The whole haemophilia gene therapy field will be hoping that a case of liver cancer in etranacogene dezaparvovec’s pivotal trial is not treatment related.
Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
Early data show that Freeline’s project can get factor IX levels into the normal range, but the group is still looking for the “Goldilocks” zone.
And bleeds seen with the “transformative” project were played down.
Waning factor VIII levels with giroctocogene fitelparvovec once again raise the spectre of Biomarin’s valrox.
Crispr Therapeutics might have stolen the Ash headlines, but a similar approach from Bluebird is also making progress.