Gene & Cell Therapies – It’s Show Time!

12 January, 2018

dna_1-1.jpgGene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the removal of deleterious genes or genome sequences.

In the last several years there has been tremendous progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. Advances in delivery technologies are also creating more opportunities for genome editing, including ex vivo delivery to cells with DNA-free components and in vivo delivery with efficient and tissue-specific vectors. The many successes outlined in recent preclinical studies and the current progression of genome editing in clinical trials is a source of significant optimism for the future of the field and is reflected in the willingness of pharma, new biotechs and investors to make significant financial commitments.

Nevertheless, many challenges still remain to fully realize the potential of genome editing for gene and cell therapy. Central to these challenges are the persistent issues of safety and delivery. Rapid advances are being made both for increasing the specificity of genome-editing tools and also increasing the sensitivity of methods used for assessing this specificity genome-wide. However, it remains unclear whether all off-target effects can be accounted for in a therapy that targets one site with billions of DNA base pairs, involves the modification of millions of cells, and is custom prepared for each patient. Questions still remain about how the human immune system will respond to genetically modified cells or the in vivo administration of genome-editing tools.

FDA_flag.pngThat being said, it seems that hurdles are overcome every day. The recent approvals by the FDA of three gene and cell therapies indicate that a therapeutic paradigm shift is upon us.

  • On August 30, 2017, Novartis received the first-ever FDA approval for a CAR-T cell therapy, Kymriah, for children and young adults with B-cell ALL.
  • On October 18, 2017, the FDA approved Kite's Yescarta, a CAR-T cell therapy that treats adults with certain types of large B-cell lymphoma.
  • On December 19, 2017, the FDA approved Luxturna, developed by Spark Therapeutics. Luxturna is a novel gene therapy that treats patients with a rare form of inherited vision loss.

All three of these programs demonstrated dramatic efficacy in previously hopeless clinical situations, paving the way for a busy pipeline of advanced therapeutics expecting to seek FDA approval over the next year. In fact, FDA Commissioner Scott Gottlieb anticipates that the FDA may approve one gene and cell therapy each month over the course of 2018 under the Regenerative Medicine Advanced Therapies designation created last year to expedite review of these unique therapies.

To learn more about this exciting field of advanced therapeutics, ShareVault partnered with Mike Rice of Defined Health’s Gene and Cell Therapeutics and Rare Diseases practices to present a webinar discussing the exciting advances in the field of gene and cell therapies and its remaining challenges, such as its quite complex reimbursement issues.

During the webinar recording, Mike discusses:

  • Investment and deal trends driving the recent advances in gene transfer and cell processing that have enabled the realization of advanced therapies
  • Implications for biopharma business models and changes needed to make curative intent treatments with respect to complexities such as individual manufacturing and single or short course administration.
  • Market access and risk-sharing strategies for high value priced therapies that currently have minimal data from small patient study cohorts with limited longitudinal evidence of durable therapeutic benefit.

To learn more surrounding the exciting potential and the remaining hurdles for advanced gene and cell therapies request your copy of the webinar recording today.