News

The rolling submission of a biologics license application to support the approval of the investigational BCMA-directed CAR T-cell therapy ciltacabtagene autoleucel for the treatment of patients with relapsed/refractory multiple myeloma has been completed.

Physician offers patient counseling pearls for selected retinal diseases.

AAV2 TrkB-2A-mBDNF resulted in early signs of efficacy in preclinical studies of glaucoma and humanized tauopathy, which could be translatable to other neurodegenerative polygenic disorders.

As more therapies continue to be approved, efforts will focus on toxicity prevention and mitigation strategies for associated unique and potentially life-threatening adverse reactions.

The multitumor-associated antigen-specific T-cell product MT-401 is under investigation as a potential treatment option for patients with acute myeloid leukemia following allogeneic stem cell transplant in both the adjuvant and active disease settings.

CAR T-cell therapies are now incorporated into National Comprehensive Cancer Network guidelines as a recommended third-line strategy in this setting, with the most recent addition being lisocabtagene maraleucel.

The Canada Foundation for Innovation has awarded C$5,187,685 to the Canadian Cancer Trials Group at Queen’s University in Kingston, Ontario, Canada, to fund the infrastructure for a new research platform to coordinate the development of new cancer cell therapies.

Enrollment has completed for the phase 3 GEM-3 study for beremagene geperpavec for patients with dystrophic epidermolysis bullosa. Topline results are anticipated before the end of 2021.

Novel gene therapy demonstrates improved visual acuity and retinal thickness.

The AAV-mediate gene therapy BS01 allowed 4 patients with retinitis pigmentosa who had complete or near-complete blindness to perceive light and motion.

Follow-up of 4.8 years to a phase 1 trial demonstrated that deep and durable responses are attainable in a large cohort of children and young adult patients with B-cell acute lymphoblastic leukemia who were treated with CD19-directed CAR T-cell therapy followed by allogeneic hematopoietic stem cell transplantation.

Multiple myeloma is currently incurable with many patients experiencing relapses.

Etranacogene dezaparvovec was "highly unlikely" to be the cause of hepatocellular carcinoma in a patient with hemophilia B enrolled in the phase 3 HOPE-B trial, according to an independent investigation.

The FDA has approved idecabtagene vicleucel as the first BCMA-directed CAR T-cell therapy for patients with relapsed/refractory multiple myeloma after 4 or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

The tumor infiltrating lymphocyte therapy LN-145 was feasible, safe, and effective in patients with pretreated advanced cervical cancer.

The FDA has granted an orphan drug designation to the CD34+ cell therapy CLBS12 as a potential treatment for patients with thromboangiitis obliterans.

In 2021, Abeona Therapeutics is anticipating to complete enrollment in its phase 3 VIITAL study and additional findings for ABO-102 and ABO-101.

NurOwn was safe and led to improvements in function and cognition at 28 weeks compared with baseline for patients with progressive multiple sclerosis.

As a multi-systemic disorder, Fabry disease can impact the functions of various organs. As a genetic disorder, Fabry can impact the offsprings, parents, and relatives of the patient. Therefore, its effects are far-reaching.

RP-L301 demonstrated improvements in hemoglobin at 3- and 6-months for the first 2 patients treated in a phase 1 study for pyruvate kinase deficiency.

The FDA has granted a fast track designation to the placental-derived natural killer cell therapy CYNK-001 as a potential treatment for adult patients with recurrent glioblastoma multiforme.

Penn Medicine's Stephen Schuster, MD, offers an overview of an eventful year in CAR T-cell therapy at the NCCN Virtual Annual Meeting.

SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.

SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.

Matthew Lunning, DO, discusses the findings from pivotal CAR T-cell therapy trials in LBCL, the potential to utilize these agents in the outpatient setting, how off-the-shelf allogeneic products could further transform outcomes, and the importance of shortening brain-to-vein time for this patient population.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.

Ninety percent of patients with cerebral adrenoleukodystrophy who were treated with elivaldogene autotemcel were alive and free of major functional disabilities at 2-years.

AB-205 showed an encouraging safety profile along with a robust ability to eliminate oral and gastrointestinal severe regimen-related toxicities in patients with systemic lymphoma who were undergoing high-dose therapy and autologous hematopoietic stem cell transplantation.