The latest Lentiglobin data impress, but launch worries could clip Bluebird’s wings.
Celgene’s deal-making skills have come under the microscope of late, but the group finally seems to have struck a good bargain with Acceleron and luspatercept.
The private UK group could steal the thunder of rivals like Spark, Uniqure and Biomarin.
In the seven months since its founding the company has attracted $793m of financing. Now it must execute.
Padua gambit seems to have worked as new gene therapy looks the equal of Pfizer/Spark Therapeutics.
The company struggles to move the needle at the SITC meeting with an up-to-date cut of its first-line melanoma trial of NKTR-214 and Opdivo.
Novartis's $4m price target for AVXS-101 fuels concerns that too many new gene therapies could overburden payers without a hard look at how they are financed.
Takeda will soon report phase III results with its dengue fever vaccine, while Regenxbio needs an improvement with its cholesterol-lowering gene therapy candidate,…
Krystal Biotech shares have surged 53% so far this week on gene therapy data in two patients, but rivals are waiting in the wings.