Once thought to be a frontrunner in gene therapy, uniQure’s fame may no longer be coveted, as the biotech looks to reorganize by slashing its workforce, in an attempt to stay afloat. But how did things go awry for the company that was once in its prime in the DNA game? The Danish company, which […]
Alchemab Therapeutics has been awarded a grant of £1.7 million ($2 million) from Innovate UK’s Biomedical Catalyst (BMC) 2022 funding competition to accelerate development of a first-in-class disease modifying therapy for Huntington’s disease. Working in collaboration with Medicines Discovery Catapult (MDC), Alchemab is carrying out preclinical studies to progress its panel of antibodies towards first […]
A trial to look at gene therapy, BV-01, for Huntington’s Disease has been given the go ahead to take place in France and has ‘the potential to change the course of the devastating disease. Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, received clearance for the phase 1/2 […]
Prilenia Therapeutics has raised €55M in Series A funding to launch late-stage clinical trials of its small molecule candidate drug in Huntington’s disease and amyotrophic lateral sclerosis, incurable conditions that have very limited treatment options. Dutch life sciences VC Forbion led the round and was joined by new Chinese investor Morningside Venture Investments and the […]
Barcelona-based SOM Biotech is developing treatments for incurable diseases such as Huntington’s disease with drugs that are already on the market for other diseases. Mission: To repurpose commercially available drugs for the treatment of neurological diseases lacking effective treatments. Using existing drugs to treat these rare, orphan diseases lets the company get the treatments to […]
Update (23/01/2019): The FDA has given uniQure regulatory clearance to begin a phase I/II trial of its gene therapy for Huntington’s disease. uniQure is planning to launch the trial in the US in late 2019. Published on 23/01/2018: The EMA has given uniQure’s gene therapy candidate for Huntington’s disease Orphan Medicinal Product Designation, which the […]
Using a lab-on-a-chip device, Swiss scientists have found a way to diagnose the neurodegenerative condition Huntington’s disease from DNA in the blood in just five minutes, rather than the five hours currently required. When carrying out the diagnosis of Huntington’s patients, the patient’s DNA is typically extracted from white blood cells. Regions of the […]
A study run by Oxford BioDynamics suggests that we could predict Huntington’s more easily by analyzing epigenetic changes in the patient’s DNA. Epigenetics describes the way that the expression of our genes is regulated, often influenced by environmental exposure, without changing the genetic code. Ways of modifying gene expression include attaching chemical groups to DNA […]
uniQure has raised a substantial public offering on the NASDAQ. The funding could help the Dutch biotech advance two new gene therapies after the withdrawal of its first treatment. Amsterdam-based uniQure has raised an ambitious $128M (€107M) in a public offering at the beginning of next week. The Dutch biotech develops gene therapies for hemophilia and Huntington’s disease, for […]
Today, we’re taking a look at Anima Biotech, an Israeli company that is developing a new class of drugs that target protein translation to combat a range of diseases, including lung fibrosis, cancer, and Huntington’s disease. Mission: Since 2010, Anima Biotech has been targeting disease-causing proteins with a new class of drugs that control protein translation. […]
Polish researchers tested a new variant of CRISPR/Cas9 that increases the gene-editing system’s precision in targeting specific DNA sequences, such as the gene responsible for Huntington’s disease. CRISPR/Cas9 offers a means of editing genes by essentially ‘cutting and pasting’ sequences of DNA. A group of researchers at the Institute of Bioorganic Chemistry in Poland has developed […]