News

Review top news and interview highlights from the week ending January 14, 2022.

Exciting new therapies for sickle cell disease are on the horizon. We explored some of the most promising investigational therapies in recognition of National Blood Donor Month.

Six of 7 patients reported improvements in dry mouth after treatment with AAV-hAQP1.

Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the phase 1/2 study evaluating MDG1011.

Genprex will launch the open-label, multicenter, phase 1/2 Acclaim-2 trial in the first quarter of 2022.

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed improving outcomes in hemophilia with fitusiran.

ADVM-062 is designed to deliver a functional copy of the OPN1LW gene to the foveal cones of patients with BCM.

AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed further research with tab-cel, including study 205.

Many cell and gene therapy companies are initiating new programs and collaborations with the start of the new year.

The Director of the Islet Transplant Program at the University of Alberta discussed the implantable VC-02 device's success in type 1 diabetes.

The placebo crossover cohort experienced statistically significant improvements in NSAA score over external control.

The reprioritization comes as the SIG-001 program remains on clinical hold after scarred and inviable cell spheres were observed in a treated patient.

The deputy director and chief of stem cell transplantation at Miami Cancer Institute discussed the study of axicabtagene ciloleucel vs tisagenlecleucel in relapsed or refractory diffuse large B-cell lymphoma.

Reductions in annualized bleeding rate remained significant at 2 years in the phase 3 study although recently presented phase 2 data showed significant FVIII reductions at 5 years.

The hematologist/oncologist from Mayo Clinic discussed the growing presence of CAR T-cell therapies in lung cancer and melanoma.

Review top news and interview highlights from the week ending January 7, 2022.

AVRIOBIO announced that it is shifting priorities to focus on their gene therapy programs in Gaucher disease.

Findings from the phase 2 ENSIGN and ELIANA studies suggest DNA sequencing predicts ALL relapse.

The DSMB has recommended that enrollment expand and doses be escalated from 3.3 to 5 mg in 1 cohort of the KIK-AS study.

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the long-term follow-up and FREEDOM-1 trials of FCR-001.

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed gene therapy programs in GM1 gangliosidosis and Fragile X syndrome.

Event-free survival was 68.3% at 6 months, 48.3% at 12 months, and 48.3% at 24 months in patients treated with AUTO1.

The hematologist/oncologist from Rutgers Cancer Institute of New Jersey discussed ongoing research examining the CAR T-cell therapy ciltacabtagene autoleucel.

The phase 3 GEM-3 study met both its primary and secondary end points compared with placebo in dEB.

The chief of Cancer Immunotherapy at Rutgers Cancer Institute of New Jersey discussed unmet needs with cellular therapies in metastatic epithelial cancer.

Data from a phase 1/2 trial of AMT-130 supports continued enrollment for a higher-dose cohort of 6 x 1013 vg.

The hematologists from Moffitt Cancer Center and MD Anderson Cancer Center discussed the need for collaboration between academic and community practices for patients who receive CAR T-cell therapy.

Christopher R. Flowers, MD, reviewed challenges of treating indolent B-cell lymphoma.

The director of the Buffalo Neuroimaging Analysis Center and professor of Universtiy of Buffalo discussed avenues of research into reversing neurodegeneration in MS.

Patients in the liso-cel arm had more favorable overall least square mean changes from baseline to day 126 vs the SOC arm in quality of life scores.