• Developing Control Strategies For Gene Therapy Products — What Role Should CMOs Play?
    Developing Control Strategies For Gene Therapy Products — What Role Should CMOs Play?

    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.

  • Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals
    Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals

    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.

  • CMC For Cell & Gene Therapies — 4 Topics To Discuss During Pre-IND Meetings With FDA
    CMC For Cell & Gene Therapies — 4 Topics To Discuss During Pre-IND Meetings With FDA

    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.

Fibrocell: Where Gene Therapy Meets Synthetic Biology
Fibrocell: Where Gene Therapy Meets Synthetic Biology

“We had to decide whether to spend a lot of money to bring on a sales force and marketing team, and expand manufacturing to meet demand, or take our resources and refocus them on these rare disorders.”  

  • State Of The Cell And Gene Therapy Sector — Clinical, Financial, And Regulatory Perspectives
    State Of The Cell And Gene Therapy Sector — Clinical, Financial, And Regulatory Perspectives

    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.

  • Science Driving Gene Therapy Development At bluebird bio
    Science Driving Gene Therapy Development At bluebird bio

    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.

  • ISCT’s Miguel Forte On Academic-To-Industry Translation
    ISCT’s Miguel Forte On Academic-To-Industry Translation

    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.

  • 4 Barriers To Cell And Gene Therapy Development For Rare Diseases — And How To Overcome Them
    4 Barriers To Cell And Gene Therapy Development For Rare Diseases — And How To Overcome Them

    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

  • Bridging The Public Knowledge Gap Around Cell And Gene Medicine
    Bridging The Public Knowledge Gap Around Cell And Gene Medicine

    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.

  • CAR-T And The Voice Of The Patient
    CAR-T And The Voice Of The Patient

    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.

  • Clinical Trial Networks: Overcoming The Complexities Of Regenerative Medicine Studies
    Clinical Trial Networks: Overcoming The Complexities Of Regenerative Medicine Studies

    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.

  • The FDA’s Dr. Peter Marks On The Current State Of Cell And Gene Therapies
    The FDA’s Dr. Peter Marks On The Current State Of Cell And Gene Therapies

    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.

  • New Approaches To Market Access And Reimbursement For Cell And Gene Therapies
    New Approaches To Market Access And Reimbursement For Cell And Gene Therapies

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

  • Is Genetic Testing Creating a Moral Dilemma For Clinical Researchers?
    Is Genetic Testing Creating a Moral Dilemma For Clinical Researchers?

    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.

  • A Sit Down With ARM’s CEO, Janet Lambert
    A Sit Down With ARM’s CEO, Janet Lambert

    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.

  • Surveying The U.S. Regulatory Landscape For Cell And Gene Therapies
    Surveying The U.S. Regulatory Landscape For Cell And Gene Therapies

    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. 

  • 4 Future Scenarios For Cell And Gene Therapy Adoption
    4 Future Scenarios For Cell And Gene Therapy Adoption

    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?

  • Summarizing FDA’s New Guidance On Gene Therapy For Hemophilia
    Summarizing FDA’s New Guidance On Gene Therapy For Hemophilia

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

  • FDA’s New Guidance On Gene Therapy For Retinal Disorders — A Primer
    FDA’s New Guidance On Gene Therapy For Retinal Disorders — A Primer

    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.

  • Gene Therapy For Rare Disease — Examining FDA's New Draft Guidance
    Gene Therapy For Rare Disease — Examining FDA's New Draft Guidance

    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.

More From Cell & Gene

C&G EDITOR IN CHIEF, ERIN HARRIS

  • Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals
    Part 2: ISCT’s Miguel Forte On The Society’s Near-Term Goals

    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.

  • ISCT’s Miguel Forte On Academic-To-Industry Translation
    ISCT’s Miguel Forte On Academic-To-Industry Translation

    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.

  • CAR-T And The Voice Of The Patient
    CAR-T And The Voice Of The Patient

    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 Dr. Peter Marks On The Current State Of Cell And Gene Therapies
    The FDA’s Dr. Peter Marks On The Current State Of Cell And Gene Therapies

    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.

  • A Sit Down With ARM’s CEO, Janet Lambert
    A Sit Down With ARM’s CEO, Janet Lambert

    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.

CELL AND GENE INDUSTRY EVENTS

2018 PDA Cell and Gene Therapy Conference October 23 - 24, 2018
Bethesda, MD
Stem Cell Research, Cell and Gene Therapy November 9 - 10, 2018
Atlanta, GA
Annual Cardiologists Meeting 2018 November 26 - 27, 2018
Madrid
Blockchain Technology for the Life Sciences: A Comprehensive Primer November 27 - 27, 2018
1pm-2:30pm EST, Online Training
Phacilitate Leaders World January 22 - 25, 2019
Miami, FL
Patients As Partners March 11 - 12, 2019
Philadelphia, PA
Car-T Congress USA March 20 - 22, 2019
Boston, MA
American Society of Gene & Cell Therapy (AGCST) April 29, 2019 - May 2, 2019
Washington, DC
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INDUSTRY INSIGHTS

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