The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, today released a Therapeutic Developers’ Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications. The statement, developed by ARM’s Gene Editing Task Force and signed by
The generation of autologous T cells expressing a chimeric antigen receptor (CAR) have revolutionized the field of adoptive cellular therapy. CAR-T cells directed against CD19 have resulted in remarkable clinical responses in patients affected by B-lymphoid malignancies. However, the production of allogeneic CAR-T cells products remains expensive and clinically challenging. Moreover, the toxicity profile of
Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic effects in mice. These research findings are published online in the journal Science Translational Medicine. Prior to joining Ohio State as a professor
Researchers have created the world’s most detailed atlas of the genetic code of the human retina, and it could help treat and prevent blindness The transcriptome of human neural retina at a single‐cell level defines the gene expression profile in major cell types in the neural retina and can be used as a benchmark to
The UK’s departure from the European Union looks likely to dent many sectors at least in the short to medium term, but the biopharmaceutical industry is one that might just continue to shine, however damaging the exit from the trade bloc proves to be. The quality of innovation coming out of labs, with 25 of
Researchers at Carnegie Mellon University (CMU) and Nanyang Technological University (NTU) in Singapore have developed an organ-on-an-electronic-chip biosensor technology that can measure the electrophysiology of heart cell structures in three dimensions. The biosensor array essentially self-rolls around elongated spheroids of stem cell-derived cardiac cells—similar to how a “slap bracelet” wraps around a wrist—giving researchers the
Salk scientists develop a new gene-editing tool that could help treat many disorders caused by gene mutations The ability to edit genes in living organisms offers the opportunity to treat a plethora of inherited diseases. However, many types of gene-editing tools are unable to target critical areas of DNA, and creating such a technology has
The FDA approved upadacitinib (Rinvoq, AbbVie), a 15-mg, once-daily oral Janus kinase inhibitor, for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to methotrexate. The approval is supported by data from the SELECT program, one of the largest registrational phase 3 clinical programs
For transplant patients, the body’s own defences can be fatal. Breakthrough research offers new hope Source Robert Lechler on Financial Times “Although I was well aware of the time ticking away throughout the procedure — everyone was — I could only continue to work carefully and systematically and, at all costs, efficiently,” surgeon Joseph Murray
California Institute for Regenerative Medicine awarded a $9.28 million grant will support a clinical trial at City of Hope to evaluate chimeric antigen receptor T-cell therapy for patients with HER2-positive breast cancer that spread to the brain. About 20% of breast cancers are HER2-positive, and nearly half of women with this type of breast cancer develop brain
