Category REGENERATIVE MEDICINE

AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1

New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who

Read More


Boston University researchers create new protocol to improve gene therapy tool production

Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus

Read More


FDA Grants Breakthrough Therapy Designation for Genentech’s Gazyva (Obinutuzumab) in Lupus Nephritis

The designation is based on the results of the Phase II NOBILITY study that showed Gazyva, in combination with standard of care, helped more people achieve a complete renal response than standard of care alone Genentech today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Gazyva® (obinutuzumab) for

Read More


SanBio Granted Regenerative Medicine Advanced Therapy Designation from the U.S. FDA for SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury

The SanBio Group, a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results

Read More


Zylka Lab awarded $6.1 million from NIH to develop CRISPR/Cas9 gene therapy for Angelman syndrome, study UBE3A autism gene with Philpot Lab

Mark Zylka, PhD, in The UNC Neuroscience Center was awarded NIH funding for two 5-year projects, one jointly with Ben Philpot, PhD, to develop new knowledge with the potential to advance treatments for neurodevelopmental disorders. The National Institutes of Health have awarded two separate grants totaling $6.1 million to Mark Zylka, PhD, director of the

Read More