Cell therapy weekly: CCRM and GE Healthcare to assist in scale-up of degenerative disc disease therapy
This week: FDA places sickle cell disease trial on hold and new partnership will develop CAR-T for solid cancers.
The news highlights:
CCRM to assist with commercialization of off-the-shelf therapy for degenerative disc disease
FDA clinical hold delays commencement of sickle cell trial
Australia-US partnership will see development of CAR-T cell therapies for solid cancers
DiscGenics (UT, USA) will enlist the support of CCRM (ON, Canada) and GE Healthcare (UK) to scale and optimize the manufacturing process for its injectable cell therapy for degenerative disc disease. ‘Discogenic cells’ introduce progenitor cells, isolated from donated intervertebral tissue, to the damaged disc. A first-in-human trial is underway that aims to treat 60 patients with lumbar degenerative disc disease in three cohorts with either a single injection of discogenic cells, saline or sodium hyaluronate.
“Manufacturers need to be able to duplicate product attributes at larger scale as they progress through clinical trials and into commercial phases, so it’s critical to develop scaled up manufacturing processes early on,” explained Phil Vanek, GM of Cell and Gene Therapy Strategy at GE Healthcare Life Sciences.
“The DiscGenics project is an excellent case study of [our] ability to successfully accelerate scale-up and process development,” commented Michael May, President and CEO, CCRM. “This is yet another example of how CCRM and GE Healthcare are fulfilling their mission of supporting cell and gene therapy by industrializing cell manufacturing and delivering results that help customers develop better treatments for patients.”
Vertex Pharmaceuticals (MA, USA) and CRISPR Therapeutics’ (Switzerland) phase I/II trial of an investigational ex vivo CRISPR gene-edited therapy, CTX001, for sickle cell disease has been delayed following the placement of a clinical hold on the trial by the FDA. Following the hold, shared in CRISPR Therapeutics dropped 19%. According to the two companies, the status of a phase I/II trial of CTX001 in Europe in adults with transfusion dependent β-thalassemia is unchanged.
"We're working with the FDA and will respond as soon as we can about whatever questions they have regarding the trial," added CRISPR spokesman Chris Erdman.
An US$9.6 million partnership between Mesoblast Limited (Melbourne, Australia) and Cartherics Pty Ltd (NY, USA) will see development of allogeneic CAR-T cell therapies able to target multiple receptors for solid cancer indications. The funding has come from collaborators in the Australian Government's Cooperative Research Centres Program, including Monash University, Hudson Institute of Medical Research and Cell Therapies Pty Ltd, and intellectual property will be jointly owned by the two companies.
“We’re very excited to now be in a position to produce unique, timely and cost-effective off-the-shelf therapies that may remove many barriers to treatment for cancer,” said Alan Trounson, CEO, Cartherics.
Mesoblast Chief Executive Silviu Itescu stated, “With our combined technology platforms and expertise, we are ideally placed to greatly increase accessibility to this very promising new field of cancer therapeutics through the development of highly-scalable, allogeneic cellular immunotherapies.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.