Novartis/gene therapy: corporate DNA
The control premium for AveXis is hefty, but should be given the benefit of the doubt
PMID: 29769001 [PubMed - in process]
PMID: 29769000 [PubMed - in process]
(Michigan State University) A Michigan State University veterinary ophthalmologist has modified a gene therapy that reverses blindness in dogs that have a certain form of a disease known as progressive retinal atrophy, or PRA, and is now looking to advance the treatment for human use. Simon Petersen-Jones in the College of Veterinary Medicine has received a five-year, $8.2 million grant from the National Institutes of Health to further the therapy for people who have a type of retinitis pigmentosa.
12 June 2018, Boston, United States
Delivery of large DNA constructs is necessary for combinatorial, anti-aging gene therapy. However, this is hindered by the lack of a non-inflammatory vector with sufficient packaging space and the ability to effectively spread through tissue. It is possible that a mitochondrion, altered to be able to secrete therapeutic proteins, could serve as an ideal gene delivery vector in this regard (for post-mitotic cells at least). Initial transfection of mitochondria with a therapeutic gene vector could be undertaken by MITO-Porter, i.e., a dual-layered fusogenic liposomal-based delivery system.
Publication date: Available online 18 May 2018 Source:Cytotherapy Author(s): Judith A. Arcidiacono, Steven R. Bauer, David S. Kaplan, Clare M. Allocca, Sumona Sarkar, Sheng Lin-Gibson The development of standards for the field of regenerative medicine has been noted as a high priority by several road-mapping activities. Additionally, the U.S. Congress recognizes the importance of standards in the 21st Century Cure Act. Standards will help to accelerate and streamline cell and gene therapy product development, ensure the quality and consistency of processes and products, and facilitate their regulatory approval. Although t...
ConclusionWith the promising clinical trial results that have been observed for the latest AAV gene therapies and continued pre-clinical successes, the question is no longer whether a therapy can be developed for certain neurodevelopmental disorders, but rather, how quickly.
Authors: Li R, Wang QF, Li WW, Niu XC Abstract OBJECTIVE: To explore a new approach for treating renal insufficiency with gene therapy by implanting decorin (DCN)-expressing fibroblasts within the renal tissue of rats with renal failure to neutralize TGF-β1 activity. MATERIALS AND METHODS: The 5/6 kidney of the selected male SD rats were removed under aseptic conditions. The rats were grouped randomly after the establishment of the model. There were 10 rats in the sham-operated group (Group A), 10 in the operation control group (without treatment, Group B), 10 in the blank control group [treated with empty...
10 - 11 October, London, UK. SMi introduces their inaugural event on Cell &Gene Therapy which takes place between the 10th and 11th October 2018, with an interactive pre-conference workshop on the 9th October. Cell and gene therapeutics have revolutionised modern medicine and mark a new generation in biomedical and agricultural sciences. The latest developments in cell and gene therapies and specifically in gene editing, technologies present unlimited research opportunities ranging from novel therapeutic tools to a potential revolution in the field of drug discovery.
Publication date: Available online 26 April 2018 Source:Stem Cell Reports Author(s): Aslam Abbasi Akhtar, Genevieve Gowing, Naomi Kobritz, Steve E. Savinoff, Leslie Garcia, David Saxon, Noell Cho, Gibum Kim, Colton M. Tom, Hannah Park, George Lawless, Brandon C. Shelley, Virginia B. Mattis, Joshua J. Breunig, Clive N. Svendsen Trophic factor delivery to the brain using stem cell-derived neural progenitors is a powerful way to bypass the blood-brain barrier. Protection of diseased neurons using this technology is a promising therapy for neurodegenerative diseases. Glial cell line-derived neurotrophic factor (GDNF) has prov...