This week in cell therapy: Kymriah will be available for children but not adults on the NHS and CRISPR Therapeutics and ViaCyte will collaborate on the development of gene-edited diabetes therapy.
The news highlights:
New hurdle in immunotherapy development
Kymriah rejected for adult treatment, follows recent pediatric approval
CRISPR Therapeutics and ViaCyte will collaborate on the development of gene-edited diabetes therapy
New research from the University of Notre Dame (IN, USA) has demonstrated that peptides can move and adapt in order to facilitate receptor binding. In the case of T-cell receptors, which are utilized in immunotherapy, this can lead to the modified T-cells binding to more than one peptide which could have very different properties to the target peptide and could explain why T-cell therapy sometimes affects healthy cells.
“What’s significant is that people try to make predictions for developing these models for therapy, and about the kinds of ways you can recognize targets,” commented Brian Barker, chair of the Department of Chemistry and Biochemistry (Notre Dame) and principal investigator. “And this is a new, unanticipated complication.”
Despite recent approval for Kymriah (Novartis; Switzerland) as a pediatric treatment on the NHS, it has been deemed too expensive or adult patients. This news puts it on par with Yescarta (Kite, a Gilead company; CA, USA), which recently received the same judgement.
“Tisagenlecleucel is not recommended, within its marketing authorisation, for treating relapsed or refractory diffuse large B-cell lymphoma in adults after 2 or more systemic therapies,” NICE said in the draft guidance.
However, in a press release published with the guidance, NICE commented that “NICE does recognise that Novartis’ tisagenlecleucel-T, also known as Kymriah, has significant clinical benefits, and would welcome further discussions on its cost-effectiveness.”
CRISPR Therapeutics (Switzerland) and ViaCyte, Inc. (CA, USA) have announced they will collaborate on the discovery and development of gene-edited allogeneic stem cell therapies for diabetes. Under the agreement, the two companies will aim to develop an immune-evasive stem cell line and subsequently a product candidate to commercialize worldwide. The deal is initially worth up to US$25 million in either cash or CRISPR stock.
“Creating an immune-evasive gene-edited version of our technology would enable us to address a larger patient population than we could with a product requiring immunosuppression. CRISPR Therapeutics is the ideal partner for this program given their leading gene editing technology and expertise and focus on immune-evasive editing…[this] could be a transformational therapy for patients with insulin-requiring diabetes,” commented Paul Laikind, Chief Executive Officer and President of ViaCyte.
For more weekly cell therapy news, read previous editions of the cell therapy weekly.