FDA Approves First Gene Therapy for Bladder Cancer

PLUS: New Trial for Hereditary Angioedema

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Here are the top stories in the world of medical biology:

  • First Gene Therapy for Bladder Cancer Approved READER REQUEST

  • Phase I/II Clinical Trial Initiated for Hereditary Angioedema

  • CRISPR Shows Promise for Retinitis Pigmentosa READER REQUEST

  • TALENs as Targeted Therapy for HPV-Induced Cancer READER REQUEST

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📣 First Gene Therapy for Bladder Cancer Approved

What is non-muscle invasive bladder cancer (NMIBC)? NMIBC is a form of bladder cancer that is present in the surface layers of the bladder - it has not invaded deeper into the bladder or other parts of the body. Currently, the first treatment option for NMIBC is to add Bacillus Calmette-Guerin (BCG), a bacteria, through a catheter. Yes, you read that right - BCG is a bacteria that does not cause serious disease and it is added into the bladder to help “turn on” the immune system in the bladder and kill the bladder cancer. 🤯 However, BCG therapy will fail in 40-50% of patients leaving them with only one other option: removal of the whole bladder.

1/ Adstiladrin® is the first gene therapy approved for BCG-unresponsive NMIBC.

2/ The FDA approval of Adstiladrin® was based on the results of a Phase 3 clinical trial where 51% of patients had a complete response to the gene therapy eliminating their bladder cancer and remaining free of cancer recurrence for at least 12 months.

How Adstiladrin® gene therapy works 👇🏼

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