Cell therapy weekly: Kyverna Therapeutics appoints new Senior Vice President – RegMedNet

This week: Kyverna Therapeutics appoints new Senior Vice President, Nucleus Biologics obtains ISO 13485:2016 Certification for manufacture and distribution of cell and gene therapy media, rare pediatric disease designation granted to iECUREs investigational gene editing product candidate for OTC deficiency and construction completed on Sheffield Gene Therapy Innovation and Manufacturing Centre.

The cell therapy company focusing on regenerative treatment of serious autoimmune diseases, Kyverna Therapeutics (CA, USA),has appointed Tom Van Blarcom as Senior Vice President, Head of Research. Kyvernas therapeutic platform utilizes advanced T-cell engineering and synthetic biology techniques to suppress and eliminate the autoreactive immune cells responsible for inflammatory and autoimmune diseases.

President and CEO of Kyverna, Dominic Borie stated, We are excited to welcome Tom to the Kyverna team. His broad experience in cell therapy research across a wide range of diseases will be invaluable in supporting our work developing engineered T-cell therapies for the treatment of autoimmune diseases. Toms leadership and extensive industry experience will be a critical pillar of our company as we advance our Regulatory T-cell platform and CAR-T programs to achieve our mission of bringing curative living medicines to life to free patients from the siege of autoimmune disease.

Nucleus Biologics (CA, USA) announced that it has received an ISO 13485:2016 certification from the British Standards Institution (London, UK) for the manufacture and distribution of media for the cell and gene therapy industry. ISO 13485 is the industry standard for quality management systems regulating medical devices and associated services and ensures that the design, development and production of a product consistently fulfils customer and regulatory requirements.

David Sheehan, CEO and Founder of Nucleus Biologics acknowledged, This milestone is the result of years of effort to extend our leadership in custom cell culture media for the cell and gene therapy market. Now, therapy developers have one partner that can offer everything from formulation development support to cGMP 2,000-liter media manufacturing all governed by strict adherence to the ISO 13485 level quality system. Our history of product innovations, quality and collaborations will only expand as we help our customers speed the time from discovery to cure.

iECURE (PA, USA) reported that the US FDA has granted rare pediatric disease designation to GTP-506 for treatment of Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder. iECURE is a gene editing company developing mutation-agnostic in vivo gene insertion therapies to treat liver disorders with significant unmet need. GTP-506 is a potential single dose dual vector gene editing product candidate, designed to restore metabolic function through cleavage of the PCSK9 gene locus and insertion of a functional OTC gene into the cleavage site.

Joe Truitt, CEO of iECURE stated, Receiving Rare Pediatric Disease Designation for GTP-506 for the treatment of OTC deficiency highlights the dire need for new treatment options for this devastating pediatric disease. GTP-506 is a potentially transformative therapy for babies born with OTC deficiency and we expect to file an investigational new drug application with the FDA for our first-in-human clinical trial in mid-2023.

The University of Sheffield (UK) announced the completion of construction for The Sheffield Gene Therapy Innovation and Manufacturing Centre (GTIMC). The state-of-the-art center will provide translational and regulatory support in conjunction with training and skills programs in good manufacturing practice. The GTIMC is one of three innovative centers in a new 18 million network funded by LifeArc (London, UK) and the Medical Research Council (London, UK), with support from the Biotechnology and Biological Sciences Research Council (Swindon, UK).

Mimoun Azzouz, Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield stated, Sheffield has emerged as one of the leading players in cell and gene therapy and this national network of partners, facilities and training programs will allow us to stay at the cutting edge of translational discoveries for new and potentially life changing treatments. Seeing the construction work completed is an exciting milestone for the team. It brings us closer to being fully operational and able to progress new and exciting discoveries, which will benefit patients and families worldwide.