American regulators have approved a gene therapy that treats an ‘inherited form of vision loss’.
FDA Commissioner Scott Gottlieb said: “The approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases.”
He added: “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”
Luxturna helps patients with ‘biallelic RPE65 mutation-associated retinal dystrophy’, which the FDA said affects around 2000 people in the US, and can ultimately lead to blindness.
In tests – which a total of 44 people took part in, the impact of Luxturna was best demonstrated during an obstacle course experiment. Measuring participants ability ‘from baseline to one year’ in navigating the course at varying lights, those that took the therapy performed better than those who didn’t.
“This one-time gene therapy for an inherited disease represents a first-of-its-kind breakthrough that may lay the groundwork for the development of gene therapies for other conditions that are not adequately addressed today,” added Jeffrey Marrazzo, chief executive officer at Spark Therapeutics.
It has been a game-changing year for gene therapy. Innovators Magazine reported approvals by the FDA for therapies targeting cancer; one which is said to have ‘blockbuster potential’ – while the other could spell ‘the end for blood cancer’.
Check out this FDA video to learn more about gene therapy.