Latest Conference Coverage

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).

Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.

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.

Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.

Updated data at ARVO show that AGTC-501 had sustained efficacy and safety at 18 months for patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.

New data from cohort 4 showed outer retinal restoration following treatment with OpRegen.

The post-hoc analysis of the OPTIC trial explored the effects of neutralizing antibodies on patients treated with ADVM-002.

There was a continued trend toward improvement of overall survival with omidubicel at 73% compared with UCBT at 60%.

The analysis of data from ZUMA-7 demonstrated significant efficacy for axi-cel over second-line standard treatment.

Tisa-cel was associated with a more favorable safety profile in the analysis.

In this population, median progression-free survival was nearly 40 months.

A phase 1/2 multicenter study is underway following MB-106’s IND approval.

KUR-502 elicited promising response rates for patients with relapsed or refractory B-cell malignancies in a phase 1 study.

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.

Preliminary findings from the phase 1 clinical trial demonstrate good cell persistence and expansion in vivo.

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 37 evaluable patients, the objective response rate in the study was 89% (95% CI, 75%-97%) with complete responses in 78%.

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.

Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.

Peter J. McAllister, MD, FAAN, presented promising data from the phase 2 STEMTRA trial at AAN 2022.

While the lower IV dose was generally well-tolerated, the higher dose yielded cases of serious cytokine release syndrome.

The phase 1, investigator-initiated trial continues to recruit participants with B-ALL.

The novel regimen from BioNTech demonstrated encouraging results in patients with testicular or ovarian cancer.

AFM13 is currently also being evaluated on its own in lymphoma in a phase 2 registrational study.

The first in-human phase 1 clinical trial is being conducted in Japan.