Fierce Biotech10d
Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
The Scientist1d
Correcting the Mutation Behind a Genetic Eye Disease
Base editing corrected a mutation that causes macular degeneration, highlighting the potential of gene therapy to treat ...
LUXTURNA Continues To Lead In Retinal Gene Therapy With Increased Market Penetration Delveinsight
LUXTURNA has significant market potential due to the high unmet need for effective treatments in rare genetic conditions. With its FDA approv ...
Technology Networks8d
Gene Therapy Offers Hope for Dravet Syndrome Treatment
Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...
Evaluating DNA impurities in recombinant adeno-associated virus
A new study in the journal Human Gene Therapy indicates that DNA impurities derived from plasmid and host cell DNA are ...
EurekAlert!9d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Fierce Pharma9d
Sarepta reports one death after patient received DMD gene therapy Elevidys
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...
OXB honoured at 2025 CDMO Leadership Awards in 'Cell & Gene Therapy – Global' category
OXB honoured at 2025 CDMO Leadership Awards in 'Cell & Gene Therapy – Global' category Oxford, UK – 21 March 2025: OXB (LSE: ...