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
Source Human Gene Therapy Clinical Development Background: Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for the treatment and prevention of diseases that until recently were only managed chronically. The objective of this study was to examine the characteristics
Sangamo Therapeutics and Pfizer have reported additional findings from the Phase I/II Alta clinical trial of investigational gene therapy SB-525 to treat patients with severe haemophilia A. SB-525 is made up of a recombinant adeno-associated virus serotype 6 vector (AAV6) that encodes the complementary deoxyribonucleic acid for B domain deleted human Factor VIII (FVIII). During
We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such
