INSIGHTS ON CELL & GENE COMMERCIALIZATION ISSUES

• Strategies To Address The Viral Vector Manufacturing Shortage

  Accelerating the production of your viral-vector based therapy requires you to understand the strategies available to address the viral vector manufacturing shortage as well as selecting the most cost-effective solution for advancing your therapeutic.

• Cell & Gene Therapy Deals: Emerging Trends To Watch

  The pace of deal-making in cell and gene therapy is faster and occurring much earlier in the drug development process compared to what has historically been seen for other innovative therapies. To understand how the landscape is evolving, we reviewed more than 30 deals in the sector and compared them to deals that were executed for mAbs between 1999 and 2013.

• Non-Hodgkin’s Lymphoma CAR T-Cell Therapy: Where Do We Go From Here?

  Following the success of Kite/Gilead’s Yescarta and Novartis’ Kymriah in treating relapsed or refractory diffuse large B-cell lymphoma, key players are pursuing therapies in earlier treatment settings and in mantle cell lymphoma, chronic lymphocytic leukemia, and follicular lymphoma.

• Planning For Commercial Success – Best Practices For Designing Cell & Gene Therapy Clinical Trials

  Cell and gene therapy applications have, in the last three years, made the jump from pure research products to commercialized products with high clinical efficacy, particularly for hematologic malignancies. This webinar elaborates on how to design early development programs and clinical trials for CGTs to ensure that the product can be commercialized successfully in all target markets i.e. can be reimbursed and adopted successfully despite small patient numbers, rare diseases, complex administration etc.

• Mandatory Hub For Orphan-Designated Pediatric Therapy

  UBC successfully provided Hub services to ease the barriers to access and simplify a very complex treatment initiation in pediatric patients suffering from SMA.

• Pricing Reimbursement Recap From Meeting On The Mesa

  At last month’s Meeting on the Mesa, ARM’s annual can’t-miss event, attendees were treated to insightful keynotes, panel discussions, and breakout sessions that covered all the major topics impacting the cell and gene sector. One such plenary discussion, moderated by Audentes’ Chairman and CEO, Matt Patterson, was particularly valuable in that the panelists offered insight on their companies’ path to commercialization.

• Developing Novel Policy Proposals To Support Access To Gene And Cell Therapies

  The decades-long focus on more “personalized” medicines has paved the way for the recent and anticipated advances in gene and cell therapies and progress in many other areas. The first generation of gene and cell therapies has the potential to transform the way we treat some diseases and can bring new hope to patients with many rare and serious conditions.

• CAR T-Cell Therapies: Current Limitations & Future Opportunities

  Novel technologies to address areas left unaddressed by Kymriah and Yescarta have started streaming into the research arena. This article focuses on the barriers to widespread commercial adoption of the currently available CAR T cell therapies, and opportunities for developers of next-generation treatments.

• A Strategy to Drive Down Therapy Costs: Manufacturing Improvements

  Indeed, earlier this year at World Advanced Therapies & Regenerative Medicine (WATRM) Congress in London, it was predicted that manufacturing will be a major future differentiator between therapy companies. I spoke to Dr. Paul Lammers, MD, MSc, president, CEO, and Director of Triumvira Immunologics about the therapy pricing and manufacturing’s role in it.

• What’s A Cure Worth? ICER Publishes Value Assessment Framework For Curative Therapies

  The Institute for Clinical and Economic Review (ICER) recently conducted a study of value assessment methods considered for “cures” or “potential cures” and solicited input from international HTA bodies. The research will form the basis of a value framework that will be applied in the assessment of potential cures and other treatments that qualify as what ICER refers to as “single or short-term transformative therapies.”