PA Consulting’s Paul Stroemer, cell and gene therapy expert, comments on the development and use of innovative gene therapies to treat rare diseases in an article by Joy Persaud in Raconteur's Future of Healthcare report.
Commenting on the use of gene therapy for Retinitis Pigmentosa, Paul explains: “Retinal disorders are good candidates for gene therapy,” adding “Loss of tissue in the eye is gradual, giving a wide treatment window. Nerve cells in the eye don’t divide, meaning that there is no requirement for administering further treatments. The therapy is permanent – one and done.”
Paul continues, saying that one eye is treated at a time, which means the un-treated eye will note experience any adverse reactions.
Commenting on the use of gene therapy for Spinal Muscular Atrophy (SMA), Paul explains that: “Patients with this disease lack the survival motor neuron protein, which is essential for the normal functioning of motor neurons. Without it, these nerve cells deteriorate and eventually die,” adding that “Diagnosis can be made via gene screening, which can be done at birth.”
Explaining the procedure, Paul says: “When injected, it passes into the nerves and provides the correct gene to make enough of the protein and thereby restore nerve function.”
He adds: “Given that there were practically no other viable permanent treatment options available before, the significance of Zolgensma’s introduction as a treatment for SMA should not be underestimated.”