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Gene Therapy

Novel Nano System Delivers mRNA to Inflammatory Leukocytes

An artist's rendering incorporates the images of the Müller glia-derived rod photoreceptors. These photoreceptors were structurally no different from real photoreceptors and they became integrated within the circuitry of the visual pathway
Gene Therapy

Blind Mice See after Gene Factors Stimulate Genesis of Rod Photoreceptors

Gene Therapy

Genome Sequencing Identifies Cause of Early Infantile Epileptic Encephalopathy

Astellas Pharma sees potential in Quethera's gene therapy program
Gene Therapy

Astellas Expands Its Eye Disorder Pipeline with Quethera Purchase

Sarepta Therapeutics will pay more than $38 million for rights to up to three Lacerta Therapeutics preclinical CNS-targeting gene therapy candidates
Gene Therapy

Sarepta Licenses Up to Three CNS-Targeting Gene Therapies from Lacerta

Spark Therapeutics saw its share price tumble about 30% in early-morning trading after it reported mixed preliminary data this morning from a Phase I/II trial of its hemophilia A gene therapy candidate SPK-8001. [Source: wildpixel/Getty Images]
Gene Therapy

Spark Therapeutics Stock Tumbles after Mixed Results from Phase I/II Trial...

Regeneron Pharmaceuticals demonstrated its VelocImmune technology in a study published in 2015 in Proceedings of the National Academy of Sciences showing the rapid generation of fully human neutralizing antibodies against Middle East Respiratory Syndrome coronavirus (MERS-CoV) and development of a humanized mouse model for MERS-CoV infection (pictured). Regeneron will use VelocImmune and other VelociSuite platform technologies in a collaboration with bluebird bio to develop new immune cell therapies for cancer. [Source: PNAS]
Gene Therapy

Regeneron to Invest $100M in bluebird bio as Companies Partner in...

Pulmonary ionocytes (orange) extend through neighboring epithelial cells in the upper respiratory tract of the mouse
Gene Therapy

Rare Lung Cell Type Appears to Play Important Role in Cystic...

A month after releasing preliminary positive results from a Phase I/IIa trial of its Duchenne Muscular Dystrophy (DMD) gene therapy candidate AAVrh74.MHCK7.micro-Dystrophin (pictured)
Gene Therapy

FDA Places Clinical Hold on Sarepta DMD Micro-Dystrophin Gene Therapy Trial

A cross section of a mouse spinal cord
Gene Therapy

Paralyzed Mice Walk Following Drug Therapy

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