Biotech venture capital firm Atlas Ventures has launched a gene therapy company, Generation Bio, to develop gene therapies that do not rely on viral vectors for gene delivery. The technology behind the company is a result of co-founder Prof. Robert Kotin’s work in creating a lipid nanoparticle platform for the delivery of closed-ended DNA.
The company’s initial work will focus on diseases which affect the liver as the organ preferentially takes up the liquid nanoparticles that will be used to deliver genetic material to cells. This will migrate to the nucleus and form episomes which have high levels of expression without integrating with the host DNA. The approach could potentially bypass the issues of immune response associated with using viral vectors, as well as allowing for redosing and the variance of dose levels according to patients, something that is not possible with current gene therapy approaches.
The company has spent a year developing its GeneWave platform, and the capacity to manufacture it inhouse. It has also demonstrated the approach in animal models, showing the viability of dose dependent expression in the liver. The diseases that are being focused on currently are progressive familial intrahepatic cholestasis, phenylketonuria, glycogen storage disease 1a, and ornithine transcarbamylase deficiency.
President and CEO Geoffrey McDonough commented, ‘This is a breakthrough category of therapeutics which will continue to expand in the future. One aspect of our platform which is important to us is scale—by removing viral capsid from our manufacturing process we are increasing the scale and reducing the cost of our platform. Combined with the druglike properties of GeneWave, we aim to greatly expand the number and variety of diseases able to be addressed by gene therapy.’
Source: Atlas-backed Generation Bio promises ‘druglike’ gene therapy. Press Release