A successful Initial Targeted Engagement for Regulatory Advice on CBER products (INTERACT) meeting with CBER paved the way for the early product manufacturing activities and very early preclinical safety and efficacy evaluations. A pre-Investigational New Drug (Pre-IND) meeting provided additional clarity on the IND-enabling activities needed to support an initial IND submission in support of a Phase 1 first-in-human (FIH) study.

With the recent breakthroughs in cell and gene therapy, there is increased emphasis on the design and implementation of different supply chain models to support the movement of materials and drug product across the chain of care. Unlike more traditional supply chains, many of these therapies have unpredictable sources and manufacturing and infusion locations. The most extreme example is loosely referred to as the “vein-to-vein” supply chain — particularly for autologous therapies.

Nicole Gularte is currently battling cancer for the 8^th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.

The FDA published a new draft guidance on August 10, 2018 entitled, Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics. The comment period for the draft guidance closed on October 12, 2018 and approximately 20 comments were submitted to the docket. The guidance provides sponsors with recommendations for designing and conducting first-in-human (FIH) multiple expansion cohort trials within their oncology development programs.

Progress in development of gene and cell therapies around the world has potential to transform standards of care for a range of diseases and address significant areas of unmet need in healthcare over the coming years. In the U.S. alone, almost 20 gene and cell therapy products have been approved thus far,¹ with many other development programs reaching later clinical stages. The technology platforms of many of these drugs also offer the potential for curative efficacy and expansion for use in multiple indications.

Cell & Gene Meeting on the Mesa took place last month in La Jolla, CA, and it’s no surprise that ARM delivered meaningful data and addressed cutting-edge research to more than 1,200 attendees. I caught up with ARM’s CEO Janet Lambert after the Meeting to get her take on top highlights, the most informative panel discussions, as well as a look ahead to next year’s event. Here’s what she had to say.

Commercial viability of pharmaceuticals depends on getting medicines to patients, which requires developers to evaluate investigative therapies using outcomes that are quantifiable, easy to interpret, and clinically relevant. However, clinical relevance is often a matter of stakeholder perspective: what developers view as meaningful may not appear as such to patients and caregivers.

Biopharmaceutical processes — and the technologies that enable them — have experienced remarkable progress in the past 30 years, since the first biopharmaceuticals were approved for human use. This article will highlight some of today’s top trends in bioprocessing, as this exciting field continues to flourish.

CMOs are positioned to play a leading role in standardizing control strategies for gene therapy because so many gene therapy products are funneled through CMOs for commercial scale production.

In part two of my discussion with Miguel Forte, CEO at Zelluna Immunotherapy and ISCT’s Chief Commercialization Officer, he answers my questions about the Society’s near-term goals and objectives as well as his top areas of focus as CCO.

Seeking early guidance from regulators can be invaluable when navigating preliminary product development strategies. The need for feedback increases significantly when working with cell and gene therapy products. 

The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.

For patients with sickle cell anemia, just getting through the day can be a struggle. Symptoms of the disease include vision problems, swelling of the hands and feet, and periodic episodes of pain that can last from a few hours to a few weeks. In this article Nick Leschly, CEO at bluebird bio, discusses the progress being made with its investigational gene therapy for sickle cell disease.

In part one of my discussion with Miguel Forte, CEO at Zelluna Immunotherapy and Chair of Commercialization Committee at ISCT, he answers my questions about academic-to-industry translation, the root cause of unproven therapies, how the industry is combatting them, and more.

A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations

According to a review of recent research studies that measured U.S. public opinion related to gene medicine, the public is not yet aware of gene medicine, is unfamiliar with the terms being used to describe the topic, and, when faced with multiple therapeutic options, is worried about making informed decisions.

Cell & Gene Editorial Advisory Board member answers questions about his message to pharma executives, researchers, scientists, and doctors, what the industry should consider when developing new payment structures, and more.

The FDA’s RMAT and breakthrough designations have created a streamlined environment for product sponsors. To effectively leverage this regulatory policy environment, sponsors must conduct high-quality clinical trials that are often operationally complex. Clinical trial networks, capable of managing the array of regenerative medicine technologies, are well suited to manage this complexity.

Peter Marks, MD, PhD, Director Center for Biologics Evaluation and Research at U.S. Food and Drug Administration answered some of my questions about the current state of the FDA’s regulations around cell and gene therapies, its stance on international harmonization required to make cell and gene therapies more streamlined, the reasons why it is critical for companies to begin working with the FDA early in the development process, and more. Read on for Dr. Marks' insightful responses.