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 ...
MyChesCo on MSN21h
ChromaTan and Landmark Bio Receive NIIMBL Grant to Advance AAV Gene Therapy Manufacturing
ChromaTan Inc. and Landmark Bio PBLLC have been awarded a grant from the National Institute for Innovation in Manufacturing ...
Technology Networks14h
Gene Therapy Offers Hope for Dravet Syndrome Treatment
Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...
Fierce Biotech1d
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 ...
EurekAlert!1d
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 ...
Preclinical study demonstrates improved gene therapy approach to treating a rare and devastating disease in children
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Fierce Pharma1d
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 ...
BioSpace1d
Arbor Raises $73.5M To Ride Out Gene Editing’s ‘Gartner Hype Cycle’
The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.
EurekAlert!7d
IL2RG gene therapy for X-SCID
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
The American Journal of Managed Care1d
Data Gaps Leave Long-Term Impact of Ex Vivo Gene Therapy in DMD Uncertain
Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.
Fierce Pharma9d
Roche overhauls Spark gene therapy unit, recording $2.4B in full impairment
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...