Alexandra Ward, MA

Three of the 9 patients who received the infusion demonstrated best overall response of Stable Disease, and investigators determined dose level 2 to be the biologically effective dose.

The phase 1, open-label, single-arm study demonstrated favorable safety and robust efficacy for the autologous GPRC5D-directed CAR-T cell therapy.

Data were presented at ASCO 2021 and EHA 2021 for the initial 19 patients. Now, investigators are painting a fuller picture with an additional 9.

“Therapy was found to be safe and tolerable. This initial data formed the basis for further exploration that is currently ongoing in the IGNYTE-ESO study,” investigator Sandra P. D’Angelo, MD, tells CGTL.

The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.

Investigators in Ukraine share data on the safety and efficacy of autologous mesenchymal stem cell administration for type 2 diabetes complications under the hypothesis that success is dependent on the administration route and quality of the autologous MSC population.

In a late-breaking presentation at ASGCT 2022, investigators shared preclinical results of a gene-editing strategy that seeks to reactivate developmentally silenced fetal hemoglobin (HbF, α2γ2) in order to replace defective sickle hemoglobin (HbS, α2βS2).

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

The 3 patients in cohort 1 have exhibited clinically meaningful improvements and no safety concerns. Follow-up for the 5 patients in cohort 2 is ongoing.

At ASCGT 2022, Italian investigators report updated safety and efficacy data from a phase 1/2 trial, in which all evaluable subjects achieved platelet transfusion independence and reported improved median platelet counts.

The hold was originally placed after reports of 2 serious adverse events in pediatric patients.

Although the agency has cleared the trial to restart, Pfizer will extend the voluntary hold on dosing new patients while the company talks through conditions of the restart.

The company reports positive data from first 2 patients dosed, but the agency determined there is insufficient information to support dose escalation.

The CIFFREO trial is underway in 11 countries but was halted in December 2021 amid safety concerns, including the death of a participant in a phase 1b study in the non-ambulatory cohort.

The treatment seeks to improve immune reconstitution and graft-versus-leukemia immune response without increasing the risk of GvHD in children and young adults with hematologic malignancies.

The real-world data support the recently published results from a phase 3 study, which showed most patients achieved transfusion independence.

The greatest improvements were seen in the Pain, Fatigue, and Physical Functioning domains.

In this late-breaking poster presented at the Tandem Meetings, investigators recommend that patients with primary or secondary central nervous system lymphoma be included in future clinical trials for CAR T-cell therapy.

Armed with an average of 14.3 months of follow-up data, investigators share updates from a cohort in the phase 2 study evaluating cilta-cel in lenalidomide-refractory patients with progressive multiple myeloma after 1-3 prior lines of therapy at the 2022 Tandem Meetings.

Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge Biologics, discusses the dual approach of her company in manufacturing and also developing adeno-associated viral (AAV) -based gene therapies, including FBX-101 for Krabbe disease.