under intense pressure, and the discussion explored how to deliver treatments like this on a larger scale in the future. The real challenge lies ahead in delivering these therapies to more patients, with challenges such as scale and cost being a key focus.
Aldevron and Danaher are companies that have been involved in the manufacturing of the gene-editing therapy. Mark Wetzel is associated with mRNA CDMO Services at Aldevron, while Sandy Ottensmann is the Vice President and General Manager of the Gene Writing & Editing Business Unit at IDT. Danaher Corporation is also associated with the panelists.
The treatment in question, produced for "Baby KJ", was the Novartis gene therapy Onasemnogene Abeparvovec (Zolgensma), which has been approved since 2020 for spinal muscular atrophy (SMA) caused by SMN1 gene mutation. The production scale-up to larger patient populations is ongoing, with an emphasis on early treatment in specialized centers.
As the gene-editing field continues to evolve, discussions such as this one are crucial in addressing the challenges that lie ahead and finding solutions to make these life-changing therapies accessible to more patients.
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