Cell therapy weekly: Funding for islet-replacement diabetes and retinitis pigmentosa therapies
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This week: New funding will support development of cell-based therapies and Solid Biosciences partial clinical hold is lifted.
The news highlights:
Partial clinical hold on high dose SGT-001 resolved, additional information requested on full clinical hold
NIH grant will further development of replacement beta cells for type 1 and type 2 diabetes
New trial will assess MSCs to improve heart function in heart failure patients
ReNeuron wins grant for retinal cell therapy development
Partial clinical hold on high dose SGT-001 resolved, additional information requested on full clinical hold
Following the partial clinical hold placed on the high dose of SGT-001 in Solid Biosciences Inc.’s (MA, USA) trial program for Duchenne muscular dystrophy, the FDA’s questions regarding the manufacture of the product have been addressed, resulting in the hold being lifted. A later full clinical hold, in response to a serious adverse event in the first patient dosed with SGT-001 in the IGNITE DMD trial, remains in place pending additional information.
“We believe SGT-001 has the potential to significantly benefit patients with DMD. We are working with the FDA to better understand the unexpected event that resulted in the clinical hold and put a plan in place to monitor and manage potential events in the future. I am happy to share that the treating physician has reported the patient is doing well,” explained Ilan Ganot, Chief Executive Officer of Solid Biosciences. “We are also pleased that we were able to lift the earlier partial clinical hold on the high dose of SGT-001 through our existing manufacturing process, which is an important ongoing development effort for our company.”
NIH grant will further development of replacement beta cells for type 1 and type 2 diabetes
ViaCyte, Inc. (CA, USA) has received a Small Business Innovative Research Phase III award from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK; MD, USA) to further the development of stem cell-derived functional human beta cells in vitro. ViaCyte has two product candidates in human clinical trials using pancreatic progenitor cells. Following implantation, the cells should develop into human islet tissue, capable of regulating blood glucose.
“We are tremendously grateful to the NIDDK for their support of our research characterizing pluripotent stem cell-derived human beta cells in vitro as well as our investigations into manufacturing methods for developing these cells into therapeutic products,” said Kevin D’Amour, Vice President, Research and Chief Scientific Officer at ViaCyte.
“This SBIR funding will allow us to more completely explore and establish this more mature cell population as an additional option to our existing transformative cell therapies for type 1 and insulin-requiring type 2 diabetes.”
New trial will assess MSCs to improve heart function in heart failure patients
A trial to assess the anti-inflammatory properties of mesenchymal stem cells (MSCs) to improve heart function in patients with heart disease. There are nearly six million Americans with heart failure and the trial, utilizing CardioCell’s proprietary MSCs, manufactured by Stemedica Cell Technologies (both CA), could offer new strategies to treat patients with heart failure and left ventricular assist devices (LVAD).
“We have developed compelling evidence that one of the major mechanisms leading to progressive myocardial dysfunction in patients with heart failure is the presence of persistent and inappropriate inflammation,” said Stephen Epstein, director of Translational and Vascular Biology Research at MedStar Heart & Vascular Institute (DC, USA).
“If we are successful in showing stem cells improve outcomes in LVAD patients, the results would extend to the general population of heart failure patients and, in the process, fundamentally transform current paradigms for treating heart failure patients.”
ReNeuron wins grant for retinal cell therapy development
ReNeuron Group plc (UK) has received a new £1.5 million grant from Innovate UK to advance development of its human retinal progenitor cell (hRPC) candidate for retinitis pigmentosa, currently being tested in a US Phase I/II trial. The funding will allow generation of further cell banks of the hRPCs and development of assay for late-stage clinical development and therapy commercialization, in collaboration with ReNeuron’s partners, the Cell & Gene Therapy Catapult, University College London (both London, UK) and Roslin Cell Therapies (Edinburgh, UK).
Sharon Grimster, General Manager Wales, ReNeuron, commented: “This is a challenging but vital part of the broader commercial manufacturing strategy for the therapy and we are pleased to be working with leading academic and commercial manufacturing partners on this important program of work.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.