Gene Therapy Could End Lifelong Transfusions For Rare Blood Disorder

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...
BioPharma Dive

Setting cell and gene therapies up for success with a smarter CMC strategy

Being proactive — especially when it comes to testing, process development and preparing for commercialization — can greatly decrease the risk that your CGT will receive a CRL.

New gene therapy now administered as Sickle Cell Anemia treatment in St. Louis Children’s Hospital

According to the CDC, nearly 100,000 Americans have Sickle Cell Anemia, or 1 in every 265 African American births.
The Miami Times

FDA-approved sickle cell therapy gives teen a new life, free from pain

Nineteen candles lit up the cake for Caden Major this year, but the true celebration was something he once thought impossible ...

Novel gene editing technology enables selective destruction of cancer cell DNA using fewer targets

Researchers from the Department of Biomedical Engineering at UNIST and the Center for Genomic Integrity at the Institute for Basic Science (IBS) have announced an advance in cancer gene therapy. Their ...

Parkinson's disease at forefront of Bayer bet on cell and gene therapies

Bayer's pioneering cell and gene therapies for Parkinson's disease are at the forefront of an exciting but risky test of CEO ...

VintaBio™ Files Patents for Scalable Manufacturing Platform Built to Accelerate Gene Therapy Commercialization

VintaBio™, a technology-forward company, announced today that it has filed provisional patents with the US Patent Office ...

Science history: A tragic gene therapy death that stalled the field for a decade — Sept. 17, 1999

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...