White Paper: Moving Clinical Development Forward in 2023
In today’s cell and gene therapy landscape, there are over 1,400 companies striving to move their products forward into clinic—a critical step in commercializing these groundbreaking treatments. Clinical development is a complex endeavor and the path to market requires careful consideration of factors ranging from chemistry, manufacturing, and controls (CMC) and regulatory strategy to study design, CRO selection, and clinical trial diversity.
White Paper: The Price is Right: Cell and Gene Therapy Approvals and Market Access in 2023
Starting health economic evidence development and market access planning as early as possible in clinical development for a cell and gene therapy is important demonstrating the value to payers and health technology appraisal (HTA) organizations. In a dynamic regulatory and market access landscape, balancing the requirements of regulators and the expectations of payors can be challenging since there is no one-size-fits-all approach.
White Paper: The Future is Now: Cell and Gene Therapy Innovation, Challenges, and Perspectives
Cell and gene therapies have the potential to revolutionize treatment of a wide range of diseases. With six new therapies approved in 2022, including the first allogeneic T-cell therapy, and at least as many slated for approval this year, the sector is poised for rapid growth. This innovation comes with challenges, though, and many consider 2023 to be a seminal year for establishing a strong foothold for advanced therapies in mainstream medicine.
Executive Summary: State of the Industry
Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), kicked off the event with a briefing on the state of the cell and gene therapy development landscape. With 475 members worldwide, ARM focuses on convening the sector, providing data and analysis, engaging with stakeholders, and enabling the development of advanced therapies. One of their key initiatives is modernizing healthcare systems to enable access to these novel therapeutics. Highlights of this briefing are provided in this summary.
Article: Innovations in Gene Therapies
Despite innovation to date in gene therapy, safety concerns with adeno-associated virus (AAV) vectors and uneasiness around cost and affordability has brought into focus the need for even more advancement in the field. In this Pharma Live article, Precision’s Phil Cyr discusses some of the innovations that are occurring to address these issues.