Source Transparency Market Research Gene Therapy Market – Snapshot The global gene therapy market is expanding at an exponential pace due to promising therapeutic outcomes of gene therapy, high prevalence and rise in incidence of cancer, and large number of clinical research pipeline products. In terms of revenue, the market was valued at US$ 17.0
Source Global Data Gene therapy involves the delivery of complex treatments to patients. In recent years, there have been several promising clinical trial results within this field targeting an array of inherited neurodegenerative disorders, genetic diseases, and various cancers. Approvals based on these clinical trials have consequently encouraged interest in gene therapy from both industry
Researchers at ETH Zurich have refined the famous CRISPR-Cas method. Now, for the very first time, it is possible to modify dozens, if not hundreds, of genes in a cell simultaneously. Everyone’s talking about CRISPR-Cas. This biotechnological method offers a relatively quick and easy way to manipulate single genes in cells, meaning they can be
Safety, innovation and affordability need not be mutually exclusive goals for cutting-edge treatments. Source NYT Spinal muscular atrophy is a condition often fatal by age 2. Babies with the disorder have a rare genetic mutation that prevents the nervous system from controlling certain muscles. As they grow to be toddlers, these children struggle to move their limbs,
Source https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.026#utm_source=ETOC&utm_medium=email&utm_campaign=hum Corneal diseases are among the most prevalent causes of blindness worldwide. The transparency and clarity of the cornea are guaranteed by a delicate physiological, anatomic, and functional balance. For this reason, all the disorders, including those of genetic origin, that compromise this state of harmony can lead to opacity and eventually vision loss.
Source STAT A common saying at the Food and Drug Administration is: “In God we trust, all others must bring data.” The independent evaluation of science is an essential element of the FDA’s dual role of protecting the public health and promoting innovation to bring new therapies into practice. In fact, with certain exceptions, the
The results of an early trial in 31 brain cancer patients finds immune activity boosted in the tumor, and possibly longer survival. An inducible, tumor-localized gene therapy has been tested for the first time in glioblastoma patients. The two-part approach, which involves receiving an injection of an immune-activator gene into the brain tumor site and
The gene editor targets cancer, blood disorders and blindness Since its debut in 2012, CRISPR gene editing has held the promise of curing most of the over 6,000 known genetic diseases. Now it’s being put to the test. In the first spate of clinical trials, scientists are using CRISPR/Cas9 to combat cancer and blood disorders
Source STAT Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for
Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness. People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight. The experimental treatment aims to
