Synpromics joined the BSGCT in Sheffield at the end of June to share the latest progress in gene and cell therapy. The Annual Meeting kicked off to a great start with a welcome networking reception, sponsored by Synpromics, held in the beautiful Winter Garden. This provided an excellent opportunity to catch up with current and possible future collaborators.

Synpromics team at BSGCT

The Synpromics team at the BSGCT Annual Meeting

The opening key note speech by Roger Barker (University of Cambridge) set the tone for the conference. His talk about the treatment of Parkinson’s disease with transplanted reprogrammed neuronal stem cells was inspiring. It was interesting to see how far along the current gene therapy treatments are progressing. For example, the imminent start of a new clinical trial to treat Cystic Fibrosis using a recently developed lentiviral vector. 

Alongside the advances in therapy treatments was the progress in scaling up these treatments. From improving and increasing viral production with new producer cell lines to developing personal CAR-T into a one cell therapy treatment for all patients (allogenic treatment). There was particular focus on how this would benefit production, decrease time and therefore reduce the cost of each treatment, increasing accessibility for patients. Talks about AAV mediated therapies highlighted the importance of getting the model of AAV right before progressing to clinical trial. For example, which combination of AAV serotype and capsid might be best. The challenge here comes from the different behaviour of the therapy from proof of concept in animal models to humans and its lack of predictability.

There was a strong emphasis on the translation of gene therapy from academic to clinical applications, with talks addressing issues such as cell delivery and storage (freezing CAR-T cells, viability post-thaw, delivery to patient room in hospital, delivery to patient) and increasing viral production to name a few. What was particularly interesting was the support available for the translation of research to bedside. This is necessary because it was clear that a huge number of affiliations and ground work are needed to progress a therapy to clinical trials.

An exciting key note from Stephan Grupp (Children’s Hopsital of Philadelphia) walked the audience through the process of developing the approved CAR-T treatment Kymriah; a great example of taking gene therapy research into the clinic to change patients’ lives. 

Dr Maha Tijani

Dr Maha Tijani presenting

Among many thought-provoking discussions, Dr Maha Tijani shared some updates from our partnership with UCL. The project aims to use our synthetic promoter technology to develop novel promoters for CNS gene therapies. Dr Michael Roberts also presented some of our latest progress in his talk: ‘Mastering Gene Control to Enable the Next Generation of Gene Medicines’. Michael’s presentation can be viewed here.

Dr Michael Roberts

Dr Michael Roberts presenting

Synpromics will be heading to California in October for the ARM Cell & Gene Meeting on the Mesa so we look forward to catching up with you there.