Gene Therapies
Mary Johnson (han at labome dot com)
Synatom Research, Princeton, New Jersey, United States
DOI
//dx.doi.org/10.13070/mm.en.14.3202
Date
last modified : 2025-05-30; original version : 2024-05-12
Cite as
MATER METHODS 2024;14:3202
Abstract

A select list of FDA-approved gene therapies.

2025
  • March 5, ENCELTO revakinagene taroretcel-lwey from Neurotech Pharmaceuticals. Indicated for the treatment of adults with idiopathic macular telangiectasia type 2. ENCELTO secretes recombinant human ciliary neurotrophic factor.
2024
  • November 13, KEBILIDI eladocagene exuparvovec-tneq from PTC Therapeutics. Indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency. KEBILIDI is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the dopa decarboxylase gene which encodes the AADC enzyme. I
  • April 25, BEQVEZ Fidanacogene elaparvovec-dzkt from Pfizer. Indicated for treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who: currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. BEQVEZ consists of a recombinant viral capsid (AAVRh74var) derived from a naturally occurring AAV serotype (Rh74) vector containing the human coagulation factor IX (FIX) transgene modified to a high-specific factor IX activity variant known as FIX-R338L.
2023
  • June 29, ROCTAVIAN valoctocogene roxaparvovec-rvox from BioMarin Pharmaceutical. Indicated for treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. ROCTAVIAN consists of an AAV serotype 5 capsid containing a DNA sequence encoding the B-domain deleted SQ form of the human coagulation factor VIII.
  • June 22, ELEVIDYS delandistrogene moxeparvovec-rokl from Sarepta Therapeutics. Indicated for treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. ELEVIDYS is an adeno-associated virus serotype rh74 (AAVrh74) based vector containing the ELEVIDYS micro- dystrophin transgene under the control of the MHCK7 promoter.
  • May 19, VYJUVEK beremagene geperpavec from Krystal Biotech. Indicated for treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. VYJUVEK is a live, replication defective herpes simplex virus 1-based vector containing a transgene for human type VII collagen protein.
2022
  • December 16, ADSTILADRIN nadofaragene firadenovec-vncg from Ferring Pharmaceuticals. Indicated for treatment of adult patients with high-risk Bacillus Calmette-Guerin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors. It is a recombinant adenovirus serotype 5 vector containing a transgene encoding human interferon alpha-2b.
  • November 22, HEMGENIX etranacogene dezaparvovec-drlb from CSL Behring. Indicated for treatment of adults with hemophilia B (congenital Factor IX deficiency) who: currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. HEMGENIX is a non-replicating recombinant AAV5 containing a codon-optimized DNA sequence of the gain-of-function Padua variant of human factor IX (variant R338L), under the control of a liver-specific promotor 1.
2019
  • May 24, ZOLGENSMA onasemnogene abeparvovec-xioi from Novartis. Indicated for treatment of patients less than 2 years of age with spinal muscular atrophy with biallelic mutations in the survival motor neuron 1 gene. ZOLGENSMA contains a recombinant self-complementary adeno-associated virus serotype 9 with a transgene encoding the human survival motor neuron (SMN) protein, under the control of a cytomegalovirus enhancer/chicken-beta-actin hybrid promoter.
2017
  • December 19, LUXTURNA voretigene neparvovec-rzyl from Spark Therapeutics / Roche. Indicated for treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 genetically modified to express the human RPE65 gene.
2015
  • October 27, IMLYGIC talimogene laherparepvec from BioVex / Amgen. Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery. IMLYGIC is a live, attenuated herpes simplex virus type 1 that has been genetically modified to express human granulocyte-macrophage colony-stimulating factor.
ISSN : 2329-5139