B-cell acute lymphoblastic leukemia (B-ALL) is a life-threatening and highly aggressive form of blood cancer. It is the most common childhood cancer, making up 35% of pediatric cancer cases, but it ...

Use of DYNE-101 is leading to improvements in muscle, and specifically finger, function in myotonic dystrophy type 1 patients ...

Tenaya Therapeutics (TNYA) announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for ...

UBS upgraded Cryoport (CYRX) to Buy from Neutral with a $10 price target UBS believes that scaling cell and gene therapy service offerings ...

It's early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions ...

Due to hemophilia A patients switching to newer treatments, Takeda Pharmaceuticals is discontinuing both Hemofil M and ...

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

For the first time, scientists say, they have evidence that using a biologic drug to remove sticky beta amyloid plaques from ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

Count Doug Kramer among the players who weren't really given a chance by the Matt Eberflus regime to show what they can do but he's back on a 1-year deal.