CG From the

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. Japan Pharma: Top Tier With Tons Of Opportunity

    Opportunities related to the world’s third largest pharma industry tend to get drowned out by U.S. domestic events, and news from Europe, China, and even India. But if you haven’t been, it’s time to start paying attention to Japan again. There are opportunities for partnering, providing services to Japan Pharma, and for market penetration by foreign biopharma companies.

  6. Cell Therapy Trial Creates Skin Cells For Rare Disease Patients

    Abeona Therapeutics recently conducted a Phase 1/2 clinical trial for individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is an often fatal connective tissue disorder marked by skin blistering and caused by a lack of a collagen protein responsible for anchoring the dermal and epidermal layers together.

  7. Our 2018 Outsourcing Outlook: Focus On … Amazon?

    It’s time for our 2018 industry outlook. But don’t worry if you’ve already had your share of forecasts slipped into your stockings. I promise ours brings something different to the prognostication party. And it should help readers to consider carefully what you need to focus on to make it another successful year.

  8. Cell Therapy Trials Present Unique Challenges For Pharma

    Maria Fardis, CEO of Iovance Biotherapeutics, has spent 18 years working in the pharma industry. Although most of her experience has been in small molecules and proteins, she now works in the cell therapy space and notes these treatments are a new and exciting space with meaningful differences in how clinical trials are conducted.

  9. Magenta Therapeutics Finds A “Support Chain” Beyond CDMOs

    Biotechs focused on cell and gene therapies need support beyond the traditional outsourcing of development and manufacturing to CDMOs. These elongated “support chains” start within the healthcare system, run cells out to a CDMO for processing, and then circle back to the patient. Jason Gardner, co-founder, CEO and president of Magenta Therapeutics, believes he’s found a partner to help.

  10. CRS Files Lawsuit Against FDA

    The watchdog group Center for Responsible Science (CRS), along with clinical trial participants and the father of a deceased trial participant, has filed a lawsuit against the FDA for denial of CRS’ citizen petition, which was originally submitted in June 2014 and has since had several amendments.