source
- Father invests $70 million in gene therapy quest for daughter with ultra‑rare NGLY1 deficiency, faces FDA scrutinyMatt Wilsey has spent a decade and roughly $70 million trying to develop a gene therapy for his daughter Grace, who suffers from NGLY1 deficiency, an ultra‑rare genetic disorder. NGLY1 deficiency…
- Northwest Biotherapeutics' brain cancer therapy faces 931-day delay in UK approvalNorthwest Biotherapeutics, a public biotech developing a brain cancer treatment, filed a marketing application with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in late Decembe…