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New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.

The assistant professor from University of Nebraska Medical Center discussed the current limitations of CAR T-cell therapy in multiple myeloma.

REGENXBIO previously announced positive data in patients with wet AMD who were treated with the same gene therapy.

Sarepta Therapeutics has also initiated the pivotal phase 3 EMBARK study in pediatric patients with DMD.

Jake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s technology and focus on mRNA therapies.

Intellia Therapeutics is also initiating a phase 1/2 study of a CRISPR therapy for hereditary angioedema in New Zealand.

The hematologist from Moffitt Cancer Center discussed the FDA approval of brexucabtagne autoleucel in relapsed/refractory B-cell acute lymphoblastic leukemia.

Longer follow-up is needed to determine the potential of CAR T-cell therapies in difficult-to-treat malignancies.

The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

Allen Feng, PhD, chief scientific officer, HebeCell, discussed the company’s NK cell technologies.

Data from the FORTE trial were presented at the 18th International Myeloma Workshop.

The hematologist/oncologist discussed unmet needs and novel therapies in multiple myeloma and give practical advice for community oncologists.

Review top news and interview highlights from the week ending October 8, 2021.

Allogene continues to investigate and characterize the chromosomal abnormality and its relationship to the gene editing.

The director of the Powell Gene Therapy Center at the University of Florida discussed adverse events in gene therapies.

Thorsten Graef, MD, PhD, chief medical officer, Acepodia, discussed data on ACE1702 presented at ESMO 2021.

Forge Biologics and Solid Biosciences have also partnered to help develop a gene therapy for Duchenne muscular dystrophy.

Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies.

Investigators have identified the maximum tolerated dose and continue to refine the recommended phase 2 dose in the phase 1 part of the study.

The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed safety concerns with CAR T-cell therapies.

Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.

The chair of the International Collaborative Gaucher Group Registry discussed the place of gene therapies in the Gaucher treatment landscape.

Overall response rate was 73% in all patients treated with ide-cel at a median follow-up of 24.8 months (range, 1.7-33.6).

The chief executive and chief medical officers of Vor Biopharma discussed the company’s future research and plans.

Patients treated with RGX-314 also had stable BCVA and central retinal thickness.

ZUMA-3 showed that a single infusion of the CAR T-cell therapy elicited a high and durable response rate in heavily pretreated patients with relapsed/refractory B-ALL.

The associate professor from The University of Texas MD Anderson Cancer Center discussed eligibility for autologous stem cell transplant in patients with multiple myeloma.

A clinical hold was previously placed after patients treated with the highest dose developed lower extremity weakness.