Industry Insights

1. What Pharma Can Learn From A Parent Turned Patient Advocate 4/2/2020

   I am humbled and honored to introduce you to Andrew McFadyen — founder of the Isaac Foundation. Andrew is a husband, a father, and an advocate who tirelessly works to advance actionable dialogue around access, care, and policy for patients with rare diseases. Recently, I had the opportunity to chat with Andrew about the unique challenges that patients and their families face when dealing with serious illness and how we may better serve their needs.

2. Why Controlling CQAs Isn’t Good Enough For Gene & Cell Therapies 3/31/2020

   One of the most frequent statements made in the biopharmaceutical industry is the need to “control a product’s critical quality attributes (CQAs) by controlling the process’ critical process parameters (CPPs).” While the statement is accurate literally, it does not convey the true technical requirements for controlling product quality.

3. Looking Beyond Alpha-Beta Cells for Cell Therapies, Part 2 3/30/2020

   As the limitations of chimeric antigen receptor (CAR)-modified αβ T cells have become clearer, cell therapies of the innate immune system (such as γδ T and NK cells) have been advanced to meet unmet needs.

4. The ABCs of Oncology: Looking Beyond Alpha-Beta Cells for Cell Therapies Part 1 3/26/2020

   In this article, part one of a two-part series, we explore the evolution of cell therapies of the innate immune system (such as γδ T and NK cells) to understand why these approaches offer great hope for oncology and the potential value they may offer second-generation cell therapy companies. Next week we will dive deeper into this burgeoning industry and the potential commercial impact these therapies may offer.

5. Beyond COVID-19: The Need For Sustainable Change In The Life Science Industry 3/25/2020

   Aside from the obvious economic impact, the most important implication from this pandemic may be our limited ability to rely on healthcare systems and governments for proper diagnosis and treatment of infectious diseases.

6. Stem Cell Therapy Trial Offers Hope To Osteoarthritis Patients 3/17/2020

   Cynata Therapeutics is producing products called mesenchymal stem cells (MSCs), which have huge therapeutic potential for numerous unmet medical needs. The company’s Cymerus technology addresses many of the challenges and complexities of manufacturing stem cells at commercial scale.

7. U.S. Manufacturing On The Rise: Add Another CDMO To The Heartland 3/16/2020

   Ascending U.S.-based CDMOs may exemplify a national taking back of our own supply-chain destiny. Now there’s another CDMO, Andelyn Biosciences, taking shape in Central Ohio, and targeting gene-therapy services.

8. Why Complex Disease Needs Gene Therapy 3/3/2020

   The promise of gene therapy has never been greater. Indeed, following recent approvals in large global markets, innovations and companies are growing at breakneck pace. While gene therapy is poised to revolutionize the treatment of rare monogenic diseases, the need to treat chronic and complex diseases looms large on the horizon.

9. An Analysis Of FDA’s Recently Issued Gene Therapy Guidances 2/20/2020

   In this article, we'll examine key changes to FDA's six finalized draft guidance documents on its framework to advance gene therapy. We'll also review a new draft guidance on determining “sameness” of gene therapy products for orphan drug designation/exclusivity.

10. Part 2: Cell And Gene Therapy Is Transforming Healthcare 2/13/2020

    Healthcare sector players are neither adequately equipped nor strategically placed near patients to accommodate the anticipated volume of clinical trials to evaluate cell and gene therapies (CGT) in development. While the industry is adjusting and deploying the required capabilities, the transformation is evolving and incomplete. Real change requires rethinking how current sector players are adopting and how they will operate in the future.