News

UCARTCS1A is the first allogeneic CAR T-cell product developed to target CS1 and SLAMF7, both of which are highly and consistently expressed in multiple myeloma.

The 4-day annual meeting of ISPOR—The Professional Society for Health Economics and Outcomes Research—will cover a wide range of topics. Here are 5 themes to keep an eye out for.

The low-dose cohort of RP-A501 conferred cardiac LAMP2B transgene expression.

The ongoing, phase 1/2 PrE0404 trial is evaluating the combination of ixazomib and ibrutinib in patients with relapsed/refractory mantle cell lymphoma with the goal of improving upon single-agent BTK inhibitor therapy in this patient population.

No adverse effects on weight, general activity, or survival in knockout mice and wild type littermates were observed throughout the study.

The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.

Following 2 unexpected serious adverse events, the trial was put on clinical hold as of February 2021.

The partnership will allow for the development of disease-specific AAV vectors that will serve Biogen's current gene therapy pipeline.

The therapy was generally well-tolerated and offered substantial pain relief in patients.

Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.

MB-106, a CD20-targeted CAR T-cell therapy that has shown promise in the treatment of B-cell non-Hodgkin lymphoma, is now being considered for patients with relapsed or refractory CD20-positive chronic lymphocytic leukemia.

A new study presented at ASGCT found that patients with indications of hemostatic efficacy experienced few adverse effects with gene therapy treatment BAY 2599023.

An autologous lentiviral-based gene therapy demonstrated significant benefit, including high overall and event-free survival rates, immune reconstitution, and metabolic correction, for patients with ADA-SCID.

Researchers observed 20-25% frequencies of this 4.9 kb deletion in HSPCs prior to xenotransplantation and at 17 weeks post-engraftment.

Nina Shah, MD, discusses the newfound role of BCMA-directed CAR T-cell therapies in multiple myeloma, how the field may select between ide-cel and cilta-cel if the latter is approved, and novel cellular therapies in clinical development.

Early results from the MOMENTUM study show that treated patients experienced fewer vaso-occlusive episodes and related hospital admissions.

In a presentation at ARVO, Friederike Kortuem, MD, MSc, explains that treatment with voretigene neparvovec-rzyl led to a short-term change in the foveal morphology in a patient with visual impairment that included nyctalopia and decreased visual acuity in early childhood.

Strategies for improving outcomes across hematologic cancers and solid tumors range from addressing cytokine release syndrome and neurotoxicity mediators, immune rejection, on-target off-tumor toxicity, post-infusion control limitation, and immunosuppressive tumor microenvironment.

A podcast interview with study author Paul Yang, MD, PhD, on the current research and future implementation of the agent.

Arsen Osipov, MD, discusses the potential for ongoing research with immunotherapeutic approaches in gastrointestinal malignancies, including CAR T-cell therapy, bispecific T-cell engagers, and vaccines.

Based on findings from the SPEARHEAD-1 trial, Adaptimmune plans to submit a biologics license application to the FDA in 2022 for afamitresgene autoleucel to treat synovial sarcoma.

Recent Foundation for Angelman Syndrome Therapeutics grants funded the hotline as well as work to develop cell line models for rarer genotypes of AS.

Investigators used an adeno-associated viral vector to deliver a normal functioning copy of the RPGR gene via subretinal injection.

Lineage Cell Therapeutics CEO Brian Culley shares a clinical trial update on their leading cell therapy candidate and discusses the important role of the FDA as more players enter the cell and gene therapy space.

The single-injection investigative gene therapy may help with providing continuous expression of aflibercept.

Dr. Abramson discusses the significance of the FDA approval of lisocabtagene maraleucel in refractory large B-cell lymphoma and provided insight into the efficacy and safety profiles of the CAR T-cell therapy as reported in the TRANSCEND NHL 001 trial.

The AAV8 vector–based gene therapy was associated with improvements in 2 measures of visual function.

A non-viral gene therapy sustained drug-delivery product that delivers anti-VEGF to the eye may replace the need for repeated intravitreal anti-VEGF injections and improve vision in patients with wet AMD.

Optical coherence tomography characteristics may be predictive of the efficacy of intravitreal injection of allogeneic human retinal progenitor cells, according to research presented at ARVO 2021.

The full regulatory approval of agalsidase beta has blocked the accelerated approval pathway for AVR-RD-01 as a treatment for patients with Fabry disease.