Gene Therapy Just Delivered Dopamine Directly Into the Brain. Now Comes the Hard Part.
BBM-P002's Phase 1 data in 10 Parkinson's patients shows dual-target AAV gene therapy can restore dopamine synthesis. The FDA...
Cell and Gene Therapy
CNS Gene Therapy Trials Are About to Break Every Monitoring Assumption Your CRO Has
BBM-P002's multicenter Phase 1 Parkinson's data signals a wave of CNS gene therapy trials that will overwhelm standard eClinical...
The Cancer Nobody Saw Coming: What One Gene Therapy Tumor Tells Us About a Broken Safety Surveillance System
A rare gene therapy-linked cancer case exposes fatal gaps in long-term safety monitoring—and why FDA's 15-year follow-up mandate isn't...
One Cancer Case Is All It Takes: Why Gene Therapy’s Post-Market Safety Infrastructure Is Still Broken
A rare gene therapy-linked cancer case exposes the gap between FDA's 15-year CAR-T surveillance mandate and real-world monitoring systems...
Intellia’s CRISPR Submission Is a Regulatory Stress Test the FDA Has Never Faced Before
Intellia's nex-z BLA for ATTR amyloidosis is the FDA's first in vivo CRISPR reckoning—and the agency's gene therapy playbook...