BioWorld

AAV-delivered PPT1 gene therapy shows promise for CLN1 disease

Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
BioSpace

Sarepta’s Not Done With Gene Therapy Yet as LGMD Asset Yields Promising Data

Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
PharmTech

The Push for Momentum, Not Mistakes in Orphan Drug Development

This article outlines common CMC problems that are obstacles that steal momentum and create costly detours in the development ...
BioSpace

FDA’s Second Platform Designation Goes to Krystal After Sarepta Withdrawal

After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major ...
Rowan Today

Virtua Health College researchers achieve breakthrough with first gene therapy targeting white matter in fatal childhood brain disorder

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

Fruit fly study reveals genetic targets for rare childhood brain cancer treatment

Using fruit flies, University of Wisconsin–Madison researchers have developed a new model for investigating the genetic ...