Philippe Duchateau, PhD, Chief Scientific Officer at Cellectis, discusses the company’s gene editing technology which could potentially treat rare diseases such as sickle cell anemia.
Sickle cell anemia is an inherited blood disorder. Early symptoms usually occur in childhood and include swelling, fatigue, and jaundice. As the disease progresses, there is an increased risk of infections, delayed growth, and periodic episodes of pain (sickle attacks). Over time, organ damage can occur, creating problems in the spleen, brain, eyes, lungs, liver, heart, kidneys, penis, joints, bones, and/or skin.
Sickle cell anemia is caused by a single mutation in the HBB gene. TALGlobin01 is an autologous cell-based gene therapy product designed by Cellectis to repair the mutated HBB gene and subsequently restore production of Hemoglobin A in sickle cell anemia.
Preliminary clinical data on the use of TALGlobin01 in sickle cell anemia patients were recently presented at The American Society of Hematology Meeting & Exposition (ASH 2021). As Dr. Duchateau notes, these data indicated that the use of TALGlobin01 resulted in up to 70% of Homology Directed Repair (HDR)-mediated HBB gene correction in the hematopoietic stem and progenitor cells (HSPCs) of sickle cell anemia patients.
Cellectis is also investigating the use of TALEN-edited hematopoietic stem cell (HSC) therapy in lysosomal storage disorders. As Dr. Duchateau explains, most patients with lysosomal storage disorders receive enzyme replacement therapies that cannot cross the blood-brain barrier; however, a TALEN-edited HSC therapy could, which means such a therapy could effectively treat neurological symptoms associated with some lysosomal storage disorders.
To stay up to date with the latest developments in rare diseases, follow us on Twitter (@CheckRare)