Rare Gene Therapy Side Effect: Toddler Develops Tumor Linked to Treatment
A 13-month-old boy, Adam, developed a golf-ball sized brain tumor after undergoing experimental gene therapy for a rare disease. His parents chose the...
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Capsida Biotherapeutics Unable to Determine Cause of Child’s Death in Gene Therapy Trial
Capsida Biotherapeutics remains uncertain about the cause of a child’s death in a gene therapy trial conducted last September. The investigation has s...
Five Years After Deadly Disaster, Rare Disease Community Secures New Treatment Hope
Joshua Jacob Gonzalez’s parents suctioned his saliva every five minutes to prevent choking. After a groundbreaking treatment, they noticed a life-chan...
Rare Amoeba Infection Kills 78-Year-Old Man After Six-Month Battle with Necrotic Lesions
A 78-year-old man developed severe necrotic lesions and ulcers over six months, baffling doctors. Yale physicians later identified the cause as Acanth...
Mother of Girl Who Received Bespoke Gene Therapy Launches New Biotech to Scale Treatments
Julia Vitarello, whose daughter Mila received a custom gene therapy eight years ago, is launching a new biotech to scale bespoke treatments. Her previ...
Pfizer and BridgeBio Secure Patent Win: Key Biotech Developments Today
Italian pharma giant Chiesi acquires KalVista Therapeutics for $1.9 billion, adding a rare-disease treatment to its portfolio. Meanwhile, major biotec...
Intellia's CRISPR Gene Therapy Lonvo-z Slashes Swelling Attacks in Rare Disease Trial, Paving Way for Approval
Intellia Therapeutics announced Monday that a single dose of its CRISPR-based gene editing treatment, lonvo-z, dramatically reduced swelling attacks i...