Table 5.

Types of claims in practice.

Type of Claim	Drug Name	Latest Stage of Development	Patent Application	Claims
Functional definition	Casimersen	Marketed	WO2006000057 [62]	1. An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
	Patisiran	Marketed	WO2010048228 [63]	1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of transthyretin (TTR), wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region complementary to a part of an mRNA encoding transthyretin (TTR), wherein said region of complementarity is less than 30 nucleotides in length and the antisense strand comprises 15 or more contiguous nucleotides of SEQ ID NO:170, SEQ ID NO:450, SEQ ID NO:730, or SEQ ID NO:1010.
Structural definition	Inotersen	Marketed	WO2014179627 [64]	1. A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide consists of eight to 80 linked nucleosides and has a nucleobase sequence at least 85%, 90%, 95%, or 100% complementary to SEQ ID NO: 2 encoding transthyretin (TTR).
Scaffold claim	Vutrisiran	Pre-registration	WO2013075035 [65]	1. A double-stranded RNAi agent comprising a sense strand complementary to an antisense strand, wherein said antisense strand comprises a region complementary to part of an mRNA encoding transthyretin (TTR), wherein each strand has about 14 to about 30 nucleotides, wherein said double-stranded RNAi agent is represented by formula (III): sense: 5′ np -Na -(X X X);-Nb -Y Y Y -Nb -(Z Z Z)j -Na-nq 3′ antisense: 3′ np’-Na’-(X’X’X’)k-Nb’-Y’Y’Y’-Nb’-(Z’Z’Z’)i-Na’- nq’ 5′ (III) wherein i, j, k, and 1 are each independently 0 or 1; p, p’, q, and q’ are each independently 0–6; each Na and Na’ independently represents an oligonucleotide sequence comprising 0–25 nucleotides that are either modified or unmodified or combinations thereof, each sequence comprising at least two differently modified nucleotides; each Nb and Nb’ independently represents an oligonucleotide sequence comprising 0–10 nucleotides that are either modified or unmodified or combinations thereof; each np, np’, nq, and nq’ independently represents an overhang nucleotide; XXX, YYY, ZZZ, Χ’Χ’Χ’, ΥΎΎ’, and Z’Z’Z’ each independently represent one motif of three identical modifications on three consecutive nucleotides; modifications on Nb differ from the modification on Y and modifications on Nb’ differ from the modification on Y’; and wherein the sense strand is conjugated to at least one ligand.