How it works
Fresh genes today
It sounds tempting, but has often failed: combatting harmful gene mutations by providing fresh genes. But this kind of gene therapy has meanwhile become a reality. Swiss spin-offs and pharmaceutical companies are at the forefront. We offer an overview.
It’s a longstanding dream: replacing faulty genes with healthy variants. To achieve this, you have to insert a new gene into the genetic material of a cell. This is usually made possible by means of a virus whose genes are replaced, and which can then perform its allotted task precisely. Three forms of this therapy have already been developed:
Illustration: ikonaut
C – Nerve cells become photoreceptors
In cases of macular degeneration or retinitis pigmentosa, photoreceptors in the retina of the eye also lose their function. An institute at the University of Basel has now been able to make other cells in the retina light-sensitive by using new genes, thereby restoring some vision to a patient. Arctos Medical, a University of Bern spin-off, has developed a similar method and has now been acquired by Novartis.
B – Repairing neurodegeneration
When nerve cells responsible for muscle function mutate and lose control, muscular atrophy and often early death are the consequence. The EPFL spin-off Avrion Therapeutics and Novartis have now each developed therapies for amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy that can compensate for the defective genes. The first is still being tested; the second, Zolgensma, is already on the market. Roche has a similar therapy called Luxturna to correct defective neurons in the retina of the eye in Leber congenital amaurosis (LCA).
A – Using immune cells to fight cancer
When therapy options run out in cases of leukaemia, all hope isn’t lost: patients’ own immune cells (T-cells) can be removed from their bodies and provided with an artificial receptor (CAR) that recognises the cancer cells. The CAR-T-cells are re-injected into patients, where they destroy the cancer cells. Novartis and Janssen-Cilag have now brought such therapies onto the market: Kymriah and Carvykti.
