In recent years, tools that enable deeper analysis of single cells have made incredible progress. We can now look at individual cells from a single sample and see how they differ from one another in terms of their DNA, gene expression and protein production. With these tools, each cell tells its own story about where […]
Tag Archives: Science
Why a fulfilling career in science requires more than just brainpower
Fun fact: I live in a jungle. Well, not exactly – but my 450 square foot condo is home to 55 indoor plants, which is as close to a jungle as you can get in the city. My fascination with plants extends far beyond the walls of my home – it fostered within me a […]
Treating the whole body: The case for lentiviral gene therapy’s head-to-toe reach
We hear it regularly from patients and caregivers: There’s an urgent need for therapies that can treat the whole body to address the full range of symptoms in diseases that manifest in both the central nervous system (CNS) and peripheral tissues. Biologically, that’s a tall order. The brain is protected by an intricate blood-brain barrier […]
Our second wave: How tag technology is driving exciting preclinical results in Pompe disease research
At AVROBIO, we’ve been highly strategic in selecting our second wave of programs for our leading lentiviral gene therapy pipeline. We’re targeting three devastating lysosomal disorders in this next wave – Pompe disease, Hunter syndrome and Gaucher disease type 3 – each of which impacts multiple organ systems throughout the body and is marked by […]
Leading the way in bringing gene therapy to the CNS with conditioning: A renaissance for busulfan?
Effective treatment of the central nervous system (CNS) through gene therapy is a promising approach for the many genetic diseases with neurological manifestations. It’s a particular hurdle for diseases with both CNS and systemic involvement, where an effective therapy would need to work on both sides of the blood–brain barrier. Prominent among this group are […]
Removing barriers to achieve widespread clinical adoption of lentiviral gene therapy
Lentiviral gene therapy, involving the genetic modification of stem cells outside of the body, requires a conditioning regimen in order to create the necessary space for engraftment of gene-modified cells in the bone marrow. Busulfan is an established conditioning agent, building on decades of use in bone marrow transplants as well as the treatment of […]
We saw it coming in pharma: Now gene therapy is biotech 2.0 for rare diseases
It seems so clear now: gene therapy is on a roll. As 2017 came to a close, the first gene therapy was approved by FDA and more than 2,500 gene therapy clinical trials are ongoing, including over 120 trials in pivotal Phase 2/3 or Phase 3 studies (here). Remarkably, gene therapy is now the second […]
