GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patient’s own cells in the body. The cells produce then a therapeutic protein according to the template that the introduced genetic material (DNA) provides
GeneQuine is focused on the development of gene therapy agents for the treatment of osteoarthritis. Osteoarthritis is a degenerative joint disorder characterized by cartilage loss and inflammation. Patients affected by osteoarthritis experience joint pain as well as swelling and stiffness of the joints leading to … Read more
Genome Editing with CRISPR-Cas9
Here’s a short list of some common diseases that might be curable or preventable with gene editing:
Duchenne muscular dystrophy
There are hundreds of other more rare genetic disorders.… Read more
Gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the disease—meaning it’s caused by the deletion of or mutations in a single gene. AVXS-101 is our clinical-stage, proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1—designed to prevent further muscle degeneration caused by SMA through:
Delivery of a fully functional human SMN gene into target motor neuron cells
Production of sufficient levels of SMN protein required to improve motor neuron function
Rapid onset of effect in addition to … Read more
– Advantagene Inc.
– Bluebird Bio
– Human Stem Cells Institute
– Oxford BioMedica Plc
– Shanghai Sunway Biotech Co. Ltd.
– Sibiono GeneTech Co. Ltd.
– Spark Therapeutics, LLC
– UniQure N.V.
– Vical Inc.
– ViroMed Co. Ltd. dba VM BioPharma … Read more
We need to keep eye on http://elixirgen.com/ This is new company so not alto of detail but they are trying.
I will keep this in my alert list.
http://hereandnow.wbur.org/2013/07/17/therapy-down-syndrome… Read more