Today, researchers and clinicians are under great pressure to develop therapies capable of reversing or significantly impacting the progression of disease. In recent years, the arrival of the cell and gene therapy industries have introduced the possibility of providing transformative, durable and potentially curative outcomes for a diverse range of life-threatening conditions, as well as injuries, degenerative diseases, genetic disorders, and cancers. [Read more…]
An Expert’s Perspective on Accelerated Pathways for Cell Therapy Approvals
Over the past few years, the regulatory landscape for cell therapy development has grown increasingly complex. There are now accelerated pathways for advanced therapy medicinal products (ATMPs) in several countries worldwide, including the U.S., Japan, and South Korea. While the possibility for accelerated commercialization has resulted from these changes, substantial complexity has also been introduced, making it a more elaborate process to move cell therapy products from “bench to bedside.”
In the interview with Yaron Ramati, Director of Regulatory Affairs at Pluristem Therapeutics, we get an expert’s perspective on how the regulatory environment has changed and new opportunities that exist for bringing cell therapy products through the clinical trial process and into the global marketplace. [Read more…]
FDA Regulation of Cellular and Tissue-Based Products with Dr. Riam Shammaa
In this interview with Dr. Riam Shammaa, Medical Director at the Canadian Centres for Regenerative Therapy (CCRT), we discuss FDA regulation of human cells, tissues, and cellular and tissue-based products (HCT/P’s), as well as the difference between 351 and 361 products.
While the FDA has a critical role in market oversight, individual market participants are also responsible for understanding the regulatory framework affecting the sale, distribution, and utilization of cell therapy, gene therapy, and tissue engineering products. This interview is aimed to help you understand the FDA framework affecting HCT/Ps within the U.S, as well as the framework and recommendations implemented by Health Canada.
Breaking: FDA Announces Program to Help Manufacturers of HCT/Ps Understand Regulatory Pathways for Specific Products
The U.S. Food and Drug Administration is announcing a temporary program called the Tissue Reference Group (TRG) Rapid Inquiry Program (TRIP), which will operate as part of FDA’s Tissue Reference Group.
The program will assist manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps), including those stakeholders that market HCT/Ps to healthcare professionals and/or patients, obtain a rapid, preliminary, informal, non-binding assessment from the FDA regarding how specific HCT/Ps are regulated.
The agency intends to respond to inquiries that contain sufficient detail for evaluation within three days of receipt, as resources permit. The program will be available until December 31, 2019.
View full FDA announcement:
Source: U.S. FDA
Abeona Becomes First Company to Secure Two RMATs
Abeona Therapeutics announced the U.S. FDA granted a Regenerative Medicine Advanced Therapy (RMAT) designation to ABO-102, the company’s AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.
It was the 16th RMAT designation awarded by the FDA and a landmark achievement in that it makes Abeona the first company to achieve two RMAT designations.
Headquartered in Dallas, TX, Abeona (NASDAQ: ABEO) is a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases. Abeona’s first RMAT designation was for its EB-101 gene therapy for Epidermolysis Bullosa in January 2018. [Read more…]
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