Prevail's gene therapy scores orphan, rare pediatric disease privileges; Germany's ITM secures €40M loan
→ Prevail Therapeutics’ gene therapy for Gaucher disease now has both orphan drug and rare pediatric disease designations from the FDA. The latter was specifically for the treatment of neuronopathic Gaucher disease, the most severe form of the condition. PR001 is designed to transfer the GBA1 gene to patients.
→ Privately-held biotech ITM (Isotopen Technologien München) has scored a €40 million loan from the European Investment Bank to fund the development of diagnostics and therapies to address a range of cancers such as neuroendocrine tumors and bone metastases.
→ With its latest fast track designation the FDA has offered to speed up Nanobiotix’s head and neck cancer program for NBTXR3, a hafnium oxide nanoparticle designed to enhance radiotherapy. The designation — which covers patients with locally advanced disease who are not eligible for platinum-based chemotherapy — means Nanobiotix may now enjoy more frequent communication with regulators and possibly other accelerated pathways down the road. The treatment is also being tested in soft tissue sarcoma and liver cancer, among others.