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Published on April 25, 2023
Bluebird bio has submitted to the FDA for approval of its sickle cell gene therapy. It’s a one-time treatment that adds a functional β-globin gene into the patient’s own hematopoietic (blood) stem cells. Bluebird’s move comes soon after Vertex and CRISPR Therapeutics submitted for their gene editing therapy in the…
Published on September 19, 2022
Just a month after they nabbed approval for Zynteglo to treat beta thalassemia, bluebird’s SKYSONA (elivaldogene autotemcel), also known as eli-cel, has been granted accelerated approval by the FDA. The company says the drug slows progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy…
Published on June 14, 2022
Just one day after it received a positive recommendation for one gene therapy, bluebird got another. This one is for betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions. The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted…
Published on June 10, 2022
A U.S. FDA advisory committee unanimously approved bluebird bio’s elivaldogene autotemcel (eli-cel) gene therapy for early active cerebral adrenoleukodystrophy (CALD) in certain patients. The committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a PDUFA goal date set for September 16,…
Published on March 8, 2022
Bluebird acknowledged Monday in a regulatory filing that Gina Consylman, the company’s CFO and treasurer, had given notice Friday of her resignation, effective April 3. Bluebird shares finished trading at $5 a share, down from $13.14 on November 15, when the post-spinoff Bluebird Bio started trading its shares. Shares of…
Published on February 17, 2021
Bluebird Bio said it has temporarily and voluntarily suspended two clinical trials assessing its LentiGlobin gene therapy for sickle cell disease (SCD; bb1111) following reports that two participants in the earlier-phase study developed blood cancers—one of acute myeloid leukemia (AML), the other of myelodysplastic syndrome (MDS). Bluebird said it temporarily…
Published on February 7, 2024
Sickle cell disease (SCD) will be the first focus of the U.S.’s new Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cell and gene therapies for vulnerable populations. Gene therapies are some of the most expensive treatments in the world. The Centers for Medicare &…
Published on January 17, 2024
The U.S. Food and Drug Administration (FDA) has approved Vertex and CRISPR Therapeutics’ Casgevy (exa-cel), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the latest sign the gene and cell therapy market is maturing. This market is…
Published on December 11, 2023
In a landmark moment for sickle cell disease treatment and CRISPR, two gene therapies were approved for the condition by the U.S. FDA. One of them,Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]), is the first ever FDA approved CRISPR/Cas9 genome-edited cell therapy. Simultaneously, Bluebird Bio received the FDA OK…
Published on October 17, 2023
By William A. Haseltine Regenerative medicine has emerged as a promising treatment option for chronic medical conditions. Specifically, cell and gene therapies, innovative forms of regenerative medicine, have shown great potential in personalizing and targeting disease treatment. These cutting-edge therapies leverage a patient’s cells and genes to repair and regenerate…
Published on May 18, 2022
Kriya Therapeutics has announced a $270 Million Series C financing that will support advancement of the company’s pipeline and continued scaling of its engineering, manufacturing, and computational platforms. Kriya has a portfolio of gene therapies with initial focus areas in ophthalmology, oncology, rare disease, and chronic disease. “We believe gene…
Published on April 20, 2021
Nearly six years after announcing a potentially $2.5 billion therapeutic development collaboration followed by an additional up to $1.175 billion agreement in 2019, Vertex Pharmaceuticals has, once again, thrown more money into the pot for CRISPR Therapeutics to lead the development, manufacturing, and commercialization of CTX001, the CRISPR-Cas9 gene-edited therapy…
Published on March 29, 2021
The FDA has approved Bristol Myers Squibb’s and bluebird bio’s CAR-T cell therapy for treating the blood cancer multiple myeloma, the first cell-based gene therapy approved for this indication and is the first B-cell maturation antigen (BCMA)-directed therapy. Abecma (idecabtagene vicleucel) was approved for patients with multiple myeloma whose cancer…
Published on March 9, 2021
Bristol Myers Squibb and bluebird bio’s autologous CAR T-cell therapy for multiple myeloma, idecabtagene vicleucel, succeeded in inducing at least a partial response in 73% of treated patients in a recent Phase II trial, with 33% having a complete response or achieving remission. Multiple myeloma is a cancer of the…
Published on December 8, 2020
The CRISPR-Cas9 gene-edited therapy CTX001 has shown a consistent and sustained positive response in 10 patients treated for a pair of blood disorders—sickle-cell disease (SCD) and beta thalassemia—reported CRISPR Therapeutics and Vertex Pharmaceuticals the developers of the therapy. These are the first clinical studies of a CRISPR gene-editing candidate sponsored…