1️⃣ Hemgenix (etranacogene dezaparvovec): Gene therapy that introduces a functional gene into the body. This enables the production of functional factor IX (a protein necessary for normal blood clotting), enhancing the body’s natural clotting ability.
2️⃣ Skysona (elivaldogene autotemcel): Personalized gene therapy – uses the patient’s own blood stem cells and adds functional copies of the ATP Binding Cassette Subfamily D Member 1 gene to the cells. This enables the body to break down the very long chain fatty acids . Thus it slows down the progression of damage to the brain and decline in neurologic function.
3️⃣ Zynteglo (betibeglogene autotemcel): Gene therapy that can address the underlying genetic cause of beta-thalassemia by adding functional copies of a modified β-globin gene to the patient’s own hematopoietic stem cells (HSCs).
4️⃣ Roctavian (valoctocogene roxaparvovec): Gene therapy that uses an adeno-associated virus 5 that codes for human Factor VIII (lacking in people with hemophilia A), together with a human liver-specific promoter that encourages translation in hepatocytes, not liver endothelial and sinusoidal cells, where Factor VIII is ordinarily synthesized.
5️⃣ Zolgensma (onasemnogene abeparvovec): Gene therapy, which replaces the function of the survival motor neuron gene, by delivering a new, working SMN gene to the motor neuron cells throughout a child’s body to keep the muscles working as they should.
6️⃣ Zokinvy (lonafarnib): HGPS and processing-deficient Progeroid Laminopathies are genetic disorders. They are characterized by the accumulation of harmful proteins in the body, leading to premature ageing. lonafarnib (protein farnesyltransferase inhibitor FTI that reversibly binds to the farnesyltransferase CAAX binding site) blocks the building up of progerin and progerin-like proteins.
7️⃣ Danyelza (naxitamab): IgG1 monoclonal antibody directed against GD2 disialogangliosides. It binds to GD2 on the surface of neuroblastoma cells and induces both complement-dependent cytotoxicity and antibody-dependent cell-mediated cytotoxicity thus killing cancer cells.
8️⃣ Myalept (metreleptin): Synthetic analogue of the hormone leptin. Works as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy.
9️⃣ Luxturna (voretigene neparvovec): Uses a neutralised virus as a vector to carry a functional gene into the affected eye tissue, enabling the body to produce any proteins missing due to genetic mutation.
Brineura (cerliponase alfa): Slows the progressive decline in motor function caused by abnormal motor signalling in the brain by restoring the normal levels and activity of tripeptidyl peptidase TPP1 gene.