Published on Independent UK / LinkedIn via Spencer Knight A three-year-old girl with a rare disorder called AADC deficiency has become...

Published on CAR T Vision (via CAR T Vision Coalition website) A new international coalition, CAR T Vision, has launched with a bold...

Published on The Associated Press Sarepta has suspended shipments of Elevidys and paused a trial arm after a second non-ambulatory...

Published on Spencer Knight via LinkedIn Eli Lilly has announced plans to acquire Verve Therapeutics in a deal worth up to $1.3 billion....

Published on Spencer Knight via LinkedIn Supernus Pharmaceuticals is acquiring Sage Therapeutics in a $795 million all-cash deal....

Published on Spencer Knight via LinkedIn BioNTech has signed a definitive agreement to acquire CureVac in an all-stock transaction valued...

Published on Spencer Knight via LinkedIn SpliceBio has secured €119 million in Series B funding, the largest ever for a biotech in...

Published on Spencer Knight via LinkedIn Missy Christy had already beaten cancer once. Then, as a new mother, she faced it again—this...

Published on Spencer Knight via LinkedIn Glioblastoma remains one of the deadliest brain cancers with limited treatment options. Two new...

Published on Spencer Knight via LinkedIn Sanofi has announced a definitive agreement to acquire Blueprint Medicines in a $9.1 billion...

Published on Spencer Knight via LinkedIn Lani Watson, 32, lived with severe lupus that left her in constant pain and unable to walk. In a...

Published on Spencer Knight via LinkedIn Sanofi has agreed to acquire Vigil Neuroscience in a $470 million deal, reinforcing its...

Published on Spencer Knight via LinkedIn Elliott Collins, 34, lived with haemophilia B for nearly three decades, relying on frequent...

Published on Spencer Knight via LinkedIn siRNA therapies are seeing renewed momentum, with major investments and regulatory wins...

Published on Spencer Knight via LinkedIn At just nine months old, KJ was diagnosed with CPS1 deficiency, a rare metabolic disorder...

Published on Spencer Knight via LinkedIn A packed week at ASGCT brought a wave of new CGT developments totaling over $240 million in...

Published on Spencer Knight via LinkedIn GSK is acquiring efruxifermin alfa from Boston Pharmaceuticals for up to $2 billion, including...

Published on Spencer Knight via LinkedIn HAON Life Sciences, a Dublin-based biotech startup, has received €10.7 million from Ireland’s...

Published on Spencer Knight via LinkedIn Emma Dimey was diagnosed with stage 4 colorectal cancer at just 23. After 11 years of failed...

Published on Spencer Knight via LinkedIn Eisa Hussain was diagnosed as an infant with leukocyte adhesion deficiency type 1 (LAD-1), a...