Scientists of John Hopkins University School of Medicine summarize the current methods of human pluripotent stem cells (HPSCs) myogenic commitment/differentiation, and describe the current status of hPSC-derived myogenic cell transplantation. Muscular dystrophies are a group of genetic muscle disorders that cause progressive muscle weakness and degeneration. Within this group, Duchenne muscular dystrophy (DMD) is the
CARsgen Therapeutics Co. Ltd., a clinical-stage biopharmaceutical company today announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational CT053 CAR-T cell therapy. CT053 is a fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor (CAR) T Cell therapy for the treatment of
A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge. Source EPR According to a new report, ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies
The International Society for Cell and Gene Therapy has created a group to tackle the rising number of commercial cell bank services that are misleading patients. The International Society for Cell and Gene Therapy (ISCT) has announced the formation of a global consortium to combat the growing number of unproven commercial cell banking services. The group will
“The Italian government has decided to invest 60 million euros on CAR-T therapies, 10 million for each of the 6 pharmaceutical workshops”. This was stated by Health Minister Roberto Speranza, explaining to the joint committees of the Senate and the House the program lines of his department. CAR-T cell therapy consists of genetic manipulation of
The announcement by the Health Minister Speranza: “60 million to 6 workshops to produce Car-T against cancer” The Car-t therapies are “a strategic segment on which the government has decided to invest 60 million euros, foreseeing 10 million for each pharmaceutical workshop”, or high-tech laboratories in which the patient’s immune cells against tumors are modified.
Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects. This new technique is called the “prime editing” and is being developed
Source NIH Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates
Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options Source FDA The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older
A more efficient approach to gene therapy that could lower costs and improve patient outcomes has recently been developed by a team from Scripps Research. This work, published on October 17 in the journal Blood, offers a potential alternative to the standard process of delivering gene therapy, which is expensive, time-consuming, and requires many steps to administer healthy
