Table 3.

Gene based therapies: List of Food and Drug Administration approved therapies and investigational therapies showing promise

Therapy or drug	Indication	Mechanism of action	Approval status
Janssen COVID-19 vaccine	Prevention of 2019 coronavirus disease (COVID-19) for individuals 18 yr of age and older	Recombinant, humanadenovirus type 26 vector which expresses the SARS-CoV-2 “S” antigen after entering human cells thus eliciting immune response against COVID-19	Emergency use authorization (EUA) on February 27, 2021[70]. Pause placed on vaccine use on April 13, 2021[71]. FDA lifted vaccination pause on April 23, 2021[72]
Pfizer-BioNTech COVID-19 Vaccine[73-75]	Prevention of COVID-19 for individuals 16 yr of age and older	modRNA forumated in lipid particles when delivered to host cells express SARS-CoV-2 “S” antigen, thus eliciting immune response against COVID-19	EUA on December 11, 2020
Moderna COVID-19 vaccine[76-78]	Prevention of COVID-19 for individuals 18 yr of age and older	modRNA forumated in lipid particles when delivered to host cells express SARS-CoV-2 “S” antigen, thus eliciting immune response against COVID-19	EUA on December 18, 2020
Lumasiran[79]	Primary hyperoxaluria type 1	HAO1-directed small interfering ribonucleic acid	Approved in Nov 2020
Viltolarsen[80]	Duchenne muscular dystrophy	Antisense oligonucleotide directed to exon 53 skipping	Approved in August 2020
Brexucabtagene autoleucel[81]	Relapsed/refractory mantle cell lymphoma	Genetically modified autologous CD19 T cells directed against CD19 expressing cancer cells	Approved in July 2020
Golodirsen[82]	Duchenne muscular dystrophy	Antisense oligonucleotide directed	Approved in December 2019
Givosiran[83]	Acute hepatic porphyria	Double-stranded small interfering RNA that degrades the ALAS1 mRNA in hepatocytes via RNA interference	Approved in November 2019
Onasemnogene abeparvovec-xioi[84]	Spinal muscular atrophy (SMA)	AAV9-based gene therapy which encodes the human SMN protein	Approved in May 2019
Inotersen[85]	Polyneuropathy of hereditary transthyretin-mediated amyloidosis	Transthyretin-directed antisense oligonucleotide	Approved in October 2018
Axicabtagene ciloleucel[86]	Relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy	Genetically modified autologous CD19 T cells directed against CD19 expressing cancer cells	Approved in October 2017
Tisagenlecleucel[87]	Refractory or relapsed B-cell precursor acute lymphoblastic leukemia (ALL)	Genetically modified autologous CD19 T cells directed against CD19 expressing cancer cells	Approved in August 2017
Nusinersen[88]	SMA	Survival motor neuron-2 (SMN2)-directed antisense oligonucleotide	Approved in December 2016
Eteplirsen[89]	Duchenne muscular dystrophy	Antisense oligonucleotid that binds to exon 51 of dystrophin pre-mRNA	Approved in September 2016
Talimogene laherparepvec[90]	Genetically modified herpes simplex virus, type 1 used as oncolytic viral therapy	They utilized the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma who had the recurrence after the initial surgery	Approved in October 2015
Giroctocogene fitelparvovec[91]	Moderately severe to severe hemophilia A	Factor VIII gene delivery using recombinant adeno-associated viruses as vectors	Investigational in phase 3 trial
Inclisiran[92]	Heterozygous and possibly homozygous familial hypercholesterolemia	Small-interfering ribonucleic acid which decreases hepatic production of PCSK9	Investigational phase 3 trial
Volanesorsen[93]	Familial chylomicronemia syndrome	Antisense oligonucleotide that targets the messenger RNA for apo-CIII	Conditional approval by European Medicines Agency’s (EMA) but not by FDA
CRISPR-Cas9 gene editing[94]	Sickle cell disease and β-thalassemia	CRISPR-Cas9based allele editing of the BCL11A erythroid-specific enhancer in autologous CD34+ cells	Investigational- FDA Fast Track Designation for CTX001 in sickle cell disease

AAV: Adeno-associated virus; ALAS1: Aminolevulinate synthase 1; BCL11A: B cell lymphoma/leukemia 11A; HAO1: Hydroxyacid oxidase (glycolate oxidase) 1; modRNA: Nucleoside-modified messenger RNA; SMN: Survival motor neuron 1; FDA: Food and Drug Administration.