Gene Therapy

Thermo Fisher Launches Medium to Support Cell, Gene Therapy

by Samantha McGrail

Thermo Fisher Scientific recently launched a GMP-manufactured cell culture medium that supports the large-scale growth and culture of functional natural killer (NK) cells for cell and gene...

bluebird bio Submits BLA for Gene Therapy to Treat β-thalassemia

by Samantha McGrail

bluebird bio recently announced the completion of its rolling submission of its Biologics License Application to FDA for betibeglogene autotemcel (beti-cel) gene therapy in patients with...

Novartis Acquires Gene Therapy Company to Expand Portfolio

by Samantha McGrail

Novartis recently acquired Arctos Medical to add a pre-clinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology to its ophthalmology portfolio. Optogenetics has...

Thermo Fisher Launches System to Scale Gene Therapy Workflows

by Samantha McGrail

Thermo Fisher Scientific recently launched the integrated Gibco AAV-MAX Helper Free AAV Production System to enable scalable gene therapy workflows. The solution aims to simplify AAV vector production...

FDA Clears IND for Neurogene’s Gene Therapy for Batten Disease

by Samantha McGrail

FDA recently cleared Neurogene’s Investigational New Drug (IND) application for its CLN5 Batten disease gene therapy, NGN-101.  NGN-101 is an adeno-associated virus (AAV) gene therapy and...

FDA Halts BioMarin’s Trial Assessing Gene Therapy in Rare Disease

by Samantha McGrail

FDA recently placed a clinical hold on BioMarin Pharmaceuticals Phase 1/2 trial evaluating its AAV5-phenylalanine hydroxylase (PAH) gene therapy in adults with the rare disease, phenylketonuria...

NeuExcell, Spark Therapeutics to Develop Gene Therapy for HD

by Samantha McGrail

NeuExcell Therapeutics and Spark Therapeutics recently announced a gene therapy collaboration to develop a safe and effective treatment for patients with Huntington’s Disease (HD).  Under...