Cellular immunotherapy and gene therapy - Clinical
LINKS TO ABSTRACTS:
Presentation ID p431-1
Presenter: Irit Avivi, Israel
The study authors conclude that in the lack of prospective randomized trials evaluating CAR-T s versus chemo-immunotherapy, a propensity score, comparing CAR-T with a Pola-based regimen was performed, demonstrating a tendency for prolonged progression-free and overallsurvival in R/R DLBCL patients treated with CAR-T.
Presentation ID p431-2
Presenter: Wolfgang Miesbach, Frankfurt, Germany
The study authors conclude that following a single dose of etranacogene dezaparvovec, human factor IX activity increased, without the need for prophylactic immunosuppression, into the mild-to-normal range at 26 weeks in patients with severe/moderately severe HB. Patients were able to discontinue prophylaxis and bleeding was abolished in the majority. Safety was consistent with early phase AAV5 studies and together these data support a favorable safety and efficacy profile.
Presentation ID p431-3
Presenter: Evangelia Yannaki, United States
The study authors conclude that these results demonstrate durability and stability of response after betibeglogene autotemcel (beti-cel) gene therapy in patients with TDT with no AEs considered related to beti-cel by the investigator >2 years post-infusion. Sustained levels of HbAT87Q and effective iron reduction improved hematologic parameters and lowered iron burden.
Presentation ID p431-4
Presenter: Maria Pia Cicalese, Italy
The study authors conclude that efficacy of γ-RV-GT in Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID) is sustained with a follow-up up to 19.2 years. Due to the identified risk of leukaemogenesis, patients will continue long-term follow-up to closely monitor the safety of the product. EMA CHMP confirmed that the risk/benefit balance remains favorable for Strimvelis in its approved indication.
The study authors conclude that their interim results show that Temferon is well tolerated by patients, with no dose-limiting toxicities identified to date. The results provide initial evidence of Temferon potential to modulate the tumor microenvironment of glioblastoma multiforme, as predicted by preclinical studies.