Uniqure’s safety scare shakes haemophilia gene therapy
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.
Key upcoming clinical catalysts for biotech
Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
Finding the next Prevail
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
Freeline takes the haemophilia B gene therapy fight to Uniqure
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.
Ash 2020 – hints of durability emerge for Uniqure's gene therapy
And bleeds seen with the “transformative” project were played down.
Ash 2020 – durability worries hit Sangamo and Pfizer’s haemophilia A gene therapy
Waning factor VIII levels with giroctocogene fitelparvovec once again raise the spectre of Biomarin’s valrox.
Bluebird’s early-stage sickle cell gene therapy shows promise
Crispr Therapeutics might have stolen the Ash headlines, but a similar approach from Bluebird is also making progress.
