The U.S. Food and Drug Administration has recently approved Adzynma, the first recombinant (genetically engineered) protein product indicated for prophylactic or on demand enzyme replacement therapy in adult and pediatric…
Today, from Revio, we would like to share an overview on the FDA’s scientific guideline to “Human Gene Therapy for Rare Diseases”, that provides recommendations on the manufacturing, preclinical, and…
Today from Revio we’re happy to announce a new pilot program developed by the U.S. Food and Drug Administration (FDA) to help further accelerate the development of novel drug and…
Revio is pleased to announce the upcoming 76th Cellular, Tissue, and Gene Therapies Advisory Committee scheduled for October 31, 2023, from 9:00am - 5:00pm ET. Before diving into the agenda…
Today from Revio we are sharing with you an overview on the FDA’s Guidance for Industry "Human Gene Therapy for Retinal Disorders". This document focuses on issues specific to gene…
Today, from Revio, we will provide an overview on the EMA’s Guideline on Xenogeneic Cell-Based Medicinal Products. This guideline offers general principles for the development of these types of products.…
FDA Approves First Cellular Therapy to Treat Patients with Type 1 Diabetes Recently, the FDA granted approval to the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor…
Recently, the FDA has given its approval to Roctavian from BioMarin Pharmaceutical Inc. It is an adeno-associated virus vector-based gene therapy intended to treat adults who have severe Hemophilia A…
The FDA has officially granted approval on June 22nd 2023 to Elevidys, marking it as the first gene therapy for addressing Duchenne muscular dystrophy (DMD) in paediatric patients aged 4…
Today, from Revio, we would like to share an overview on the EMA’s scientific guideline “Questions and answers: Comparability considerations for Advanced Therapy Medicinal Products (ATMP)”. Comparability remains an important…
Revio is a Regulatory Affairs Consultancy – Created to support biopharmaceutical companies and public institutions, working with Advanced Therapy Medicinal Products (ATMPs)