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Partnering across the globe to advance gene therapy

Ultragenyx and Daiichi Sankyo use tech innovations to overcome pandemic travel challenges

DNA base sequence in autoradiogram

Ultragenyx announced in 2020 a partnership with leading Japanese pharmaceutical company Daiichi Sankyo, who would in-license Ultragenyx gene therapy technology. The agreement included a technology transfer that would enable Daiichi Sankyo to use Ultragenyx’s clonal producer cell line (PCL) and bioprocessing technologies to produce Daiichi Sankyo’s own adeno-associated virus (AAV) gene therapy programs. The team crafted this partnership with plenty of in-person, hands-on learning in mind for both teams in the lab. However, when the COVID-19 pandemic hit and travel was prohibited, the teams had to find creative alternatives to complete the transfer of these highly complex technologies.

Further complicating matters, Ultragenyx Gene Therapy team members working in Massachusetts are a full 12 time zones away from their Daiichi Sankyo counterparts in Japan. These gene therapy processes were challenging enough to demonstrate in person, and now the teams needed a way to collaborate in real-time across time zones, oceans, and languages.

“Overcoming the logistical and technical challenges presented by the pandemic required a novel approach,” said David Maheu, senior director of pilot plant, technology transfer, and business operations at Ultragenyx. “Thankfully, our counterparts at Daiichi Sankyo shared our determination to completing this technology transfer and were open to utilizing innovative means to make it happen.”

The two teams landed on a creative approach to transferring these gene therapy processes in an entirely virtual setting – they paired recorded, translated video demonstrations with real-time, two-way collaboration facilitated through wearable technology. Ultragenyx Gene Therapy scientists and engineers ultimately developed more than 80 discrete videos to demonstrate in detail each of the critical steps for these cellular and bioanalytical processes. In addition to these videos, the teams in the labs made use of wearable technology made by RealWear® that allowed them to be “in the room” via live streaming across sites.

Daiichi Lady with Aviators

The hands-free, voice-activated wearable headsets have integrated cameras and microphones, allowing the Ultragenyx team to simulate working in-person with the Daiichi Sankyo scientists and engineers. During these interactive live streams, Ultragenyx scientists could see the processes from the Daiichi Sankyo team member’s point of view and offer guidance and troubleshooting in real time. While this technology was critical to virtual collaboration, the dedication shown by both teams was extraordinary. Massachusetts-based Ultragenyx team members frequently worked their typical Eastern Time hours and then stayed late into the night to collaborate with their Daiichi Sankyo counterparts when the workday began in Japan.

Overcoming challenges is part of Ultragenyx’s core values of being relentless, dynamic, and seeing the possibility.

In November 2021, the initial milestones of the technology transfer were successfully completed – ahead of schedule. “Overcoming challenges is part of Ultragenyx’s core values of being relentless, dynamic, and seeing the possibility,” said Dennis Huang, executive vice president and chief technical operations officer at Ultragenyx. “Despite restrictions on travel, our teams found creative solutions to collaborate in real-time from different continents and time zones, and our achievement of the transfer is a testament to the remarkable commitment and engagement by teams at both Daiichi Sankyo and Ultragenyx.”

Jeff Blake is senior director of corporate communications at Ultragenyx.