CG From the

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. Cell Therapy Trial Targets Type 1 Diabetes In Children

    Getting adult patients to take part in a clinical trial is difficult. When developing a treatment for a childhood disease, that recruitment challenge becomes even more difficult. The combination of patient age and the experimental nature of a treatment can cause concern amongst both parents and the FDA. Caladrius Biosciences is a company dealing with that challenge. The company is developing a cell therapy technology based on a patient’s own T-cells.

  8. 5 Best Practices For Engaging With Patient Advocacy Groups

    Patient recruitment has always been a challenge for companies conducting clinical trials. But when you are developing treatments in the rare disease space, finding patients to take part in your trials becomes an even greater hurdle to overcome.

  9. Are Your Cell Therapy Samples Getting The Preservation They Deserve?

    With the number of biologics now in development and soon to be making their way into clinical trials, the preservation of cells, tissues, and organs are suddenly increasing in importance. Companies handling the manufacturing, storage, and transportation of these materials need to be focused on improving the yield and extending the shelf life of these time and temperature sensitive biologics.

  10. How One Rare Disease Firm Merged Gene Therapy With Patient Centricity

    Abeona Therapeutics is named after the Roman goddess who was the protector of children as they start out on their journey. Since launching as a private company in 2013, Abeona has partnered with a dozen international foundations to fund its development efforts. Currently it is seeking a treatment for children with Sanfilippo Syndrome, and engaging caregivers to determine how to best meet the needs of patients taking part in trials.