Stem Cell Therapy & FDA Regulations: The Battle Over Access and Innovation
A revolution is underway in the world of medicine—one driven by the extraordinary potential of stem cells. In Episode 7: Stem Cell Revolution – Homologous Schmologous, experts dive deep into the science, regulatory challenges, and ethical debates surrounding stem cell therapy. The episode breaks down complex concepts such as homologous vs. non-homologous use, minimal manipulation, […]
FDA Grants Breakthrough Therapy Designation to Seres’ SER-155
Seres Therapeutics announced that the FDA granted Breakthrough Therapy designation to SER-155, their lead investigational drug, for reducing bloodstream infections in adults undergoing stem cell transplants. This designation is based on positive Phase 1b clinical trial data showing a significant reduction in infections compared to a placebo. A meeting with the FDA to discuss the […]
The FDA’s New Sheriff in Town? RFK Jr.’s Bold Vision for Stem Cell Therapy
Robert F. Kennedy Jr. is making waves in the medical community with his stance on stem cell treatments, and it’s stirring up quite a storm in healthcare circles. As one of President-elect Trump’s key health advisers, Kennedy has pledged to shake up the FDA’s approach to stem cell therapy regulation[1]. The Current Landscape Right now, […]
Somite Therapeutics Achieves Key Milestone with FDA Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 in Duchenne Muscular Dystrophy
Somite Therapeutics has announced a major breakthrough in the fight against Duchenne Muscular Dystrophy (DMD), securing both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA for their lead therapeutic candidate, SMT-M01. These designations are critical in the development of therapies targeting rare diseases, offering benefits such as tax credits for […]
Wave Life Sciences’ Groundbreaking FDA Designation Signals Hope for Duchenne Muscular Dystrophy Patients
Wave Life Sciences has achieved a significant milestone with the FDA’s Rare Pediatric Disease Designation for their innovative treatment, WVE-N531, aimed at boys suffering from Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping. This designation underscores the critical need for advancements in stem cell research and gene therapy for rare diseases like […]
Revolutionizing Crohn’s Disease Treatment: TRX103 Gains FDA Clearance
Tr1X, a trailblazing biopharmaceutical company, has announced a significant milestone in the fight against treatment-refractory Crohn’s Disease. The FDA has granted clearance for the Investigational New Drug (IND) application of their innovative therapy, TRX103, an allogeneic engineered Tr1 Treg cell therapy. This breakthrough offers new hope for patients who have not responded to conventional treatments. […]
FDA Accepts BLA for Ryoncil: First Allogeneic “Off-the-Shelf” Cellular Medicine and First Cell Therapy for Children in the United States
In a significant milestone for pediatric medicine, the FDA has accepted Mesoblast’s Biologics License Application (BLA) for Ryoncil® (remestemcel-L), aimed at treating children with Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD). This acceptance marks a crucial step towards the approval of the first allogeneic “off-the-shelf” cellular medicine in the United States, and the first cell therapy for […]
Breakthrough in Muscular Dystrophy Treatment: FDA Approves Stem Cell Therapy from Myogenica
The fight against muscular dystrophy has taken a promising turn with the recent FDA approval of an Investigational New Drug (IND) application for MyoPAXon, a revolutionary stem cell-derived muscle stem cell product developed by Myogenica, a University of Minnesota startup. This landmark approval paves the way for a clinical trial aimed at treating patients with […]
BrainStorm Cell Therapeutics Aligns with FDA on Phase 3b NurOwn Clinical Trial CMC Aspects
BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, has reached an important milestone by aligning with the U.S. Food and Drug Administration (FDA) on the Chemistry, Manufacturing, and Controls (CMC) aspects of its Phase 3b clinical trial for NurOwn®, an investigational therapy for amyotrophic lateral sclerosis (ALS). This […]
iRegene Receives IND Approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease
iRegene Therapeutics has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting Investigational New Drug (IND) approval for their innovative cell therapy, NouvNeu001, aimed at treating Parkinson’s disease. This approval marks a pivotal step forward in the fight against this debilitating neurodegenerative disorder. A New Hope for Parkinson’s Patients Parkinson’s disease, characterized by the loss of dopaminergic neurons, […]