Harnessing the power of cell and gene therapies will require a next-generation level of collaboration not seen before the COVID-19 pandemic. In brief Before cell and gene therapies can meet increasing demand at the necessary speed and scale, the industry must address key challenges. A fractured supply chain, complicated manufacturing and unprecedented pricing are among
In the last issue of Blood, Boulos et al analyze the cost-effectiveness of factor IX gene therapy in patients with severe hemophilia B, in a microsimulation Markov model. Hemophilia is an X-linked disease that affects 400000 people worldwide, of whom 15% have hemophilia B caused by deficiency of factor IX (FIX), which affected the offspring of Queen Victoria,
Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her
In a breakthrough for the treatment of aggressive solid cancers, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach. Using the power of large data sets and advanced computational approaches,
Mesenchymal stem cells (MSCs) are multi-functional stem cells that are present in multiple human tissues and can be found in the spinal cord, umbilical cord blood, umbilical cord tissue, placenta tissue, adipose tissue, etc.. With low immunogenicity, multi-directional diferentiation ability, in particular homing ability, MSCs have signifcant research potential in cardiovascular diseases, nervous diseases, and
Treatment with mesenchymal stem cells (MSCs) has failed to significantly reduce inflammation in the brain of adults with multiple sclerosis (MS) in a phase 2 clinical trial. The into-the-vein treatment also failed to improve other clinical aspects of the condition. The results were shared in a study titled “Safety, tolerability, and activity of mesenchymal stem cells versus placebo
The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke
After numerous setbacks at the turn of the century, gene therapy is treating diseases ranging from neuromuscular disorders to cancer to blindness. The success is often qualified, however. Some of these therapies have proved effective at alleviating disease but come with a high price tag and other accessibility issues: Even when people know that a
The new therapy was generally well-tolerated and prevented key signs of the condition in animal models. Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman
This review aims to outline the particular approaches in the different published clinical trials for cell-based therapies for retinal diseases. The retinal pigment epithelium (RPE) is implicated in the pathophysiology of many retinal degenerative diseases. This cell layer is also an ideal target for cell-based therapies. Several early phase clinical trials evaluatingcell therapy approaches for
