News

Improvements persisted through up to 10 years of follow-up.

The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.

Review top news and interview highlights from the week ending October 1, 2021.

Treatment with the CAR T-cell therapy yielded a 60% reduction in the risk of EFS events compared with SOC with a median follow-up of 2 years.

The chief of translational Research and immunotherapy at The Angeles Clinic and Research Institute discussed the integration of tumor infiltrating lymphocyte–based therapy into the melanoma armamentarium.

The dose was previously evaluated in adult patients with no comparable inflammation.

The decision comes after 3 participants experienced serious adverse events during the phase 3 trial.

The hematologist and oncologist from UCSF Helen Diller Family Comprehensive Cancer Center discussed key findings from the phase 2 KarMMA trial.

A competing RNA-based therapy for LCA10 is currently being evaluated in a phase 2/3 trial.

CT-0508 is currently under investigation in a phase 1 clinical trial.

Directors from the Moran Eye Center discussed researching gene therapy approaches targeting HTRA1 in AMD.

CAP-1002 met primary and secondary end points in the HOPE-2 trial.

The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed JQ1 and other BET inhibitors he would like to study.

Precision BioSciences is collaborating with iECURE, a new company focused on mutation-agnostic, liver disease gene therapies.

The hematologist/oncologist from City of Hope discussed the potential for CAR T-cell therapy in earlier lines of treatment for patients with diffuse large B-cell lymphoma.

Patient-reported outcomes, 6-minute walk test, and NSAA scores all showed improvements over natural history data.

Overall response rate was 95%, including 75% of patients who achieved a complete response (CR) or better, with a median follow-up of 5.8 months at data cutoff.

Experts discuss the safety profile and cost of gene therapy for the management of sickle cell disease.

Sonny Hsiao, PhD, chief executive officer, president and cofounder, Acepodia, discussed the company’s cell therapy technologies.

The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease.

In 2 year follow-up data, 20 patients (19.4%) remained free from high-grade recurrence.

The associate professor from the UT Southwestern Medical Center Harold C. Simmons Comprehensive Cancer Center discussed the challenges faced with CAR T-cell therapy in patients with multiple myeloma.

Review top news and interview highlights from the week ending September 24, 2021.

The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.

The associate professor from The University of Texas MD Anderson Cancer Center discussed mitigating CAR T-cell therapy–related cytokine release syndrome in multiple myeloma.

Eric von Hofe, PhD, president and chief operating officer of AffyImmune, discussed advantages of the Affinity tuning platform.

Brian Culley, chief executive officer, Lineage Cell Therapeutics, discussed updated data from the phase 1/2 study of OpRegen.

Jan Davidson, MD, PhD, chief medical officer, Wugen, discussed the company’s future plans and research.