sickle cell disease

History has been created as the world captures the significance of the FDA’s approval of Casgevy (exa-cel), a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics, for the treatment of sickle cell disease (SCD). This groundbreaking therapy represents a long-awaited potential cure for the debilitatin...

Merck and Moderna Initiate INTerpath-002, a Phase III Study Evaluating V940 in Combination with KEYTRUDA for Adjuvant Treatment of Patients with Certain Types of Resected NSCLC Merck (also known as MSD outside the United States and Canada) and Moderna, Inc. have commenced the INTerpath-002 trial—a crucial Phase ...

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

Lexicon Announces FDA Approval of INPEFA (Sotagliflozin) For Treatment of Heart Failure Lexicon Pharmaceuticals, Inc. announced that the FDA has approved INPEFATM (sotagliflozin), a once-daily oral tablet, to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure vis...

Vertex Pharma and CRISPR Therapeutics are the first companies to seek FDA clearance for a gene-editing therapy. Vertex Pharmaceuticals and CRISPR Therapeutics have gotten closer to introducing exagamglogene autotemcel (exa-cel), a one-time treatment for sickle cell disease (SCD) and transfusion-dependent beta-...

FDA Approves Eli Lilly’s Jaypirca for Relapsed Mantle Cell Lymphoma Eli Lilly has received FDA approval for Jaypirca, a non-covalent BTK inhibitor, in relapsed mantle cell lymphoma (MCL) patients who have relapsed after treatment with other drugs in the class. Adult MCL patients who have previously received at l...

FDA Approves AstraZeneca’s Imfinzi for Biliary Tract Cancer Imfinzi, a checkpoint inhibitor developed by AstraZeneca, has been approved by the FDA as the first immunotherapy for biliary tract cancer (BTC), a rare and aggressive form of cancer with few treatment options. Imfinzi (durvalumab) has been approved by ...

FDA Approves AstraZeneca’s Enhertu as First Targeted Therapy for HER2-low Cancer Enhertu, developed by AstraZeneca and Daiichi Sankyo, has been approved by the FDA as the first targeted therapy for HER2-low breast cancer, a new subtype of the disease previously categorized as HER2-negative. It has the potential ...

Immunocore Receives FDA Approval for the First Uveal Melanoma Treatment, As Well As the First T-cell Receptor Therapeutics Uveal Melanoma, an aggressive eye cancer, has proven to be a tough nut to crack for researchers seeking a cure. However, with the approval of a new treatment, those with the disorder will ha...

Global Therapeutics and Syros Pharmaceuticals have teamed up to move forward to discover, develop and market novel Sickle cell disease and beta-thalassemia therapies.  Inherited blood disorders affect millions of people worldwide. As per NIH, Sickle cell disease affects around 70,000 to 80,000 Americans, wh...