Helix Therapeutics LLC aims to modify disease-linked genes on certain stem cells by introducing oligonucleotides that bind to double-stranded DNA inside a cell. Ideally, the oligos will bind tightly into the groove between strands of DNA and form a triple-stranded helix, or triplex, that prevents the gene from being read. If the results of Helix's animal experiments turn out to be replicable in humans, Helix may have a way of treating, and possibly even curing, sickle-cell disease and other disorders caused by genetic mutations.
150 Munson Street
25 Science Park, 2nd Floor
Read the full article – start your free trial today!
Join thousands of industry professionals who rely on Scrip for daily insights
With $216m in initial funding, the newly launched company is aiming to develop a cell therapy-based universal immuno-oncology option for solid tumor indications.
Seven weeks before its action date, Capricor got an FDA complete response for cell therapy deramiocel in DMD-related cardiomyopathy. It has a Phase III study nearing readout, though.
With $216m in initial funding, the newly launched company is aiming to develop a cell therapy-based universal immuno-oncology option for solid tumor indications.
Regenxbio and Solid Biosciences believe they have a next-generation approach to AAV-based gene therapies – but must distance themselves from Sarepta’s safety and efficacy problems.
The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.