Category REGENERATIVE MEDICINE

Gene Therapy Shows Early Promise as Angelman Syndrome Treatment

The new therapy was generally well-tolerated and prevented key signs of the condition in animal models. Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman

Read More


Cell-based therapies for retinal diseases: review of clinical trials and direct to consumer “cell therapy” clinics

This review aims to outline the particular approaches in the different published clinical trials for cell-based therapies for retinal diseases. The retinal pigment epithelium (RPE) is implicated in the pathophysiology of many retinal degenerative diseases. This cell layer is also an ideal target for cell-based therapies. Several early phase clinical trials evaluatingcell therapy approaches for

Read More


EMA PRAC Concludes There is No Link Between Zynteglo and AML

EMA’s safety committee, PRAC, has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). The European Medicines Agency’s (EMA’s) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known

Read More


Gene therapy appears more cost effective than current treatments for severe hemophilia B

St. Jude Children’s Research Hospital provides the first gene therapy manufacturing and distribution cost report for hemophilia B. A St. Jude Children’s Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene therapy now in development. The study appeared

Read More


Deep Pharma Intelligence Releases Analytical Report “Cell Therapies In Healthcare Landscape Overview 2021”

Deep Pharma Intelligence released a special 115-page analytical report Cell Therapies In Healthcare Landscape Overview 2021 (Stem Cells and CAR-Ts), which is the first comprehensive and systematic overview of cell therapies landscape by the company. This report is specifically focused on Stem Cell therapies and Chimeric Antigen Receptor T-cell (CAR-T) therapies, and it is the first

Read More