Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics have announced a significant milestone in medical science with the U.S. Food and Drug Administration’s (FDA) approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a revolutionary CRISPR/Cas9 genome-edited cell therapy. This approval marks a significant advancement in the treatment of sickle cell disease (SCD) for patients aged 12 and older experiencing […]
bluebird bio, Inc., a pioneer in the biotechnology industry, has announced the U.S. commercial infrastructure for LYFGENIA (lovotibeglogene autotemcel, also known as lovo-cel), an FDA-approved gene therapy for patients 12 years and older with sickle cell disease and a history of vaso-occlusive events (VOEs). The launch of LYFGENIA, leveraging bluebird’s experience in delivering ex vivo […]
Novo Nordisk acquisition of Forma Therapeutics : Danish pharma company Novo Nordisk has closed the previously announced $1.1 billion acquisition of Forma Therapeutics, a clinical-stage biopharma company focused on developing drugs for the treatment of sickle cell disease (SCD) and rare blood disorders. As per the terms of the deal announced last month, shareholders of […]
Pfizer has agreed to acquire Global Blood Therapeutics (GBT), a publicly-listed American biopharma company, in a deal worth around $5.4 billion with an aim to boost its presence in rare hematology. Global Blood Therapeutics is particularly focused on developing drugs for the treatment of sickle cell disease (SCD). The company’s sickle cell disease drug Oxbryta […]
Imara Inc. said that it has wrapped up enrollment of patients in the Ardent phase 2b clinical trial of IMR-687 (tovinontrine) for sickle cell disease. According to the US-based clinical-stage biopharma company, IMR-687 is a potent small molecule inhibitor of PDE9. Rahul Ballal — President and CEO of Imara, commenting on the Ardent phase 2b […]
Oxbryta FDA approval : Global Blood Therapeutics (GBT), a California-based biopharma company, has bagged approval for Oxbryta (voxelotor) tablets from the US Food and Drug Administration (FDA) for sickle cell disease (SCD). The FDA approval of Oxbryta is for the treatment of adults and children, aged 12 years of age and older, who are suffering […]
Adakveo FDA approval : Swiss drugmaker Novartis has bagged approval for Adakveo (crizanlizumab-tmca) from the US Food and Drug Administration (FDA) for its use in sickle cell disease. Formerly known as SEG101, Adakveo has been indicated to be used for bringing down the frequency of vaso-occlusive crises (VOCs), or pain crises, in patients, aged 16 […]