Addressing the Top Five Challenges to AAV-Based Gene Therapy with ddPCR

Bio-Rad Laboratories describes how Droplet Digital PCR’s precision and sensitivity can help AAV developers overcome purification challenges and ensure product safety and efficacy.
Catalent's manufacturing facility in Harmans, MD

The Next Generation of Cell Factories for Viral Vector Production

Although the industry still relies on transient transfection, due to the difficulty of creating stable production cell lines, a shift from adherent to suspension culture is underway.
Lonza lab

Gene Therapy Adopts New Tools to Guarantee Quality

Manufacturers are developing new analytical tools to satisfy increasingly stringent regulatory requirements.

Gene Therapies Receive a Shot in the Arm

Developers of gene therapies are taking advantage of new vector technologies, incorporating CRISPR systems, and augmenting gene therapy with targeted therapies and immunotherapies.

3D-Bioprinted Cell Therapy and Disease Modeling Applications

Rather than inject loose collections of therapeutic cells, implant 3D-bioprinted tissue constructs and gain more control over cell-based precision medicine applications.

New Tools are Democratizing the Life Sciences and Enabling Entrepreneurial Biology

Biology’s version of the desktop computing revolution is currently unfolding [with the introduction of] cutting-edge tools. However, biological innovations are much more complicated than working with silicon, requiring more testing and safety precautions. There is a logistical challenge with which biology must contend before it can become a more nimble, high-growth industry.

NIH Panel Advocates Harsh Price for Cline

In one of the first issues of GEN, we published an article about the so-called Cline affair. Martin J. Cline, MD, a professor of medicine at UCLA, achieved notoriety in 1980 when he performed the first gene therapy experiments on human subjects. Cline said the experiments were carried out in Italy and Israel while a request to perform similar experiments at UCLA was still being considered. UCLA eventually denied Cline permission and, amid the controversy surrounding the experiments, forced him to resign his chairmanship at UCLA Medical School. He also lost several research grants. The results of the experiments were reportedly inconclusive.

Gene Therapy Shows Potential for Repairing Damage Caused by Glaucoma and Dementia

Studies in rodents by a University of Cambridge-led team demonstrated how delivering two genes simultaneously using a single vector stimulated axonal transport in a humanized tauopathy model of Alzheimer’s disease, and in an experimental glaucoma model. Encouragingly, the results also showed “promising evidence” that the gene therapy might result in improvement in short-term memory in the tauopathy model of dementia. The team said the studies highlight the potential effectiveness of gene therapy for treating complex polygenic conditions that have no single genetic cause.

Doudna’s Scribe Rewrites CRISPR Drug Development with $100 Million Series B

Scribe—one of a new wave of gene editing biotech companies—focuses on developing CRISPR-based treatments through its genetic modification platform, designed to build and apply its suite of CRISPR technologies designed for therapeutic use.
Criminologist studying DNA

Manufacturing Innovation Key for Gene Therapy Sector Growth

Gene therapy developers struggle to manufacture trial materials, according to U.K. group behind network of innovation centers intended to help researchers move therapies into the clinic.
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