Spotlights 2017

Translation and Application of Gene Editing

February 2017


Prof. George M Church, Professor of Genetics at Harvard Medical School, USA

The rapidly advancing developments in gene editing have numerous implications and applications for the cell and gene therapy field – from disease modelling through to the potential editing of patient-derived cells to modify/correct disorders.

The Gene Editing spotlight will feature updates from leading groups who are focused on utilizing gene editing technologies to advance cell and gene therapeutic approaches.

Raw Materials for Cell & Gene Therapy: Getting it Right from the Start

As the cell and gene therapy sector matures, the requirement for robust and high quality supply of GMP-compliant raw and ancillary materials becomes more and more of an imperative. This spotlight looks at the key current issues with a focus on real-world experiences:
  • Donor screening and selection
  • Optimizing raw materials at source: key considerations for the safe collection and processing of cell source material
  • Characterization of raw materials: identity, purity and functionality testing
  • Understanding the regulatory requirements

Latest Developments in CAR-T Cell Manufacturing

Guest Editor - Dr Isabelle Riviere

The promising clinical results achieved in cancer patients treated with CAR-T cells has provided optimism and subsequent investment in the development of this technology. As we move towards the clinic, it is essential that reproducible manufacturing processes that generate high-quality, clinical-grade CAR-T cell products are developed and refined. This Spotlight discusses the challenges faced across the CAR-T manufacturing pathway, including standardization, automation, quality control, scaleability to meet patient demand, as well as the state of the art manufacturing platforms.

Latest Advances in Addressing Biopreservation Challenges

Examining the latest developments and thinking in preservation and packaging for cell and gene therapies, this spotlight looks at:

  • The latest advances in cryopreservation technology
  • Biopreservation Best Practices for regenerative medicine GMP manufacturing & focus on optimized biopreservation media
  • When is the best time to start identifying your biopreservation and symbiotic supply chain strategy, and how to ensure a positive impact on Cost of Goods?
  • Future goals, including alternative approaches to preservation without cryopreservation

Overcoming Downstream Bottlenecks

Ever more cell and gene therapies are entering clinical development, amplifying the requirement for novel purification systems designed specifically to meet regulatory requirements whilst increasing yield. This spotlight will provide a forum for discussing the current challenges and latest developments in delivering high purity cells and vectors including Harvesting, Purification, Enrichment and Fill-Finish strategies.

Coming in July 2017

Latest Advances in Cord Blood Applications & Commercialization

Guest Editor - Dr Joanne Kurtzberg

This timely spotlight brings you the latest advances and developments in cord blood manufacturing and clinical development. In collaboration with world renowned expert Dr Joanne Kurtzberg, this spotlight will include a selection of content from leading opinion leaders:

  • Emerging use of cord blood in Regenerative medicine
  • Advances in cord blood manufacturing
  • Clinical translation of cord blood
  • Use of cord blood in pediatric inherited metabolic diseases – update on clinical developments
  • Unrelated cord blood transplantation in non malignant and malignant diseases
  • Economics of cord blood banking

Coming in July 2017

Next Generation Vectors

Guest Editor - Dr Richard Harbottle

As cell and gene therapies move towards the clinic, critical developments in the production of alternatives to viral vectors are required to enable large-scale manufacturing and distribution. A overview of the latest developments in the design and advantages of next generation vectors will be provided, complemented by a discussion of the key manufacturing considerations to enable large-scale, quality-assured vectors for clinical use.