The Daily Pennsylvanian

Penn Medicine, CHOP to begin testing specialized gene therapy after working on new FDA protocol

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.

FDA tightens Duchenne gene therapy after deaths of two teens

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...
STAT

FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk

Risk of “acute, serious, and life-threatening” heart inflammation is now on the label of Sarepta's gene therapy for Duchenne ...

Cellectis Publishes Nature Communications Article on a Non-Viral Gene Editing Process Enabling Efficient Gene Insertion in Hematopoietic Stem Cells

Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its ...

FDA adds strongest warning to Sarepta gene therapy linked to 2 patient deaths

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year.
Hoodline

Sharonville Shines as New $60M Cincinnati Children's Gene Therapy Center Promises Jobs and Revolutionary Treatments

Sharonville, Ohio, celebrates the opening of the new Cincinnati Children's Applied Gene and Cell Therapy Center.
Fred Hutchinson Cancer Center

Scientists see foam as starting point of a path to bedside gene therapy

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...
Fierce Biotech

Lilly bags eye-catching retinal disease gene therapy from MeiraGTx in $475M deal

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...
Pharmaceutical Technology

USPTO grants patent to Genprex’s Reqorsa gene therapy

The US Patent and Trademark Office (USPTO) has granted a patent to Genprex for the use of its Reqorsa gene therapy ...
BioSpace

FDA Imposes Boxed Warning, Narrows Patient Pool for Sarepta’s Duchenne Gene Therapy Elevidys

Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...

Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

Gene therapy for sickle cell and β-thalassemia works by disrupting three-dimensional genome structure

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...