Cell therapy weekly: expanding personalized autologous cell therapy production
This week: Aspen Neuroscience (CA, USA) shared progress on advancing the automated production of its cell therapy for Parkinson’s disease and Viralgen (San Sebastian, Spain), a Contract Development and Manufacturing Organization, announced a partnership with two research laboratories to develop an innovative gene therapy for a fatal neurodegenerative disease. Plus, Sana Biotechnology (WA, USA) reported positive results from a first-in-human study of its allogeneic islet cell therapy for type 1 diabetes.
The news highlights:
- Expansion of personalized autologous cell therapy production
- Partnership to advance gene therapy manufacturing for prion disease
- Positive results from type 1 diabetes study
Expansion of personalized autologous cell therapy production
Aspen Neuroscience, a clinical-stage company, is expanding the automated production of its investigational cell therapy for Parkinson’s disease, ANPD001. This therapy uses dopaminergic neuronal precursor cells (DANPCs), derived from patient-specific induced pluripotent stem cells (iPSCs), to replace lost dopamine-producing neurons in the brain without the need for immunosuppressive drugs.
To support these advancements, Aspen has expanded its San Diego facility and partnered with Mytos (London, UK), an automated cell manufacturer, to enhance automation in producing DANPCs, enabling more efficient patient treatment. In addition, Aspen is collaborating with Cell X Technologies (OH, USA) to optimize the iPSC manufacturing stage, further acting on its intentions to develop personalized cell therapies for patients with unmet medical needs.
“This collaboration with Mytos addresses an important step for automating the manufacturing process for differentiation of DANPCs from [iPSCs],” expressed Kim Raineri, Chief Technology Officer at Aspen Neuroscience. “The Mytos platform, combined with our bioinformatics and other enabling technologies, will allow us to further automate production of ANPD001 and treat greater numbers of patients with their own cells.”
Partnership to advance gene therapy manufacturing for prion disease
Viralgen has partnered with the Broad Institute of Massachusetts Institute of Technology and Harvard University’s (MA, USA) The Vallabh/Minikel Lab and The Deverman Lab (both MA, USA) to develop a gene therapy for prion disease, a rare, fatal neurodegenerative condition resulting from misfolded proteins.
Sonia Vallabh, co-leader of the Vallabh/Minikel Lab commented on the importance of the collaboration, stating:
“We’re incredibly lucky to be doing this work at this exact moment in time. Because prion disease is a whole brain disease, we’ve faced two major challenges from the beginning: finding a molecular machine that can target our gene and delivering that machine to enough neurons in the adult human brain to make a difference. It is just now becoming imaginable that, with the right team, we could piece together answers to these two big questions in our lifetime. We’re so fortunate to have the chance to try.”
This collaboration will leverage Viralgen’s expertise in adeno-associated virus technology and its Pro10™ platform to identify the best components for optimal manufacturing and effectiveness, aiming to delay or prevent the onset of prion disease.
Positive results from type 1 diabetes study
In a first-in-human study, a type 1 diabetes patient received UP421, an allogeneic primary islet cell therapy, without the need for immunosuppression. Engineered with Sana’s hypoimmune (HIP) technology, the UP421 cells survived and functioned well four weeks after transplant, with no safety concerns and successfully evaded immune detection.
Organ and islet cell transplants typically require medication to prevent the body from rejecting them and attacking itself. Sana’s HIP technology aims to allow transplanted cells to avoid both types of rejection.
“As far as we are aware, this is the first study showing survival of an allogeneic transplant with no immunosuppression or immune-protective device in a fully immune competent individual,” said Steve Harr, Sana’s President and CEO. “Safe cell transplantation without immunosuppression has the potential to transform the treatment of type 1 diabetes and a number of other diseases. We view the insights from the current study as directly applicable to developing SC451, our HIP-modified, stem cell-derived pancreatic islet cell program for the treatment of type 1 diabetes. Thank you to everyone involved in this study.”
