Kelly Davio

While antiretroviral therapy (ART) can suppress HIV infection, ART cannot completely eradicate HIV, which remains in a latent reservoir in CD4-positive T cells during treatment; discontinuation of ART leads to rapid rebound of the virus. This reservoir forms even when ART is initiated early on in the infection, and while the most widely accepted model of how the reservoir forms involves infection of a CD4-positive T cell as it transitions to a resting state, the dynamics and timing of the reservoir’s formation have been largely unknown.

The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc’s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy, an X-linked genetic disorder caused by a defect in the gene ABCD1.

Drug company bluebird bio, Inc, has announced the publication of positive interim data for its gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent β-thalassemia.

Pfizer has dosed its first patient in a phase 1 clinical trial of an investigational gene therapy for the treatment of patients with Duchenne muscular dystrophy, a genetic disease that results in the absence of dystrophin, a protein that helps to keep muscle cells intact.

New research suggests that the composition of bacteria in the gastrointestinal tract may help predict which patients with cancer may benefit most from adoptive T-cell immunotherapy, including chimeric antigen receptor T-cell therapy.

On the closing day of the National Comprehensive Cancer Network (NCCN) 23rd Annual Conference in Orlando, Florida, Bijal Shah, MD, of Moffitt Cancer Center, presented on acute lymphoblastic leukemia (ALL), and on the lessons learned from the application of chimeric antigen receptor (CAR) T-cell therapy in this indication.

Researchers have identified a molecular target that could allow chimeric antigen receptor (CAR) T-cell therapy to be used in treating patients with glioblastoma. Although the heterogeneous expression of tumor-associated antigens limits the efficacy for CAR-redirected T cells for the treatment of glioblastoma, chondroitin sulfate proteoglycan 4 (CSPG4), a cell surface type 1 transmembrane protein, is highly expressed in a majority of glioblastoma specimens with limited heterogeneity.

Fibrocell Science has submitted an Investigational New Drug Application (IND) with the FDA for a gene therapy candidate to treat moderate to severe localized scleroderma.

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

CD19 CAR T cells are highly effective in high-risk patients with chronic lymphocytic leukemia who have failed to respond to treatment with ibrutinib, a Bruton's tyrosine kinase inhibitor, according to a new study.