Cell therapy updates: first FDA-cleared IND for an AAV-based cancer therapy
Discover the latest breakthroughs in cancer regenerative therapies in this edition of RegMedNet’s Cell therapy weekly.
The past week brought significant regulatory developments in gene therapy. Siren Biotechnology (CA, USA) achieved a milestone by securing the first US FDA-cleared IND for an adeno-associated virus (AAV)-based therapy in an oncology indication (recurrent high-grade glioma), marking the company’s transition to clinical-stage operations. Meanwhile, the FDA placed clinical holds on REGENXBIO’s (MD, USA) two Mucopolysaccharidosis treatments (RGX-111 and RGX-121) following a CNS tumor development in a Phase I/II trial patient. Both therapies use AAV9 vectors to deliver functional genes to the central nervous system for treating rare genetic disorders.