. Author manuscript; available in PMC: 2024 Mar 1.

Published in final edited form as: Am J Med Genet C Semin Med Genet. 2023 Feb 4;193(1):30–43. doi: 10.1002/ajmg.c.32031

Table 1.

Platform modalities

Status	Approach	Shared element(s)	Variable element(s)	Example/Reference	Consortia
Established	CAR-T	Cell type	T cell receptor variable region	Multiple myeloma - Idecabtagene (Gearin, 2021)	CARnation Consortium
	Cancer vaccines	Peptide subunits and linkages	Peptide sequence	Cervical cancer - Human papillomavirus vaccine (Cheng, et al., 2020)	Translational Research Cancer Centers Consortium
	mRNA vaccines	RNA, modified nucleotide subunits	Sequence	COVID-19 vaccine (Thorn, et al., 2022)	K-mRNA vaccine consortium
Emerging	Viral vector-mediated gene replacement	Viral vector particles (DNA or RNA, proteins)	Sequence (promoter, coding sequence)	Spinal Muscular Atrophy - Onasemnogene (Blair, 2022)	PAVE-GT, BGTC
Emerging	Antisense oligonucleotides	Oligonucleotides, +/− lipid nanoparticles, +/− tissue-targeting conjugates	Sequence	Spinal Muscular Atrophy – Nusinersen (Hoy, S.M., 2017)	N=1 Collaborative, 1M1M
Future	mRNA gene replacement	RNA, modified nucleotide subunits	Sequence	Propionic acidemia (Jiang, et al., 2020)	N/A
	Morpholinos	DNA, modified nucleotide subunits	Sequence	Duchenne Muscular Dystrophy (Le, B.T., et al., 2022)	N/A
	siRNAs	RNA, modified nucleotide subunits	Sequence	Hereditary transthyretin-mediated amyloidosis - Patisiran (Adams, et al., 2018)	RNAi Consortium
	Genome editing	Vector, nuclease / base editor proteins	Guide RNA sequence, “donor” DNA	Amyloidosis (Gillmore, et al., 2021)	NIST Genome Editing Consortium

BGTC – Bespoke Gene Therapy Consortium; NIST - National Institute of Standards and Technology; PAVE-GT - Platform Vector Gene Therapy; 1M1M – 1 Mutation 1 Medicine