Vaccinex Inc. a clinical-stage biotechnology company recently announced that their drug candidate, VX15 developed for the treatment of Huntington’s disease (HD) received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA).
A new study from researchers in Spain may increase understanding of a less recognized aspect of Huntington’s disease (HD): cognitive problems. The article examines the possible causes of cognitive difficulties in HD and how they might be addressed through new therapies. The report, “Cognitive Dysfunction in Huntington’s Disease: Mechanisms and…
In a recent study, the modification of a specific component of the huntingtin protein reduced neurodegenerative symptoms in an animal model’s brain and offered hope for future therapies that could effectively treat Huntington’s disease in humans. The study, “Serine 421 regulates mutant huntingtin toxicity and clearance in mice,” was published in the…
Researchers at Icahn School of Medicine at Mount Sinai in New York have discovered that a protein complex crucial for normal brain development might also be necessary in the adult brain — silencing genes that drive neurodegeneration in Huntington’s disease. The study, “Polycomb repressive complex 2 (PRC2) silences…
Scientists at the Martinos Center for Biomedical Imaging at Massachusetts General Hospital were able — for the first time — to track epigenetic factors, or non-genetic influences on gene activity, linked to Huntington’s disease in the brains of living humans. The study, “Insights into neuroepigenetics through human histone…
About 300 international scientists and industry leaders recently gathered in Cambridge, Massachusetts, for the Hereditary Disease Foundation‘s 10th biennial “Milton Wexler Celebration of Life” Symposium, to promote collaborations that can take scientific findings from the laboratory into the lives of patients as treatments. One of the highlights of the meeting,…
In recent years, Huntington’s disease has joined the group of neurodegenerative diseases characterized by aggregation of the tau protein in fibrillary tangles inside neurons. An invention by Prana Biotechnology might give new insights into the disease processes and allow for new drug candidates to be screened for activity against the…
The accumulation of toxic protein aggregates within nerve cells are a hallmark of neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s, or Huntington’s disease. Now, researchers at the University of Glasgow in Scotland have identified a protein called UBQLN2 that protects against such diseases by eliminating those protein aggregates. The study, “…
Stopping the protein TDP-43 from accumulating in mitochondria may help prevent neurodegeneration in a variety of diseases, according to researchers at the Case Western Reserve University. In a recent study, they demonstrated that once inside mitochondria, TDP-43 shuts down its ability to produce energy, leading to nerve cell death. The study, “The…
Vaccinex‘s VX15, a novel clinical stage monoclonal antibody for Huntington’s disease (HD) treatment, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). VX15 blocks the activity of semaphorin 4D (SEMA4D), a molecule that is thought to promote chronic inflammatory responses in the brain. “We are very pleased…
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