MULTIPLE sclerosis campaigners have hailed a “huge step forward” for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time. Haematopoietic stem cell transplantation (HSCT) has been described as a “game-changer” for MS after an international clinical trial showed that it could reboot patients’ immune systems and halt the progress
Researchers are making great strides toward de-veloping gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement. A review of the approaches currently under preclinical or clinical investigation is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the full-text article free on the Human
The first ever analysis of all the tweets discussing Crispr gene-editing has revealed how the public feels about the technology. The findings suggest that over a six years public approval of gene-editing has cooled and that most people chatting about the technology are worried. Twitter offers researchers a vast amount of semi-structured data and provides direct access
GE Healthcare Life Sciences is launching KUBio™ box, a fully-integrated, flexible, and adaptable biomanufacturing environment to accelerate the production of viral vector-based gene therapies. The latest addition to the KUBio modular bioprocessing portfolio, KUBio box is expected to speed gene therapies to market and contribute to increased capacity in the viral vector area. Gene therapies
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.
