All articles tagged with #genetherapy
UniQure's plans to submit its Huntington's disease gene therapy, AMT-130, for FDA approval are now uncertain after the agency indicated it no longer considers existing data sufficient, marking a significant shift from previous communications and causing a 60% drop in the company's premarket shares.
A groundbreaking gene therapy called AMT-130 has shown promising results in slowing the progression of Huntington's disease in a clinical trial, with treated patients experiencing 75% less disease progression and lower neuronal damage markers, potentially offering a new hope for this currently incurable condition.
A new gene therapy for Huntington's disease has shown to slow disease progression by 75%, offering hope for treatment and potentially transforming patient outcomes, though regulatory and cost hurdles remain before it can be widely available on the NHS.
Preliminary results from a small clinical trial show that a gene therapy injected into the brain can slow the progression of Huntington’s disease by 75%, marking a significant breakthrough in treatment for this neurodegenerative condition.
A groundbreaking gene therapy trial in the UK has successfully slowed the progression of Huntington’s disease by 75% over three years, offering new hope for treatment of this incurable genetic disorder.
British researchers have uncovered how cordycepin, a compound from the caterpillar fungus Cordyceps militaris used in traditional Chinese medicine, can slow cancer cell growth by interfering with cell growth signals. This discovery, published by the University of Nottingham, highlights cordycepin's potential as a basis for new cancer treatments that may be less harmful to healthy tissues. The study found that cordycepin is converted into cordycepin triphosphate, which mimics ATP and disrupts cancer cell growth pathways.
The FDA has approved two groundbreaking cell-based gene therapies, Casgevy and Lyfgenia, for treating sickle cell disease (SCD), with Casgevy being the first FDA-cleared clinical use of CRISPR-Cas9 gene editing for any condition. SCD, which disproportionately affects those with African ancestry, has long been underfunded in research and treatment. While these therapies offer hope, concerns about their high costs and accessibility remain, especially for those not enrolled in Medicaid. The ethical use of CRISPR in these therapies differs from more controversial applications, and the focus should now be on making these treatments more affordable and accessible to patients worldwide.
Novartis has expanded its gene therapy collaboration with Voyager Therapeutics, investing $100 million upfront to acquire capsids targeting Huntington’s disease and spinal muscular atrophy. The announcement, which comes ahead of the JP Morgan Healthcare Conference, has already positively impacted Voyager's stock, with shares increasing by 32%. Additionally, Novartis will purchase $20 million in Voyager shares, further cementing the partnership between the two companies in the field of neurologic gene therapies.
Voyager Therapeutics' shares surged 35% after announcing a licensing deal with Novartis worth $1.2 billion, including $100 million upfront. The deal grants Novartis access to Voyager's RNA-based screening platform to develop a gene therapy for Huntington's disease, with Voyager eligible for milestone payments and royalties on sales.
Voyager Therapeutics has announced a strategic collaboration and capsid license agreement with Novartis to develop gene therapies for Huntington’s disease and spinal muscular atrophy. Voyager will receive $100 million upfront and is eligible for up to $1.2 billion in milestone payments, plus royalties. The partnership leverages Voyager's TRACER capsid discovery platform, which is designed to enhance delivery of gene therapies to the central nervous system. Novartis will handle development and commercialization, while Voyager focuses on preclinical advancement.