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.
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