News

Following GS030 optogenetic therapy, the first treated patient was able to locate and count objects on a table and could identify crosswalks in the street.

The findings, which were simultaneously reported in the New England Journal of Medicine, support beti-cel as a potentially curative, one-time treatment option for these patients.

ADI-001 does not require genetic engineering to remove TCRs to avoid GvHD, making them ideal for an off-the-shelf cell therapy.

The hematologists from Moffitt Cancer Center and University of Texas MD Anderson Cancer Center discussed navigating the referral process for CAR T-cell therapy in hematologic malignancies.

Patients treated with the CAR T-cell therapy had an over 4-fold increase in event-free survival.

Review top news and interview highlights from the week ending December 9, 2021.

All evaluable treated participants experienced a reduction in vaso-occlusive events after treatment with ARU-1801.

CSL Behring plans to submit regulatory applications in the US and EU in the first half of 2022 for the AAV gene therapy etranacogene dezaparvovec.

The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in common cancers and its use as a biomarker.

Autologous adipose stem cell data in a small cohort provides a feasibility for future validation studies.

The hematologists from Moffitt Cancer Center and MD Anderson discussed managing CAR T-cell therapy-associated toxicities.

Current studies are using a polygenic risk score to evaluate the risk and clinical outcomes in primary open-angle glaucoma (POAG).

Adicet Bio reported positive interim data from the phase 1 GLEAN study.

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the company’s goal to reprogram the immune system.

The companies will combine Imugene’s CF33-CD19 oncolytic virus and Celularity’s investigational placental-derived CAR T-cell therapy.

Findings from the phase 2 INFINITY trial identified dose-dependent safety outcomes of ADVM-022 in patients with diabetic macular edema.

Further understanding of how COVID-19 vaccinations affect CAR T-cell therapy recipients is needed.

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, trial data, and further research.

Julio Chavez, MD, MD, discussed treatment options for LBCL after relapsing on CAR T-cell therapy.

The professor from National Taiwan University Hospital discussed gene therapy studies in AADC deficiency, Parkinson disease, SMA, and Pompe disease.

Review top news and interview highlights from the week ending December 3, 2021.

The cell therapy is approved for B-cell non-Hodgkin lymphoma and B-ALL in China.

The hematologist/oncologist from Mayo Clinic discussed the BASECAMP study and how it will inform future studies assessing Tmod technology.

An independent Data Safety and Monitoring Board has recommended that the phase 1 RePAIR trial continue at an accelerated pace.

The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.

Two sites in California and Missouri are actively recruiting patients with r/r LBCL who were treated with tisagenlecleucel to receive efineptakin alfa in the study.

Beam Therapeutics will assess the safety and efficacy of BEAM-101 for SCD in the phase 1/2 BEACON-101 study.

ALSFRS-R and FVC values were stable 3 months after treatment but significantly decreased 6 months after treatment, warranting further investigation.