A gene therapy platform for treating diseases

To say that your therapy has the potential to be curative is huge. This is a particularly
exciting time for Gene Therapy, because the promise and the
potential, that has existed for 30 years in the field, is now starting to finally bare fruit. AAVs will allow us to
address a number of different diseases because of their
ability to deliver genes to a number of different
tissues inside the body. With real estate, it’s about
location, location, location. With Gene Therapy, it’s about delivery to a specific location
where you need to restore the function of that gene. Adeno-associated viruses or
AAVs or small microscopic virus particles, that can
deliver DNA into cells. They have a protective outer
shell on them which consists of viral proteins. Those proteins contain the
molecular addresses to target certain cell types and
tissues for delivery of your therapy to construct. There are some advantages for using AAV over other types of viruses. One is that they are not known
to cause any human disease, and that improves the safety profile. The second which is that they
can be directed to, in fact a variety of different organs in the body, which makes them more broadly amenable to gene therapy applications. Typically, AAV vectors are used to replace a defective gene that’s
found in a genetic disorder. The real promise of Gene Therapy is to actually correct,
at the genomic level, the cause of disease which could in fact
result in a life long cure without the need to take
pills everyday, to have painful injections everyday. Within Novartis, we are
currently developing local applications for AAV
vector medicines, primarily in neuroscience as well
as in ophthalmology. Novartis is building its
AAV capabilities by using a combination of internal
innovation as well as external partnerships. We’ve built teams internally, that are now making AAV vectors. We are also looking
externally by finding partners that may have been able
to innovate the biology outside of our walls, and
bringing that internally to be able to really drive
the innovation in this space. We’ve invested in the
infrastructure needed for research, for preclinical development,
for clinical development, and most importantly
manufacturing, in order to provide these innovative medicines to patients. This is a very exciting
time for Gene Therapy.


  1. It would be nice if all people who would benefit from this technology were wealthy. Sadly, genetic diseases affect all classes equally.

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