The biotech venture capital firm Atlas Ventures has announced the creation of Generation Bio, a company that has developed a system by which the dosage of a gene therapy can be varied to maximise the potential benefit. The company has started on the back of a $25 million Series A funding round from Atlas. The cornerstone of Generation Bio’s offering is their GeneWave technology, which delivers high levels of the genetic material to target cells and can be dosed at different levels depending on the individual needs of the patient. (January 4, 2018)
This novel technology is very different from current gene therapy techniques. It does not use a viral vector to deliver the DNA to the nucleus, and it also allows for patients to receive different dosage levels depending on their need.
One issue with current gene therapies is that there is no way of altering the dose depending on how the patient progresses after the initial treatment. Often patients are given the highest possible dose, but this can lead to strong immune responses. As with all drugs, all patients react differently, and it could therefore be useful to be able to modulate the dosage in order to maximise the benefit of gene therapy for more patients.
Another benefit of this is the potential reduced cost of manufacturing the therapy. Manufacturing viral vectors for gene therapy is extremely expensive, and this has been an issue for many companies in the field of gene therapy. By not using a viral vector, Generation Bio might be able to limit these costs and potentially pass on savings to patients.
The technology uses lipid nanoparticles that are preferentially taken up by the liver, which has led to Generation Bio currently focussing on treating various diseases of the liver. They are, however, looking to modify the system so that they could deliver the gene therapy to other locations in the body. Given the current state of development of the technology and the focus on becoming clinical within two years, it seems that this move to other areas of the body will not be realised for quite some time. This gives other companies an opportunity to examine how this technology could work before committing to developing a similar system for a different disease area.
There are many potential benefits to this type of technology, but it remains to be seen how other gene therapy players may follow suit and investigate similar approaches. All eyes in the pharmaceutical industry will now be on Generation Bio as they move towards clinical studies, and they answer whether this technology will be a success.