SOLD OUT at Tome Biosciences Event at Arsenal on the Charles in Watertown, MA

First of all – have you been to the Arsenal on the Charles Life Sciences complex? It is a gorgeous hub of activity amidst ongoing new construction and renovation of historical brick buildings that, as the name suggests, were once dedicated to the military. On February 15th, the ISPE Boston Chapter’s Educational Program Committee (EPC) was on-site at Tome Biosciences to host Trends in Cell and Gene Editing as part of the chapter’s monthly program series. The event was sold out, which wasn’t a surprise, given the hot topic that cell and gene therapy continues to be.

Following some brief networking, Brooke Cote, ISPE’s outgoing EPC Co-Chair, kicked off the event, introducing Tome’s Chief Technology Officer, Matt Barrows, and Chief Scientific Officer, John Finn. John and Matt led a phenomenal discussion focused on the field of cell and gene editing, how it continues to evolve, and what this will inevitably mean for manufacturing strategies. Tome Biosciences recently launched their Programmable Genomic Integration (PGI) platform and is now furthering development of its two complementary technologies, integrase-mediated PGI (I-PGI) and ligase-mediated PGI (L-PGI, originally from Replace Therapeutics). I-PGI is an integrative gene therapy for diseases with multiple causal mutations, whereas L-PGI will be used for single mutation or for exon replacement.

During the Q&A, John and Matt fielded questions about their “outsourcing decision tree” and which has helped them as they’ve considered the most efficient approaches for moving Tome’s technology forward. As part of the discussion around bottlenecks encountered, they raised the question as to where the industry can take more of a practical, risk-based approach on materials testing. Matt reference some of the lessons learned around minimizing off-target effects from Intellia’s cleared IND application to move their in vivo CRISPR-based gene editing candidate to Phase 3. And lastly, Matt and John called out the FDA’s finalized guidance document, Human Gene Therapy Products Incorporating Human Genome Editing, as being really helpful for companies focused on these therapeutics. For those interested in learning more about the guidance, the FDA is hosting a free public webinar on February 29, 2024.

The event with Tome Biosciences is an excellent example of the programs ISPE aims to organize for our local scientific community. Please stay tuned to hear more about the next EPC event on May 16, 2024 where the focus will be on Artificial Intelligence.