CSL200 / CSL Behring - LARVOL DELTA

An optimized protocol for quality control of gene therapy vectors using nanopore direct RNA sequencing. (PubMed, Genome Res) - "Finally, we show that these insights can be used for the optimization of LV design. In summary, nanopore direct RNA sequencing is a powerful tool for the quality control and optimization of LVs, which may help to improve lentivirus manufacturing and thus the development of higher quality lentiviral gene therapies."

Gene therapy • Journal • Gene Therapies • Hepatology • Inflammation

[VIRTUAL] CAL-H, A NOVEL SELF-INACTIVATING LENTIVIRAL VECTOR ENCODING HUMAN Y-GLOBIN(G16D) AND SHRNA734 INHIBITS SICKLING IN RBC DERIVED FROM CD34+ STEM/PROGENITOR CELLS OF SICKLE CELL DISEASE PATIENTS (EHA 2020) - P1 | "Conclusion CAL-H-transduced CD34+ cells from SCD donors are able to differentiate ex vivo into mature RBCs containing sufficient HbF to prevent sickling under deoxygenated conditions. These data support the potential efficacy and safety of plerixafor-mobilized CAL-H-transduced HSPCs as a durable gene therapy for patients with SCD and the further clinical development of CSL200."

Clinical • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • CD34

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease (clinicaltrials.gov) - P1; N=1; Terminated; Sponsor: CSL Behring; Active, not recruiting ➔ Terminated; Unanticipated delays, not for safety reasons

Trial termination • Anemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease (clinicaltrials.gov) - P1; N=3; Recruiting; Sponsor: CSL Behring; Active, not recruiting ➔ Recruiting

Clinical • Enrollment open • Anemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Reperfusion Injury • Sickle Cell Disease • CD34

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease (clinicaltrials.gov) - P1; N=3; Active, not recruiting; Sponsor: CSL Behring; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • CD34

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease (clinicaltrials.gov) - P1; N=3; Recruiting; Sponsor: CSL Behring; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Pilot Study of CSL200 Gene Therapy in Adults With Severe Sickle Sell Disease (clinicaltrials.gov) - P1; N=3; Not yet recruiting; Sponsor: CSL Behring

Clinical • New P1 trial