Gene Therapy Loses Luster as Investors Eye Quicker Returns From Weight-Loss Drugs By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell ...

Scientists have created a new gene therapy for a debilitating genetic disorder called maple syrup urine disease (MSUD). The treatment can prevent recurrence of deadly symptoms in a cow calf born ...

Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders. Now it’s on the cusp of its first regulatory approval. Each year ...

Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. Roche described the restructuring in ...

The University of Bristol research team used animal models to compare patient responses to AAV therapy, studying male and female eye cells across different age groups (young, middle-aged and older ...

Tomas has become the first in the world to be treated with a new gene therapy (Handout/PA) A baby boy with a rare illness is making “incredible” progress after he became the first ever patient ...

A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...

“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine. He added that the method ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting in morning trading.