The explosion of gene therapy candidates in clinical development is being led by a diverse set of originators, including many hospital and university labs. While these are great incubators for innovation, the many small labs working in isolation do not have experience in commercializing a medicine for wide and sustained use in patients, and this could slow the acceptance of gene therapies for the people who really need them. Health authorities have recognized the importance of getting gene therapies into standard use and are supporting doing so with accelerated pathways.

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 (CT/GT). These therapies, once considered a kind of “wild-west,” are becoming more widely accepted. Still, the task of seeking approval from the FDA can be extremely challenging. It’s become vital to get your relationship with the FDA off to a solid start from day one.

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.

Current reimbursement models in general do not accommodate many of the unique factors that are common among gene and cell therapies.

Clinical trials are complex, but adding genomics to the equation has the potential to make them even more complicated. However, much of the genetic testing that is now being performed in the clinical space is done before a trial begins. That means companies need to do some thinking before adding a genetic test to the protocol.

Janet Lambert, joined the Alliance for Regenerative Medicine (ARM) as CEO just over a year ago. And what a year it’s been. Recently, I had the opportunity to talk to Lambert about her first year as CEO, what ARM is doing in cell and gene therapies from clinical and commercialization points of view, as well as its areas of short-term focus. Here’s what she had to say.

The FDA has been issuing guidance documents addressing gene therapy development issues for approximately 20 years — a remarkable dedication of resources to an area that did not have a licensed product until 2017. Of the six gene therapy-related draft guidances the agency issued last month, two represent the first of the “suite of disease-specific guidance documents on the development of specific gene therapy products” Commissioner Scott Gottlieb promised in Dec. 2017. 

Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?

The FDA recently issued six draft guidance documents. This article discusses the guidance Human Gene Therapy for Hemophilia.

This is the fifth article in a six-part series summarizing each of the FDA's gene therapy draft guidance documents. It will discuss the draft guidance Human Gene Therapy for Retinal Disorders.

The FDA recently issued for public comment six draft guidance documents. This is the fourth article in a series and will discuss the guidance Human Gene Therapy for Rare Disease.