Anemia, Sickle Cell

Agios’ Sickle Cell Trial Falls Short as Mitapivat Fails to Significantly Reduce Pain Crises

3 weeks ago talkbio0Tagged Agios, Anemia, Sickle Cell, Clinical Trials, hemoglobin response, mitapivat, pain crises, Pyrukynd, Rise Up trial

Agios; mitapivat; Pyrukynd; sickle cell disease; pain crises; clinical trial; Rise Up trial; hemoglobin response

Pressure On, Purpose Questioned as Second Round of FDA Priority Review Vouchers Revealed

3 weeks ago talkbio0Tagged Anemia, Sickle Cell, Biosimilars, Commissioner 's National Priority Voucher, Drug Approval, drug price, eli lilly, erbB-2 Receptor, Malignant neoplasm of lung, Obesity, Priority Review, public health medicine (field), Tuberculosis, Drug-Resistant, United States Food and Drug Administration, voucher program

FDA; Commissioner’s National Priority Voucher; priority review; drug approval; voucher program; Eli Lilly; Novo Nordisk; her2 lung cancer; obesity drugs; drug-resistant tuberculosis; sickle cell disease; biosimilars; public health; drug pricing; regulatory controversy

bluebird bio Rebrands as Genetix Biotherapeutics Following Private Equity Buyout

3 months ago talkbio0Tagged Anemia, Sickle Cell, Bluebird Bio, Capital Partners, Carlyle, Cerebral Adrenoleukodystrophy, commercial, David Meek, DKK1 gene, execution, FDA-approved therapies, gene therapy, Genetix Biotherapeutics, Investments, Manufacturing, Private Equity, rebranding, Thalassemia

bluebird bio; Genetix Biotherapeutics; private equity buyout; Carlyle; SK Capital Partners; gene therapy; rebranding; FDA-approved therapies; David Meek; sickle cell disease; β-thalassemia; cerebral adrenoleukodystrophy; commercial execution; manufacturing investment

NHS England Approves Groundbreaking CRISPR Gene Therapy for Sickle Cell Disease

10 months ago talkbio0Tagged Anemia, Sickle Cell, Approved, Casgevy, Clustered Regularly Interspaced Short Palindromic Repeats, England, exagamglogene autotemcel, gene therapy, Nance-Horan syndrome, NICE, Nitroglycerin/Sodium Citrate/Ethanol Solution

Casgevy, exagamglogene autotemcel, CRISPR gene therapy, sickle cell disease, NHS England, Vertex Pharmaceuticals, NICE approval

Editas Medicine Undergoes Major Restructuring, Laying Off 65% of Staff Including CMO Amid Failed Search for RENI-CEL Partner

12 months ago talkbio0Tagged Anemia, Sickle Cell, Chronic multifocal osteomyelitis, Editas Medicine, Gene Modification, RENI-CEL, restructuring, Thalassemia

Editas Medicine, layoffs, gene editing, biotech, RENI-CEL, sickle cell disease, thalassaemia, CMO, workforce reduction, restructuring.

Advancements in Hematology: Shaping the Future of Blood Disorder Diagnosis and Treatment

1 year ago talkbio0Tagged Anemia, Sickle Cell, Clinical Practice Guideline, gene therapy, Hematological Disease, Hematology (discipline), Improvement, Quality

Hematology, Blood Disorders, Sickle Cell Disease, Gene Therapy, Clinical Guidelines, Quality Improvement

Vertex’s CASGEVY Marks Historic Milestone with First Commercial Patient Infusions, Paving the Way for 2025 Launches

1 year ago talkbio0Tagged Anemia, Sickle Cell, Approved, Casgevy, commercial, CRISPR/Cas9, gene therapy, Infusion procedures, launches, United States Food and Drug Administration

CASGEVY, Vertex Pharmaceuticals, gene therapy, sickle cell disease, beta thalassemia, CRISPR/Cas9, FDA approval, commercial patient infusions, 2025 launches.