News

The Expanded Access Program will offer treatment for patients with ALS who have completed the phase 3 clinical trial and will not interfere with data or regulatory timelines.

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

ViGeneron and Biogen this week have announced a global collaboration and licensing agreement in which the partners will develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors that treat inherited eye disease.

James Gerson, MD, discusses the introduction of CAR T-cell therapy to the mantle cell lymphoma armamentarium and how induction therapy followed by stem cell transplant has maintained a role.

Genentech's tiragolumab, a novel immunotherapy for non-small cell lung cancer with PD-L1 expression, is the first anti-TIGIT therapy to be granted Breakthrough Therapy Designation.

Nivolumab was previously granted accelerated approval by the FDA for the treatment of patients with small cell lung cancer (SCLC) whose disease had progressed after platinum-based chemotherapy and at least 1 other line of therapy, but phase 3 trial results led to a decision to withdraw the indication.

January 4, 2021 - The biologics license application for the CAR T-cell product lisocabtagene maraleucel in adult patients with relapsed/refractory large B-cell lymphoma following at least 2 previous therapies continues to be under regulatory review by the FDA, and a decision on the application has not yet been reached.

Nine pediatric patients with Leber congenital amaurosis show visual improvement in clinical trials.

Researchers from the University of Turin also found that an NEFH variant showed a protective association with ALS.

Gene-editing therapy in sickle cell disease and advances in multiple myeloma dominated hematology news in 2020.

Readers favored news about ibrutinib, the first chimeric antigen receptor (CAR) T-cell therapy in mantle cell lymphoma, and the effect of the pandemic on patients with chronic lymphocytic leukemia.

In a new review, researchers outline both the underlying pathological mechanisms and potential therapeutic targets in pulmonary arterial hypertension (PAH).

December 21, 2020 - A rolling submission of the biologics license application for the BCMA-directed CAR T-cell product ciltacabtagene autoleucel for use in adults with relapsed/refractory multiple myeloma has been initiated to the FDA.

Results presented earlier this month at ASH showed a 97% overall response rate, and progression-free survival had not been reached.

Full approval has been granted by the FDA to osimertinib (Tagrisso) for use as adjuvant therapy following tumor resection among patients with non–small cell lung cancer whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations.

December 18, 2020 - Sequential immunotherapy with rituximab-based therapy and the addition of lenalidomide proved effective among patients with TP53 wild-type mantle cell lymphoma, even among those with a Ki-67 level of at least 30% and/or blastoid morphology.

Javier L. Munoz, MD, discusses the nuances of choosing treatment options for patients with CLL in the frontline and relapsed/refractory settings, as well as novel therapeutic strategies on the horizon.

Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.

December 17, 2020 — The European Commission has granted a conditional marketing authorization to the CD19-targeted CAR T-cell therapy KTE-X19 for use in adult patients with relapsed or refractory mantle cell lymphoma who had previously received 2 or more lines of systemic therapy including a BTK inhibitor.

With several clinical trials underway, ocular therapy is seen as having potential in the treatment of a number of gene-related diseases.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.

A new meta-analysis finds high rates of efficacy and high rates of toxicity in multiple myeloma patients treated with B-cell maturation agent (BCMA)-targeted chimeric antigen receptor (CAR)- T cells.

Checkpoint inhibitors have failed to improve progression-free survival and overall survival as second-line therapy and maintenance therapy in small cell lung cancer, but this class of agents continue to show encouraging activity worthy of a paradigm shift up front.

The findings help explain why costly CAR T-cell therapy does not work for some patients.

The UCSF hematologist discusses the growing portfolio for the sickle cell therapy after ASH 2020.

The historic phase 3 HOPE-B trial shows all but 1 treated patient discontinued prophylaxis after a single dose of etranacogene deaparvovec.

The second process of ARU-1801 in a new patient resulted in up to 4 times greater engraftment of transduced hematopoietic stem cells and progenitors at 6 months.

Research presented at the 2020 ASH Annual Meeting may have found an alternative path forward for patients who do not respond to immunotherapy treatment for large B-cell lymphomas.