News

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

Treatment with the first-generation precision cell therapy Orca-T led to a significant reduction in cases of graft-versus-host disease, an impressive GVHD relapse-free survival rate, a lack of treatment-related mortalities, while showcasing scalability potential.

Study examining the role of IL-17A in patients with diabetes.

The HSPC therapy showed fetal hemoglobin production in transfusion-dependent patients in a matter of months—with sustained results over 1 year.

Treatment with the investigative gene cell therapy betibeglogene autotemcel led to durable transfusion independence in 87% of pediatric patients less than 18 years of age with transfusion-dependent beta-thalassemia with a median average hemoglobin of 11.3 g/dL.

Investigators focus on biophysical method to study protein-protein interactions .

Responsiveness to treatment received immediately prior to CAR T-cell therapy may not be associated with post–CAR T outcomes in patients with relapsed/refractory diffuse large B-cell lymphoma who receive axicabtagene ciloleucel.

FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.

Patients with refractory large B-cell lymphoma have less to gain from axicabtagene ciloleucel if they have never achieved a complete response to any line of prior therapy.

Data from the ongoing HGB-206 study reports reduced hemolysis, increased total hemoglobin, and a complete resolution of vaso-occlusive crises among patients.

New WORLDSymposium findings show a treated patient had 100% reduction in the accumulating fatty substrate associated with the burdensome rare disease after 1 year.

Prior exposure to anti-CD19 therapy did not impact response to the CAR T-cell therapy lisocabtagene maraleucel in patients with relapsed/refractory large B-cell lymphoma.

These findings may have implications for patients with hematologic malignancies, suggesting the development of protective memory against SARS-CoV2 and non-SARS human coronaviruses.

February 5, 2021 - Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 tend to have favorable outcomes, especially if they are diagnosed in complete remission and further out from their cell infusion.

A CAR T-cell therapy that promises fewer side effects, and possibly lower hospital costs, wins approval after lengthy delays.

The 1 patient who did not received immunomodulatory agents demonstrated the highest level of FVIII activity.

Although safety remains a concern with gene therapy, investigators are breaking ground in cell and gene therapy, and many believe that ultimately, a string of cured cancers will follow.

February 3, 2021 - The combination of ceralasertib plus durvalumab improved overall response rate in patients with non‒small cell lung cancer who progressed on an anti-PD-1/PD-L1 therapy.

Neil Berinstein, MD, discussed the early promise with this T-cell therapy in relapsed/refractory diffuse large B-cell lymphoma.

Neurocrine Biosciences and Voyager Therapeutics have terminated the Parkinson disease portion of their partnership, ending development for VY-AADC, also known as NBIb-1817.

A substantial proportion of patients with inherited retinal disease could be treated with base editing, while therapeutic strategies that focus on common variants could be used to treat a large number of patients with the disease, according to study results.

Clinically relevant scores of 32 and 44 on the Fugl-Meyer Motor Score were documented in 2 patients who received NSI-566.

An engineered lentivirus was created to avoid some of the drawbacks associated with current gene therapy vectors for sickle cell disease (SCD) and beta-thalassemia.

January 28, 2021 — Frontline pembrolizumab continued to demonstrate clinically meaningful improvements in overall survival, overall response rate, and time to progression on next-line therapy compared with platinum-based chemotherapy in patients with locally advanced or metastatic PD-L1–positive non–small cell lung cancer without sensitizing EGFR or ALK mutations.

While these therapies continue to revolutionize the treatment landscape of SMA, treatments are not without shortcomings or challenges.

Two gene therapy techniques in separate, concurrently published trials demonstrate clinical success in mitigating monogenic hemoglobinopathies.