1. uniQure's stock surged due to positive data on its Huntington's disease candidate AMT-130; 2. Hemgenix approval and new clinical programs strengthen the pipeline; 3. Cost-cutting and strategic financing extend operating runway to 2Q28.
Recent #Gene Therapy news in the semiconductor industry
➀ Researchers at the Icahn School of Medicine at Mount Sinai have developed a lipid nanoparticle system capable of delivering mRNA to the brain;
➁ This system bypasses the blood-brain barrier and could potentially treat conditions like drug addiction and Alzheimer's disease;
➂ The study shows that the lipid nanoparticles can safely and efficiently deliver mRNA into the brain, opening opportunities for mRNA-based therapies for neurological and psychiatric disorders.
➀ MinJun Kim and his research team at SMU have received a grant to develop a nanosensor for improving gene therapy accuracy; ➁ The technology aims to quantify and classify DNA content in liposomes with unprecedented accuracy; ➂ The project is multidisciplinary, involving biochemistry, gene transport, and nanophotonics, and is expected to attract students with diverse STEM interests.
➀ Researchers at the University of Pennsylvania and Moderna have developed a lipid nanoparticle-encapsulated mRNA therapy that significantly improves survival and reduces leucine levels in a mouse model of maple syrup urine disease (MSUD). ➁ The treatment strategy considers all genetic mutations causing MSUD and shows potential as a long-term universal treatment approach. ➂ Additionally, the Wilson lab has discovered novel AAV variants with improved biodistribution properties, potentially enhancing gene therapy targeting tissues other than the liver.