Today DHC Principal Brent Morse and Senior Consultant Alicja Fiedorowicz published Part 1 — In-Process and Drug Product Control of Analytical Considerations for Gene-Modified Hematopoietic Stem and Progenitor Cell Therapies in BioProcess International.
Genome-modified hematopoietic stem and progenitor cell (GM-HSPC) therapies represent a significant frontier in the realm of personalized medicine, holding the promise of targeted interventions for a spectrum of disorders far beyond hematological conditions. Development of these therapies is tied intricately to the rich history of and advancements in hematopoietic stem cell (HSC) grafts, which have served as the cornerstone for our understanding of hematopoiesis and bone-marrow transplantation (1).
The journey toward gene-edited stem cell therapies began...
...continue reading the full article here.
Updated to add Part 2 on Starting Materials and Drug Substances.
This two-part review provides high-level analytical development considerations for ex vivo, genome-modified hematopoietic stem and progenitor cell (GM-HSPC) products derived from primary donor cells. Part 1 in BPI’s May 2024 issue addresses analytical controls for in-process drug substances and drug products. Here in Part 2, we take a step back to examine concerns for HSPC source materials.
...read the entirety of Part 2 here.
