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4 RNA Therapeutics Stocks Leading the Next Wave of Innovation
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Key Takeaways
Alnylam's RNAi portfolio generated $2.21 billion in first-half 2026 product revenues, up 94%.
Ionis reported 185% growth in first-half product sales as Tryngolza and Dawnzera gained traction.
Wave and Arcturus advance clinical RNA programs in obesity, OTC deficiency and other diseases.
RNA therapeutics are medicines that use RNA molecules to influence gene expression or protein production. Unlike many conventional drugs that act directly on proteins, RNA medicines can intervene earlier in the process by reducing disease-causing proteins, modifying RNA or providing instructions to produce therapeutic proteins. This programmable approach can expand the range of disease targets available to drug developers.
Advances in RNA design and delivery have improved the stability, specificity and durability of these medicines. Key approaches include antisense oligonucleotides (ASOs) and RNA interference (RNAi), which regulate protein production. mRNA provides instructions to produce therapeutic proteins. RNA editing makes targeted changes to RNA without permanently altering DNA. These technologies are expanding into cardiovascular, metabolic, neurological and infectious diseases.
Against this backdrop, Alnylam Pharmaceuticals (ALNY - Free Report) , Ionis Pharmaceuticals (IONS - Free Report) , Wave Life Sciences (WVE - Free Report) and Arcturus Therapeutics (ARCT - Free Report) offer exposure to several of the most important branches of RNA medicine. Their platforms span RNAi, antisense technology, RNA editing and mRNA, while their pipelines extend RNA-based treatment into cardiometabolic, neurological, rare and respiratory diseases. The mix of commercial validation and advancing clinical programs gives these companies multiple avenues to participate in the continued expansion of RNA therapeutics. All four companies currently carry a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Alnylam Pharmaceuticals
Alnylam is a pioneer in RNAi therapeutics, using small interfering RNA molecules to silence specific genes involved in disease. The company has already demonstrated the technology’s commercial potential through its RNAi products portfolio. Its commercial RNA franchise includes Amvuttra (vutrisiran) for hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN) and transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Onpattro (patisiran) for hATTR-PN, Givlaari (givosiran) for acute hepatic porphyria and Oxlumo (lumasiran) for primary hyperoxaluria type 1. Alnylam generated $2.21 billion in global net product revenues in the first half of 2026, up 94% year over year, driven primarily by Amvuttra sales with increased patient demand mainly among ATTR-CM patients in the United States.
Alnylam also has a broad RNAi pipeline spanning cardiometabolic, neurological and bleeding disorders. Nucresiran is currently being evaluated in two phase III studies for hATTR-PN and ATTR-CM, while zilebesiran is being evaluated in a phase III study to reduce cardiovascular risk in patients with uncontrolled hypertension on two or more antihypertensives, one being a diuretic. In earlier-stage development, mivelsiran is being evaluated in separate phase II studies for cerebral amyloid angiopathy and Down syndrome-associated Alzheimer’s disease. At the same time, ALN-6400 is being evaluated in two separate mid-stage studies for hereditary hemorrhagic telangiectasia (HHT) and von Willebrand disease with heavy menstrual bleeding. Alnylam plans to report initial phase I data for ALN-HTT02 in Huntington’s disease in October 2026. Initial results from the phase II HHT study of ALN-6400 are also anticipated in the second half of 2026, providing another near-term pipeline catalyst.
Ionis Pharmaceuticals
Ionis is a pioneer in ASOtechnology, developing RNA-targeted medicines designed to bind specific RNA sequences and regulate the production of disease-associated proteins. Its RNA commercial portfolio includes Tryngolza (olezarsen) for adults with severe hypertriglyceridemia and familial chylomicronemia syndrome and Dawnzera (donidalorsen) for prevention of hereditary angioedema attacks in patients aged 12 years and older. Earlier this month, the FDA approved Zanvastro (zilganersen) as the first and only disease-modifying treatment for Alexander disease in pediatric and adult patients. Ionis also receives royalties from Wainua (eplontersen), marketed with AstraZeneca for hereditary transthyretin amyloidosis with polyneuropathy and Spinraza (nusinersen), marketed by Biogen for spinal muscular atrophy. Ionis reported $74 million in first-half 2026 total product sales, up 185% year over year, driven by strong Tryngolza and Dawnzera sales.
Ionis has several additional RNA-targeted medicines in development. Last week, the company reported positive top-line results from the phase III FUSION study of ulefnersen for people living with FUS-associated amyotrophic lateral sclerosis, demonstrating a statistically significant improvement on the primary endpoint assessing functional impairment and survival versus placebo, supporting the potential to modify disease progression. Ionis and partner Otsuka plan to discuss the results with the FDA and other health authorities to pursue potential expedited regulatory submission pathways for approval. Another late-stage candidate, obudanersen, is being evaluated in phase III for Angelman syndrome, with top-line data expected in the second half of 2027. Earlier-stage pipeline candidates include ION775, a next-generation siRNA targeting apoC-III, which is undergoing phase II development for severe hypertriglyceridemia. Meanwhile, GSK’s bepirovirsen, an antisense medicine for chronic hepatitis B developed in collaboration with Ionis, is currently under FDA Priority Review, with a decision expected on Oct. 26, 2026.
Wave Life Sciences
Wave Life Sciences is advancing next-generation RNA medicines through its PRISM platform, which combines multiple RNA-targeting modalities with proprietary chemistry and insights from human genetics. The platform includes RNA editing, RNAi, antisense and splicing technologies, enabling the company to pursue a range of therapeutic approaches across rare and common diseases.
Wave Life Sciences has no marketed RNA product, so its investment case is heavily dependent on clinical execution. WVE-006, a GalNAc-conjugated RNA-editing oligonucleotide, is being evaluated in the phase Ib/IIa RestorAATion-2 study in alpha-1 antitrypsin deficiency patients with the Pi*ZZ mutation. In May 2026, Wave Life Sciences reported encouraging clinical data demonstrating dose-dependent efficacy. Data from the 600-mg monthly multidose cohort are expected in the second half of 2026, while the company is also advancing regulatory discussions with the FDA on a potential accelerated-approval pathway. WVE-007, a GalNAc-siRNA targeting INHBE, is being evaluated in a phase IIa study as a monotherapy for obesity. Wave Life Sciences has also initiated a separate phase IIa study combining WVE-007 with Eli Lilly’s tirzepatide for obesity and plans to initiate a phase II study evaluating WVE-007 as a post-incretin maintenance therapy in the second half of 2026.
Arcturus Therapeutics
Arcturus focuses on mRNA medicines, supported by its LUNAR lipid-mediated delivery system and STARR self-amplifying mRNA technology. The company developed Kostaive (zapomeran/ARCT-154), a self-amplifying mRNA vaccine for the prevention of COVID-19. Arcturus regained global rights to Kostaive from CSL Seqirus in 2026 following the termination of their collaboration. However, its first-half financials primarily reflected collaboration and grant revenues rather than direct Kostaive product sales. Arcturus reported $5 million of total revenues in the first half of 2026, down sharply from $57.7 million a year earlier, largely because of lower collaboration revenues following the wind-down of its CSL Seqirus arrangement.
Arcturus Therapeutics Holdings Inc. Price and Consensus
Arcturus’ current therapeutics pipeline is entirely focused on RNA-based candidates. ARCT-810 is an intravenously administered mRNA therapeutic in phase II development for ornithine transcarbamylase (OTC) deficiency. Last week, the company reported interim data showing that ARCT-810 reduced and/or maintained first-morning fasting ammonia within the normal range. Arcturus plans to add ARCT-2601, a next-generation mRNA therapeutic for OTC deficiency, to the ongoing phase II study of ARCT-810 in OTC deficiency near year-end 2026, under an amended study protocol. ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis in patients with Class I mutations, remains in phase II, with a decision on advancing the candidate to phase III expected in fourth-quarter 2026.
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4 RNA Therapeutics Stocks Leading the Next Wave of Innovation
Key Takeaways
RNA therapeutics are medicines that use RNA molecules to influence gene expression or protein production. Unlike many conventional drugs that act directly on proteins, RNA medicines can intervene earlier in the process by reducing disease-causing proteins, modifying RNA or providing instructions to produce therapeutic proteins. This programmable approach can expand the range of disease targets available to drug developers.
Advances in RNA design and delivery have improved the stability, specificity and durability of these medicines. Key approaches include antisense oligonucleotides (ASOs) and RNA interference (RNAi), which regulate protein production. mRNA provides instructions to produce therapeutic proteins. RNA editing makes targeted changes to RNA without permanently altering DNA. These technologies are expanding into cardiovascular, metabolic, neurological and infectious diseases.
Against this backdrop, Alnylam Pharmaceuticals (ALNY - Free Report) , Ionis Pharmaceuticals (IONS - Free Report) , Wave Life Sciences (WVE - Free Report) and Arcturus Therapeutics (ARCT - Free Report) offer exposure to several of the most important branches of RNA medicine. Their platforms span RNAi, antisense technology, RNA editing and mRNA, while their pipelines extend RNA-based treatment into cardiometabolic, neurological, rare and respiratory diseases. The mix of commercial validation and advancing clinical programs gives these companies multiple avenues to participate in the continued expansion of RNA therapeutics. All four companies currently carry a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Alnylam Pharmaceuticals
Alnylam is a pioneer in RNAi therapeutics, using small interfering RNA molecules to silence specific genes involved in disease. The company has already demonstrated the technology’s commercial potential through its RNAi products portfolio. Its commercial RNA franchise includes Amvuttra (vutrisiran) for hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN) and transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Onpattro (patisiran) for hATTR-PN, Givlaari (givosiran) for acute hepatic porphyria and Oxlumo (lumasiran) for primary hyperoxaluria type 1. Alnylam generated $2.21 billion in global net product revenues in the first half of 2026, up 94% year over year, driven primarily by Amvuttra sales with increased patient demand mainly among ATTR-CM patients in the United States.
Alnylam Pharmaceuticals, Inc. Price and Consensus
Alnylam Pharmaceuticals, Inc. price-consensus-chart | Alnylam Pharmaceuticals, Inc. Quote
Alnylam also has a broad RNAi pipeline spanning cardiometabolic, neurological and bleeding disorders. Nucresiran is currently being evaluated in two phase III studies for hATTR-PN and ATTR-CM, while zilebesiran is being evaluated in a phase III study to reduce cardiovascular risk in patients with uncontrolled hypertension on two or more antihypertensives, one being a diuretic. In earlier-stage development, mivelsiran is being evaluated in separate phase II studies for cerebral amyloid angiopathy and Down syndrome-associated Alzheimer’s disease. At the same time, ALN-6400 is being evaluated in two separate mid-stage studies for hereditary hemorrhagic telangiectasia (HHT) and von Willebrand disease with heavy menstrual bleeding. Alnylam plans to report initial phase I data for ALN-HTT02 in Huntington’s disease in October 2026. Initial results from the phase II HHT study of ALN-6400 are also anticipated in the second half of 2026, providing another near-term pipeline catalyst.
Ionis Pharmaceuticals
Ionis is a pioneer in ASOtechnology, developing RNA-targeted medicines designed to bind specific RNA sequences and regulate the production of disease-associated proteins. Its RNA commercial portfolio includes Tryngolza (olezarsen) for adults with severe hypertriglyceridemia and familial chylomicronemia syndrome and Dawnzera (donidalorsen) for prevention of hereditary angioedema attacks in patients aged 12 years and older. Earlier this month, the FDA approved Zanvastro (zilganersen) as the first and only disease-modifying treatment for Alexander disease in pediatric and adult patients. Ionis also receives royalties from Wainua (eplontersen), marketed with AstraZeneca for hereditary transthyretin amyloidosis with polyneuropathy and Spinraza (nusinersen), marketed by Biogen for spinal muscular atrophy. Ionis reported $74 million in first-half 2026 total product sales, up 185% year over year, driven by strong Tryngolza and Dawnzera sales.
Ionis Pharmaceuticals, Inc. Price and Consensus
Ionis Pharmaceuticals, Inc. price-consensus-chart | Ionis Pharmaceuticals, Inc. Quote
Ionis has several additional RNA-targeted medicines in development. Last week, the company reported positive top-line results from the phase III FUSION study of ulefnersen for people living with FUS-associated amyotrophic lateral sclerosis, demonstrating a statistically significant improvement on the primary endpoint assessing functional impairment and survival versus placebo, supporting the potential to modify disease progression. Ionis and partner Otsuka plan to discuss the results with the FDA and other health authorities to pursue potential expedited regulatory submission pathways for approval. Another late-stage candidate, obudanersen, is being evaluated in phase III for Angelman syndrome, with top-line data expected in the second half of 2027. Earlier-stage pipeline candidates include ION775, a next-generation siRNA targeting apoC-III, which is undergoing phase II development for severe hypertriglyceridemia. Meanwhile, GSK’s bepirovirsen, an antisense medicine for chronic hepatitis B developed in collaboration with Ionis, is currently under FDA Priority Review, with a decision expected on Oct. 26, 2026.
Wave Life Sciences
Wave Life Sciences is advancing next-generation RNA medicines through its PRISM platform, which combines multiple RNA-targeting modalities with proprietary chemistry and insights from human genetics. The platform includes RNA editing, RNAi, antisense and splicing technologies, enabling the company to pursue a range of therapeutic approaches across rare and common diseases.
WAVE Life Sciences Ltd. Price and Consensus
WAVE Life Sciences Ltd. price-consensus-chart | WAVE Life Sciences Ltd. Quote
Wave Life Sciences has no marketed RNA product, so its investment case is heavily dependent on clinical execution. WVE-006, a GalNAc-conjugated RNA-editing oligonucleotide, is being evaluated in the phase Ib/IIa RestorAATion-2 study in alpha-1 antitrypsin deficiency patients with the Pi*ZZ mutation. In May 2026, Wave Life Sciences reported encouraging clinical data demonstrating dose-dependent efficacy. Data from the 600-mg monthly multidose cohort are expected in the second half of 2026, while the company is also advancing regulatory discussions with the FDA on a potential accelerated-approval pathway. WVE-007, a GalNAc-siRNA targeting INHBE, is being evaluated in a phase IIa study as a monotherapy for obesity. Wave Life Sciences has also initiated a separate phase IIa study combining WVE-007 with Eli Lilly’s tirzepatide for obesity and plans to initiate a phase II study evaluating WVE-007 as a post-incretin maintenance therapy in the second half of 2026.
Arcturus Therapeutics
Arcturus focuses on mRNA medicines, supported by its LUNAR lipid-mediated delivery system and STARR self-amplifying mRNA technology. The company developed Kostaive (zapomeran/ARCT-154), a self-amplifying mRNA vaccine for the prevention of COVID-19. Arcturus regained global rights to Kostaive from CSL Seqirus in 2026 following the termination of their collaboration. However, its first-half financials primarily reflected collaboration and grant revenues rather than direct Kostaive product sales. Arcturus reported $5 million of total revenues in the first half of 2026, down sharply from $57.7 million a year earlier, largely because of lower collaboration revenues following the wind-down of its CSL Seqirus arrangement.
Arcturus Therapeutics Holdings Inc. Price and Consensus
Arcturus Therapeutics Holdings Inc. price-consensus-chart | Arcturus Therapeutics Holdings Inc. Quote
Arcturus’ current therapeutics pipeline is entirely focused on RNA-based candidates. ARCT-810 is an intravenously administered mRNA therapeutic in phase II development for ornithine transcarbamylase (OTC) deficiency. Last week, the company reported interim data showing that ARCT-810 reduced and/or maintained first-morning fasting ammonia within the normal range. Arcturus plans to add ARCT-2601, a next-generation mRNA therapeutic for OTC deficiency, to the ongoing phase II study of ARCT-810 in OTC deficiency near year-end 2026, under an amended study protocol. ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis in patients with Class I mutations, remains in phase II, with a decision on advancing the candidate to phase III expected in fourth-quarter 2026.