News

Lifileucel elicited objective responses, including complete responses, for a median duration that was not yet reached at 28.1 months for patients with advanced melanoma.

Studies are uncovering a range of potential treatment options for disorders.

CD19-directed CAR T-cell therapy brexucabtagene autoleucel will be considered by FDA for indication in adults with B-cell precursor acute lymphoblastic leukemia.

Daratumumab appears to be safe when used as consolidation therapy in patients with multiple myeloma, even soon after autologous stem cell transplant, and the agent has also demonstrated early efficacy in the setting of very good responders.

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.

A budget impact analysis suggest the burgeoning drug class may not be as available as it could be for sickle cell patients.

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.