Vertex Pharmaceuticals and CRISPR Therapeutics submit FDA rolling Biologics License Applications for gene editing-based medicine
pharmafile | April 4, 2023 | News story | Research and Development |
US-based Vertex Pharmaceuticals and Swiss CRISPR Therapeutics have announced the completion of their rolling Biologics License Applications (BLAs) to the FDA for exagamglogene autotemcel (exa-cel), an investigational treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Exa-cel (CTX001) is an investigational, autologous, ex vivo CRISP/Cas9 gene-edited therapy where a patient’s own haematopoietic stem cells are edited to produce high levels of foetal haemoglobin (HbF; haemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying haemoglobin that is naturally present during foetal development ‒ this then switches to the adult form after birth. By elevating HbF with exa-cel, there is the potential to reduce or eliminate painful vaso-occlusive crises (VOCs) for SCD patients, or eliminate transfusion requirements for TDT patients.
The BLAs are supported by the results from the ongoing phase 3 CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study CLIMB-131. The patients in these trials will have their own haematopoietic stem and progenitor cells collected from blood.
Carmen Bozic, MD, executive VP, Global Medicines Development and Medical Affairs, and chief medical officer at Vertex Pharmaceuticals, said, “The completion of our exa-cel global regulatory filings is a historic milestone. We want to thank the clinical trial participants and the sickle cell and beta thalassemia communities, as well as the physicians, nurses, coordinators, caregivers and friends who support them.”
“Within a decade, we have progressed from the discovery of the CRISPR platform to the first regulatory filings for a CRISPR-based therapy, which speaks to the transformative nature of CRISPR technology,” said Phuong Khanh (P K) Morrow, MD, FACP, chief medical officer at CRISPR Therapeutics.
James Spargo
