Hereditary Disease

Bluebird Bio secures up to $175M in debt financing

By LabPulse.com staff writers

Bluebird Bio has secured up to $175 million in debt financing with Hercules Capital, having entered into a five-year term loan facility that provides funds in separate tranches.

March 18, 2024

Bluebird Bio signs state Medicaid agreement for SCD gene therapy

By LabPulse.com staff writers

Bluebird Bio has signed its first outcomes-based Medicaid agreement for its sickle-cell disease (SCD) gene therapy Lyfgenia with the state of Michigan.

March 11, 2024

CRISPR-repaired T cells cure lethal hyperinflammation in mice

By Nick Paul Taylor

Memory T cells repaired using CRISPR-Cas9 can cure lethal hyperinflammation in mice, suggesting the approach may improve outcomes in a disease with a five-year probability of survival of around 50%.

February 2, 2024

Gene therapy, companion diagnostic hit milestones for hemophilia A

By Liz Carey

Six months after the FDA approved a one-time gene therapy for severe hemophilia A reportedly priced around $2.5 million per patient, the Center for Inherited Blood Disorders, a 340B treatment center in California, has announced the drug's first infusion.

January 11, 2024

FDA approves CRISPR-based gene therapy, cell-based gene therapy for SCD

By LabPulse.com staff writers

The FDA has approved Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene-editing therapy for the treatment of VOCs in patients with sickle cell disease (SCD). It is the first CRISPR-based gene therapy to receive FDA approval.

December 8, 2023

U.K. regulators approve 'world-first' sickle-cell gene therapy

By Matt Limb

U.K. medical regulators have approved a “world-first” gene therapy that aims to cure two blood disorders: sickle cell disease and transfusion-dependent β-thalassemia. They are the first regulatory body to approve the treatment.

November 20, 2023

Vertex Pharmaceuticals, CRISPR Therapeutics gene-editing therapy to undergo FDA advisory committee review

By LabPulse.com staff writers

An FDA advisory committee is scheduled to meet on October 31 to discuss and make recommendations on Vertex Pharmaceuticals' and CRISPR Therapeutics’ CRISPR-Cas9 gene-editing-based therapy, exagamglogene autotemcel.

October 30, 2023

CMS opens new doors for cell and gene therapy under Medicaid

By Liz Carey

A new Medicaid-focused Innovation Center model encourages agreements between pharmaceutical companies and states for cell and gene therapies (CGT).

October 18, 2023

U.K. charity to offer $1.6M in grants for research on muscle-wasting diseases

By Matt Limb

U.K. research projects will benefit from 1.3 million pounds ($1.6 million) in grants to improve diagnostic testing and potential new treatments for muscle-wasting and -weakening conditions.

September 14, 2023

Identification of hundreds of blood pressure genes lays foundation for hypertension drugs

By Nick Paul Taylor

Researchers have found hundreds of genes that may affect blood pressure, setting the stage for further studies that could inform the development of new treatments for hypertension.

September 12, 2023

Messenger RNA shows preclinical promise as in vivo gene editing treatment for sickle cell

By Nick Paul Taylor

The delivery of the editing system resulted in the near-complete correction of hematopoietic sickle cells. A preclinical test linked the approach to a 91.7% increase in functional hemoglobin and a significant fall in the number of sickled cells.

August 14, 2023

Analysis of 462M lipid test results reveals global variation in cholesterol levels

By Nick Paul Taylor

Mean total cholesterol levels exceeded the World Health Organization (WHO) target in seven of the 17 countries included in the analysis, namely Japan, Australia, North Macedonia, Switzerland, Germany, Slovakia, and Austria.

July 17, 2023

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