Daiichi Sankyo sinks $200M into new gene therapy tech from Ultragenyx
In a leap to the gene therapy space, Daiichi Sankyo has dropped $200 million to access Ultragenyx’s manufacturing technology, providing the rare disease biotech with plenty of cash and a stock boost amid a general cash crunch.
For $125 million in cash and a $75 million equity investment, Daiichi Sankyo has bought a non-exclusive license to the IP around two platforms with which it plans to develop AAV-based gene therapy products. The Japanese pharma is purchasing the stock $RARE at $60 per share, more than a third above its current price of $44.43.
Both designed to enable mammalian manufacturing, the HeLa producer cell line and HEK293 transient transfection platforms can be scalable up to 2,000L and 200-500L, respectively. In a presentation last Spring, Ultragenyx CEO Emil Kakkis noted that the former is suited for large indications while the latter has significant use in clinical trials and is viable for smaller patient populations.
“We are currently doing discovery research for gene therapy drugs using AAV vectors as one of our focused modalities toward sustained growth beyond achievement of our 2025 vision,” said Masayuki Yabuta, Daiichi Sankyo’s head of biologics. “In order to provide these drugs to patients in the future, manufacturing technology must be established early.”
Another $25 million will be due once tech transfer is complete. Daiichi is also footing all costs associated with the transfer.
“Though RARE and the DMTX team have likely been investing in these platforms the last 2+ yrs, the Daiichi deal affirms RARE’s 2017 purchase of DMTX (where the tech originated) for ~$152M as a smart move,” Jefferies’ Maury Raycroft wrote in a note. “Recall, this is actually the 2nd partnership around the platform — Bayer partnered w/ DMTX in 2014 for hemeA (now DTX201), which is using the HeLa system w/ initial clin results at ASH’19 and EAHAD’20.”