RoslinCT, a renowned cell and gene therapy Contract Development and Manufacturing Organisation (CDMO), has achieved a groundbreaking milestone in the field of medical science.
Building on its legacy since the cloning of Dolly the Sheep at the Roslin Institute in 1997, RoslinCT has now been entrusted with the manufacturing of the first-ever U.S. Food and Drug Administration (FDA) approved CRISPR-based gene therapy, CASGEVY™ (exagamglogene autotemcel, or exa-cel).
CASGEVY™: A New Hope for Sickle Cell Disease Patients
Approved by the FDA on December 8, 2023, CASGEVY™ is a CRISPR/Cas9 genome-edited cell therapy designed to treat sickle cell disease (SCD) in patients aged 12 and older who suffer from recurrent vaso-occlusive crises (VOCs).
This approval follows the world’s first authorization of the treatment by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on November 15, 2023. The therapy represents a significant advancement in treating this debilitating disease, offering new hope to patients in the US and UK.
The Science Behind CASGEVY™
CASGEVY™ is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology. It targets the erythroid-specific enhancer region of the BCL11A gene.
The therapy aims to reduce BCL11A expression in erythroid lineage cells, leading to increased production of fetal hemoglobin (HbF). HbF, naturally present during fetal development, is known to reduce or eliminate vaso-occlusive crises in patients with SCD.
RoslinCT’s Role and Capabilities
RoslinCT’s involvement in the development and manufacturing of CASGEVY™ underscores its capabilities in handling complex autologous and allogeneic cell therapies.
With state-of-the-art facilities in Edinburgh, Scotland, and Boston, US, RoslinCT is equipped to support a broad range of gene editing and induced Pluripotent Stem Cell (iPSC) capabilities. Their expertise has been pivotal in advancing this therapy from early development to commercialization.
Looking Ahead: Expanding Treatment Possibilities
The FDA is also reviewing Exa-cel for transfusion-dependent beta thalassemia, with a decision expected by March 20, 2024. This expansion of treatment possibilities further highlights the potential of CRISPR-based therapies in addressing various genetic disorders.
Conclusion
The approval of CASGEVY™ marks a significant milestone in the field of gene therapy. It not only showcases the potential of CRISPR technology in treating genetic disorders but also highlights RoslinCT’s role as a leader in the development and manufacturing of advanced therapies.
As we look forward to the broader implications of this development, it’s clear that we are entering a new era of medical science, where genetic diseases once thought incurable are now within the realm of treatable conditions.
For more information about RoslinCT and their services, visit their website.