RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (Australia), Priority Review (United States)

Structure/Sequence

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Sequence Code 29732340

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Sequence Code 29732483

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Clinical Trials associated with Patisiran sodium

NCT05023889

/ Active, not recruitingEarly Phase 1IIT

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.

Start Date03 Aug 2022

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

NCT04201418

/ CompletedNot Applicable

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Start Date18 Dec 2019

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

NCT03997383

/ Active, not recruitingPhase 3

APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

Start Date12 Sep 2019

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

100 Clinical Results associated with Patisiran sodium

100 Translational Medicine associated with Patisiran sodium

100 Patents (Medical) associated with Patisiran sodium

255

Literatures (Medical) associated with Patisiran sodium

31 Dec 2025·ANNALS OF MEDICINE

Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study

Article

Author: Mauricio, Elizabeth ; Yureneva, Elena ; Sperry, Brett W. ; Jay, Patrick Y. ; Freimer, Miriam ; Hussain, Yessar ; Bender, Shaun ; Fernandez, Joel ; Roblin, Steven ; Ananthasubramaniam, Karthikeyan ; Small, Roy ; Malhotra, Saurabh ; Shu, Francy ; Zolty, Ronald ; Capocelli, Kelley ; Desai, Urvi ; Quan, Dianna ; Egolum, Ugochukwu

BACKGROUND:

This study assessed the effectiveness and safety of patisiran in patients with V122I/T60A variant transthyretin (ATTRv) amyloidosis with polyneuropathy. These variants have been under-represented in previous trials of gene-silencing agents.

METHODS:

This was a multicenter, phase IV study conducted at 27 sites in the USA. Patients were ≥ 18 years, diagnosed with ATTRv amyloidosis with polyneuropathy and a documented V122I or T60A variant. Patisiran-treated patients were enrolled prospectively, ambispectively, and retrospectively. The primary endpoint was the proportion of patients with a stable or improved polyneuropathy disability (PND) score at 12 months vs. baseline. Safety was monitored throughout the trial.

RESULTS:

Sixty-seven patients were enrolled, of whom 58 received ≥ 1 dose of patisiran. In the efficacy population, 42/45 (93.3%) patients demonstrated stable or improved PND scores from baseline to Month 12. Patients also showed stable or improved quality of life, health status, autonomic symptoms, and cardiac function vs. baseline. Adverse events occurred in 13/42 (31.0%) patients in the prospective and ambispective cohorts; most were mild or moderate. No deaths or cardiac hospitalizations were considered related to patisiran.

CONCLUSIONS:

Patisiran demonstrated a consistent positive effect across multiple endpoints in patients with V122I/T60A ATTRv amyloidosis, including polyneuropathy manifestations.

01 Dec 2025·Transplantation Reviews

Impact of disease-modifying drugs in patients with transthyretin amyloidosis after liver transplantation: a systematic review

Review

Author: Santo, Christiane ; Dabarian, Andre ; Romero, Cristhian ; Fernandes, Fabio ; Vaz Kerges Bueno, Bruno

BACKGROUND:

Orthotopic liver transplant (OLT) was the first approved treatment for hereditary transthyretin amyloidosis (ATTRv). However, some patients continue to deteriorate due to ongoing wild-type TTR deposition and residual synthesis from extrahepatic sources. In recent years, disease-modifying therapies including TTR stabilizers (e.g., Tafamidis) and gene-silencing agents (e.g., Patisiran) have emerged, but their role in post-OLT patients remains unclear due to their exclusion from most clinical trials.

METHODS:

A systematic search was conducted in PubMed, Cochrane, and Embase (up to June 2025) using terms related to transthyretin amyloidosis, liver transplantation, and disease-modifying therapies. The objective was to evaluate clinical benefits and safety of these agents in symptomatic ATTRv patients after OLT.

RESULTS:

Disease-modifying therapies showed potential benefits in post-OLT ATTR patients. A total of 39 patients treated with tafamidis, inotersen, or patisiran were analyzed. Neurological improvements, including autonomic symptoms, NIS score, and quality of life, were based on 3 case reports and 32 patients from observational studies. Cardiovascular results were from 4 case reports, and biomarker findings from 3 case reports.

CONCLUSIONS:

Disease-modifying therapies may offer clinical benefits in post-OLT ATTRv patients. However, robust prospective studies and randomized trials are needed to confirm efficacy and ensure safety in this population.

01 Dec 2025·Current Research in Translational Medicine

Revolutionizing pediatric gene and cell therapy: The hope for lipid-based nanoparticles in blood disorders

Review

Author: Zahedipour, Fatemeh ; Ureña-Bailén, Guillermo ; Ramesh, Anjana ; Mezger, Markus ; Jaafari, Mahmoud Reza ; Wisser, Silas ; Gol, Tahereh Mohammadian

The rapidly advancing field of lipid-based nanoparticles (LNPs) as delivery systems for nucleic acids has the potential to revolutionize treatment strategies. LNPs have demonstrated exceptional versatility in delivering genetic material and therapeutic agents to target cells. In gene and cell therapy, LNPs could serve as efficient carriers for introducing genetic materials into the cells, addressing inherited genetic disorders at their root. Their minimal toxicity and immune response make them particularly suitable for pediatric applications. Additionally, the scalability and cost-effectiveness of LNP production offer practical advantages over methods such as viral vectors and electroporation (EP), improving accessibility to advanced therapies for children worldwide. In 2018, the first FDA-approved LNP-based siRNA therapy (Patisiran/ Onpattro®) for treating hereditary amyloidosis brought attention to the feasibility of LNPs for gene therapy. Eventually, authorization and approval of the mRNA-LNP vaccines against COVID-19 (Comirnaty® of BioNTech/Pfizer and SpikeVax® of Moderna) was another milestone for the development of LNP-based nucleic acid therapies. Later, LNPs were applied successfully for the delivery of pDNA, mRNA and siRNA in many types of genetic disorders and cancers. This innovative approach offers a brighter future for pediatric healthcare, where children can look forward to healthier and more fulfilling lives. This review paper provides an overview of the applications of LNPs in gene and cell therapies with a special focus on their pre-clinical application in primary cells, including natural killer cells, T cells, and hematopoietic stem cells, highlighting LNPs' efficacy, safety profile, and potential for transforming the landscape of pediatric healthcare in the future.

350

News (Medical) associated with Patisiran sodium

11 Jan 2026

·investors.alnylam.com

Alnylam Launches “Alnylam 2030” Strategy to Drive Next Era of Growth and Patient Impact

Jan 11, 2026 − Full Year 2025 Preliminary* Net Product Revenues of $2,987 Million (81% Growth vs. 2024), Driven Primarily by Preliminary* Total TTR Revenues of $2,487 Million (103% Growth vs. 2024) – − Company Provides 2026 Combined Net Product Revenue Guidance of $4,900 Million to $5,300 Million , Driven Primarily by Total TTR Net Product Revenue Guidance of $4,400 Million to $4,700 Million – – 2026 Pipeline Goals Focused on Continued Advancement of Numerous Multi-Billion-Dollar Opportunities – CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jan. 11, 2026-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its new five-year strategy, “ Alnylam 2030,” focused on scaling the Company’s operations through achieving leadership in ATTR amyloidosis, driving long-term growth through sustainable innovation, and delivering exceptional financial results with discipline and agility. Alnylam also today reported preliminary* fourth quarter and full year 2025 global net product revenues for AMVUTTRA, ONPATTRO, GIVLAARI and OXLUMO. New Five-Year Strategy: Alnylam 2030 Alnylam 2030 continues the Company’s 15-year heritage of establishing and delivering on ambitious five-year goals, the most recent of which was Alnylam P5x25. Over that time period, Alnylam has either met or exceeded all of its prior five-year goals. Alnylam 2030 is focused on continuing to successfully scale the Company’s operations. Specifically, the Company intends to achieve the following by the end of 2030: Achieve Global TTR Leadership: Build a Durable TTR Franchise Lead TTR market in revenue by 2030 and cumulatively across five-year period Launch best-in-class, next-gen silencer, nucresiran, in polyneuropathy by 2028 and cardiomyopathy by 2030 Lead TTR market in revenue by 2030 and cumulatively across five-year period Launch best-in-class, next-gen silencer, nucresiran, in polyneuropathy by 2028 and cardiomyopathy by 2030 Grow Through Sustainable Innovation: Deliver Therapies that Prevent, Halt, or Reverse Disease Deliver 2+ new transformative medicines beyond TTR with blockbuster potential Expand to 10 tissue types and >40 clinical programs Invest ~30% of revenues in non-GAAP R&D, including select external innovation Deliver 2+ new transformative medicines beyond TTR with blockbuster potential Expand to 10 tissue types and >40 clinical programs Invest ~30% of revenues in non-GAAP R&D, including select external innovation Scale with Discipline & Agility: Drive Sustained, Profitable Growth Achieve 25%+ total revenue CAGR through year-end 2030 Deliver ~30% non-GAAP operating margin Achieve 25%+ total revenue CAGR through year-end 2030 Deliver ~30% non-GAAP operating margin “The past five years have been transformational, as we delivered on our ambitious goals to expand the reach of our commercial product portfolio, grow our pipeline of transformative RNAi therapeutics, and achieve strong financial results leading to sustainable profitability,” said Yvonne Greenstreet , M.D., Chief Executive Officer of Alnylam . “The success we have delivered with the early launch momentum of AMVUTTRA in ATTR-CM has laid the foundation for our first flagship commercial franchise. Our focus for the next five-year period is clear: continue to establish durable leadership in TTR; harness topline growth to invest in a high-yielding pipeline with blockbuster opportunities; and continue to operate with financial discipline and agility to enable the Company to scale efficiently. As the pioneer in RNAi therapeutics that has delivered six approved products, Alnylam 2030 provides the strategic direction to catalyze our growth during the next stage of our evolution.” Preliminary Fourth Quarter and Full Year 2025 Commercial and Financial Performance* Total TTR: AMVUTTRA® (vutrisiran) & ONPATTRO® (patisiran) Preliminary* global net product revenues for AMVUTTRA and ONPATTRO for the fourth quarter were approximately $827 million and $32 million , respectively, together representing 151% total TTR growth compared to Q4 2024, and for the full year 2025 were approximately $2,314 million and $173 million , respectively, together representing 103% total TTR growth compared to full year 2024. Total Rare: GIVLAARI® (givosiran) & OXLUMO® (lumasiran) Preliminary* global net product revenues for GIVLAARI and OXLUMO for the fourth quarter were approximately $87 million and $50 million , respectively, together representing 26% total Rare growth compared to Q4 2024, and for the full year 2025 were approximately $308 million and $191 million , respectively, together representing 18% total Rare growth compared to full year 2024. 2026 Combined Net Product Revenue Guidance Alnylam announced today full year 2026 combined net product revenue guidance for AMVUTTRA, ONPATTRO, GIVLAARI and OXLUMO of $4,900 million to $5,300 million , representing 71% growth compared to 2025* at the mid-point of the guidance range. On a franchise level, the guidance is as follows: Total TTR (AMVUTTRA & ONPATTRO): $4,400 million to $4,700 million , representing 83% growth compared to 2025* at the mid-point of the guidance range. Total Rare (GIVLAARI & OXLUMO): $500 million to $600 million , representing 10% growth compared to 2025* at the mid-point of the guidance range. The Company plans to provide additional guidance for collaboration and royalty revenue and operating expenses when fourth quarter and full year 2025 earnings are released. 2026 Pipeline Goals Nucresiran – an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. Alnylam expects to: Advance the TRITON-CM Phase 3 trial in ATTR-CM and the TRITON-PN Phase 3 trial in hATTR-PN. Zilebesiran – an investigational RNAi therapeutic in development for the treatment of hypertension, in collaboration with Roche. Alnylam expects to: Advance the ZENITH Phase 3 cardiovascular outcomes trial. Mivelsiran – an investigational RNAi therapeutic in development for the treatment of cerebral amyloid angiopathy (CAA) and Alzheimer’s disease. Alnylam expects to: Complete enrollment of the cAPPricorn-1 Phase 2 trial in cerebral amyloid angiopathy in H1. Initiate a Phase 2 trial in Alzheimer’s disease in H1. ALN-6400 – an investigational RNAi therapeutic in development for the treatment of bleeding disorders. Alnylam expects to: Report Phase 1 data in healthy volunteers in H2. Report clinical proof of concept in hereditary hemorrhagic telangiectasia in H2. Initiate a Phase 2 trial in a second bleeding disorder in H1. ALN-4324 – an investigational RNAi therapeutic in development for the treatment of type 2 diabetes mellitus. Alnylam expects to: Initiate a Phase 2 trial in H1. ALN-HTT02 – an investigational RNAi therapeutic in development for the treatment of Huntington’s disease, in collaboration with Regeneron. Alnylam expects to: Report Phase 1 data in H2. ALN-2232 – an investigational RNAi therapeutic in development for the treatment of obesity & weight management. Alnylam expects to: Report Phase 1 data in H2. In addition, the Company plans to file Investigational New Drug (IND) applications for three to four new Alnylam -led programs by the end of 2026. Alnylam management will discuss its preliminary 2025 net product revenues, as well as 2026 goals and guidance during a webcast presentation at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, California tomorrow, Monday, January 12, 2026 at 9:00 am PT ( 12:00 pm ET ). About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com or follow Alnylam on X, LinkedIn, Facebook, Instagram, or YouTube. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding: Alnylam’s potential achievement of the goals in its “ Alnylam 2030” strategy and its ability to drive the next era of growth and patient impact; Alnylam’s ability to achieve global TTR leadership, drive long-term growth through sustainable innovation, and deliver exceptional financial results with discipline and agility; the size of the opportunities of any of the product candidates in Alnylam’s pipeline; the potential for AMVUTTRA in ATTR-CM to be Alnylam’s flagship commercial franchise; the potential for Alnylam to advance its research and development programs, including its goals and expectations regarding the clinical development of nucresiran, zilebesiran, mivelsiran, ALN-6400, ALN-4324, ALN-HTT02, and ALN-2232, including the timeline of the initiation, completion of enrollment, and reporting of data and/or proof of concept from, any clinical trials; the number of IND applications Alnylam plans to file for Alnylam -led programs by the end of 2026; and Alnylam’s projected commercial and financial performance, including the expected range of global net product revenues for 2026 should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “ Alnylam 2030” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including nucresiran, zilebesiran, mivelsiran, ALN-6400, ALN-4324, ALN-HTT02, and ALN-2232; the possibility of unfavorable new clinical data and further analyses of existing clinical data; interim and preliminary data; the possibility that clinical data are subject to differing interpretations and assessments by regulatory agencies; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC . In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. Use of Non-GAAP Financial Measures This press release contains references to forward-looking financial measures of non-GAAP R&D and non-GAAP operating margin that were not prepared in accordance with U.S. generally accepted accounting principles (GAAP). We do not provide a reconciliation of these forward-looking non-GAAP financial measures to the most directly comparable financial measures calculated and presented in accordance with GAAP on a forward-looking basis because such reconciliation is not accessible with reasonable effort, due to the uncertainty and inherent difficulty of predicting the occurrence and the financial impact of adjustments, such as stock-based compensation expense; such adjustments could be material. We use these non-GAAP measures as an indicator of financial performance for the purpose of internal evaluation of progress toward financial objectives. We also believe that these non-GAAP measures provide investors with useful information with respect to our operational goals. A non-GAAP financial measure is not, and should not be viewed as, a substitute for financial measures required by GAAP, has no standardized meaning prescribed by GAAP, and may not be comparable to the calculation of the measure by other companies. * The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2026 . View source version on businesswire.com: https://www.businesswire.com/news/home/20260111699710/en/ Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) +1-617-682-4340 Josh Brodsky (Investors) +1-617-551-8276 Source: Alnylam Pharmaceuticals, Inc. For Media Inquiries, please contact: Christine Akinc Chief Corporate Communications Officer media@alnylam.com 617-682-4340 For Investor Inquiries, please contact: Josh Brodsky VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276 Essential assets and documents related to Alnylam

Financial StatementPhase 3

08 Jan 2026

·www.businesswire.com

Acuitas Therapeutics Expands Collaboration with RNA Technologies & Therapeutics Through Strategic Investment

VANCOUVER, British Columbia--(BUSINESS WIRE)--Acuitas Therapeutics, the global leader in lipid nanoparticle (LNP) delivery systems for nucleic acid therapeutics, today announced that it has acquired a majority stake in RNA Technologies & Therapeutics (RNA T&T), a company specializing in the design, optimization and manufacture of high-quality RNA constructs. This investment will support the growth and development of RNA T&T, allowing it to further develop and scale its operations. “Having worked extensively with RNA T&T over the last number of years, we are impressed with their scientific approach and technical know-how, which brings a level of precision and consistency that is critical in RNA drug development." Clinical development of RNA therapeutics requires optimization of both the mRNA payload and the LNP carrier to maximize efficacy and safety. An integrated approach for these two key components derisks clinical programs and supports accelerated availability to patients in need. While the companies will continue to operate independently, this investment will give partners of each company streamlined access to complementary world-class RNA optimization and manufacturing know-how and LNP delivery expertise. By integrating these capabilities, partners can take a more holistic approach to RNA-LNP therapeutics, helping to reduce development complexity, accelerate clinical timelines and preemptively mitigate risk across multiple programs. “As genetic medicines expand into more complex areas such as personalized therapies, biotechnology and pharmaceutical companies are seeking efficient access to RNA and LNP design and engineering expertise,” said Dr. Thomas Madden, CEO of Acuitas Therapeutics. “Having worked extensively with RNA T&T over the last number of years, we are impressed with their scientific approach and technical know-how, which brings a level of precision and consistency that is critical in RNA drug development. Ultimately, this investment gives our partners a coordinated way to engage experts from both companies as they develop next-generation RNA-LNP therapies.” "The synergy between our organizations is a natural result of our shared commitment to scientific integrity and technical excellence," said Pierrino Torbey, Board Member and interim CEO of RNA T&T. "Acuitas is the ideal partner for our next phase of growth, as both companies are deeply rooted in science and equally committed to advance the next generation of RNA-LNP medicines. Acuitas has collaborated with RNA T&T since its inception, and together the two companies have collaborated at both the research and clinical level. This investment deepens the relationship between Acuitas and RNA T&T, while also allowing the latter to grow its operational footprint, further develop its proprietary algorithmic platform for RNA sequence optimization and expand its capabilities in small-batch GMP-grade production of personalized and n-of-few therapies.” “From the beginning, it was clear that both teams are aligned in scientific ethos and in their approach to advancing RNA-LNP therapeutics,” said RNA T&T co-founder and Scientific Advisor Mohamad-Gabriel Alameh, Ph.D., who provided the RNA design for the first personalized CRISPR therapy administered to baby KJ Muldoon. “This investment strengthens RNA T&T’s ability to support highly complex and personalized RNA programs, while allowing partners to benefit from seamless access to select the right LNP delivery vehicle from Acuitas’ extensive portfolio of clinically validated LNP.” About RNA Technologies & Therapeutics Founded in 2022 in Montreal, Quebec, RNA Technologies & Therapeutics (RNA T&T) Inc., was established by leading RNA scientists and vaccinologists with a broad expertise in nucleic acid delivery systems, including Dr. Mohamad‑Gabriel Alameh and Nobel Prize laureate Dr. Drew Weissman. The company specializes in the collaborative design, optimization, and manufacture of RNA constructs, working closely with partners to refine sequences and deliver payload with superior biological performance. RNA T&T is dedicated to advancing the next generation of RNA therapeutics through rigorous science, innovative technologies, and a commitment to enabling more precise and personalized medicine. For more information, please visit www.RNATechnologies.com. About Acuitas Therapeutics Acuitas Therapeutics Inc., the global leader in lipid nanoparticle (LNP) delivery systems for nucleic acid therapeutics, is a Vancouver-based company collaborating with pharmaceutical and biotech companies, academic researchers, and global health organizations to advance a broad range of medicines for a variety of diseases. Acuitas’ clinically validated LNP technology has had a profound global impact — most notably enabling the Pfizer-BioNTech COVID-19 vaccine, COMIRNATY®, which has protected billions of people in more than 180 countries. Its technology also enables ONPATTRO® by Alnylam Pharmaceuticals, the first FDA-approved RNAi therapeutic for treating the rare and fatal disease transthyretin amyloidosis. More recently, Acuitas’ LNP technology has delivered other groundbreaking firsts: the first in-human proof of concept for genome base editing and the first personalized CRISPR therapy. Today, Acuitas is advancing next-generation LNP to support a variety of therapeutic modalities. This includes targeted LNP for extrahepatic and in vivo CAR T-cell therapies, epigenetic medicines to modulate gene expression without altering DNA, and multivalent vaccines for infectious diseases — such as malaria, HIV/AIDS, and tuberculosis — as well as oncology vaccines, including personalized cancer vaccines. For more information, please visit www.AcuitasTx.com.

VaccinemRNADrug ApprovalImmunotherapy

17 Dec 2025

·investors.alnylam.com

Alnylam to Invest $250 Million to Add Enzymatic Ligation Platform to U.S. Manufacturing Facility to Meet Growing Global Demand for RNAi Therapeutics

Dec 17, 2025 New platform is expected to substantially increase manufacturing capacity and lower production costs, enabling investment in expanding pipeline FDA has accepted Alnylam’s enzymatic ligation manufacturing platform into its Emerging Technology Program, fast-tracking global regulatory engagement CAMBRIDGE, Mass. --(BUSINESS WIRE)--Dec. 17, 2025-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the planned expansion of its state-of-the-art manufacturing facility in Norton, Massachusetts . The Company is preparing to invest $250 million to advance what is poised to become the industry’s first fully dedicated, proprietary, siRNA enzymatic-ligation manufacturing facility. This investment is expected to meaningfully expand capacity, significantly reduce production costs, and position Alnylam to support future launches across its growing pipeline of potential new medicines. As part of the expansion, Alnylam also announced that this next-generation, scalable, enzymatic ligation manufacturing platform, siRELIS™, has been accepted into the U.S. Food and Drug Administration’s (FDA) Emerging Technology Program, accelerating dialogue with global health authorities on innovative manufacturing approaches for oligonucleotide-based medicines. This acceptance follows successful demonstration of Alnylam’s enzymatic ligation platform through production of pilot-scale batches of zilebesiran, which is being studied to reduce the risk of major adverse cardiovascular events in patients with hypertension (high blood pressure), and nucresiran, which is in development for the treatment of transthyretin-mediated amyloidosis (ATTR). “At this pivotal time with our expanding pipeline of RNAi therapeutics, Alnylam is accelerating development of siRNA manufacturing and changing what’s possible in a single facility,” said Yvonne Greenstreet , M.D., MBA, Chief Executive Officer of Alnylam . “This advance will be a critical enabler in the scaling of our pipeline to include potential treatments for diseases such as hypertension, type 2 diabetes, and obesity.” Expansion in Norton, MA The $250 million expansion of Alnylam’s 200,000 sq. ft. Norton facility will increase its capabilities to locally produce both clinical and commercial supply of siRNA oligonucleotide drug substance for patients around the world. The facility, which opened in 2021, has played a key role in the growth of Alnylam’s clinical pipeline and acts as a pipeline accelerator for early-stage programs, including programs targeting different tissue types (e.g., liver, CNS, muscle, adipose, etc.). This expansion will strengthen Massachusetts’ position as a global hub of life sciences innovation. Construction is underway, with new capabilities expected to become fully operational by late 2027. “Alnylam represents the world-class health care innovation Massachusetts is known for. Alnylam has been a leader in life sciences in our state for over two decades,” said Governor Maura Healey . “Their scientific breakthroughs in siRNA manufacturing have transformed care by providing RNAi therapeutics to people who need it most, while creating thousands of jobs across our state. We're excited to support their growth through the expansion of a manufacturing facility in Southeastern Mass., and we're grateful for their continued investment in Massachusetts.” “Alnylam’s continued investment in Massachusetts underscores the Commonwealth’s role as a global leader in biomanufacturing,” said Dr. Kirk Taylor , President and CEO of the Massachusetts Life Sciences Center (MLSC). “This expansion will bring high-quality jobs to this area and ensure patients worldwide have the opportunity to benefit from medicines made right here in Massachusetts . We look forward to deepening our partnership as we work together to advance the thriving life sciences ecosystem here in the Commonwealth.” A Breakthrough in Manufacturing at Scale Alnylam’s siRELIS™ platform builds RNAi therapeutics more efficiently using fewer materials and plant resources, and greatly expands capacity – making large-scale, sustainable production possible. “Manufacturing oligonucleotide-based medicines is highly complex, and the current manufacturing technology will struggle to meet increased demand. We have successfully applied a reproducible, less time-intensive process that increases throughput while maintaining the highest quality standards,” said Timothy Maines , Chief Technical Operations and Quality Officer at Alnylam . “The expansion of our Norton facility presents an opportunity to reinvent what’s possible in oligonucleotide manufacturing.” About Enzymatic Ligation Alnylam’s siRELIS™ (siRNA Enzymatic Ligation Synthesis) platform is a next-generation approach to siRNA manufacturing that assembles short, high-quality RNA fragments called “blockmers” into complete molecules more efficiently than traditional methods. This scalable, sustainable platform reduces use of starting materials and reliance on organic solvents and plant time, increasing capacity in a single facility. A brief video about enzymatic ligation can be viewed here. About Alnylam Manufacturing Alnylam has pioneered small interfering RNA (siRNA) manufacturing at scale, building world-class capabilities in chemistry, engineering, and quality. With a legacy of firsts and a commitment to speed, safety, and sustainability, Alnylam delivers innovative RNAi therapeutics to patients worldwide. To learn more, please visit the “Making Our Medicines” page on Alnylam’s website. About RNAi Therapeutics RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products include AMVUTTRA® (vutrisiran), ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran), which are being developed and commercialized by Alnylam , and Leqvio® (inclisiran) and Qfitlia™ (fitusiran), which are being developed and commercialized by Alnylam’s partners, Novartis and Sanofi, respectively. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram. Forward Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding the planned expansion of Alnylam’s manufacturing facility in Norton, Massachusetts , including Alnylam’s planned $250 million investment in the expansion; the ability of Alnylam’s Norton, Massachusetts facility to meet the global demand for Alnylam’s RNAi therapeutics; Alnylam’s ability to advance the industry’s first fully dedicated, proprietary, siRNA enzymatic-ligation manufacturing facility; the potential for Alnylam’s enzymatic ligation based manufacturing platform to expand manufacturing capacity, reduce production costs, enable more efficient production of RNAi therapeutics, make large-scale, sustainable production possible, and position Alnylam to invest in its pipeline and support future launches; Alnylam’s ability to accelerate development of siRNA manufacturing and to change what’s possible in a single facility; the potential for enzymatic ligation to be a critical enabler in the scaling of Alnylam’s portfolio to include potential treatments for diseases such as hypertension, type 2 diabetes and obesity; the potential for the expansion of Alnylam’s Norton facility to increase its capabilities to locally produce both clinical and commercial supply of siRNA oligonucleotide drug substance; the potential for Alnylam’s new manufacturing capabilities to become fully operational by late 2027; the potential for the expansion of Alnylam’s Norton facility to bring high quality jobs to the area and to ensure that patients worldwide have the opportunity to benefit from medicines made in Massachusetts ; and the potential for the expansion of Alnylam’s Norton facility to reinvent what’s possible in oligonucleotide manufacturing should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam P5x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, and Regeneron; the outcome of litigation; the risk of future government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC . In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. View source version on businesswire.com: https://www.businesswire.com/news/home/20251217194595/en/ Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) +1-617-682-4340 Josh Brodsky (Investors) +1-617-551-8276 Source: Alnylam Pharmaceuticals, Inc.

Oligonucleotide

100 Deals associated with Patisiran sodium

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KEGG	Wiki	ATC	Drug Bank
D10794	Patisiran sodium	N07XX12	DB14582

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Polyneuropathies	European Union	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Iceland	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Liechtenstein	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Norway	Alnylam Netherlands BV	27 Aug 2018
Amyloidosis, Hereditary, Transthyretin-Related	United States	Alnylam Pharmaceuticals, Inc.	10 Aug 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Transthyretin Amyloid Cardiomyopathy	NDA/BLA	United States	Alnylam Pharmaceuticals, Inc.	21 Feb 2023
Amyloid Neuropathies, Familial	Phase 3	United States	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Japan	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Argentina	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Australia	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Brazil	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Bulgaria	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Canada	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Cyprus	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	France	Alnylam Pharmaceuticals, Inc.	16 Jul 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01960348 (APOLLO, Pubmed \| J Formos Med Assoc)	Phase 3	Amyloidosis, Hereditary, Transthyretin-Related A97S gene variant	18	Patisiran 0.3 mg/kg	qdcmfvvdrb(vflodzkwok) = zawdgwfsvg rvorfxobxd (rinkhljrdv, -45.5 to -7.5)	Positive	01 Sep 2024
NCT02510261 (APOLLO, CTgov)	Phase 3	Amyloid Neuropathies, Familial \| Transthyretin-mediated amyloidosis	211	Patisiran (Prior Placebo Group of Study 004)	kwgrmsxaom = pjcopctrwh fmoiqiplwc (ftbatwdtal, fkwbnkbhsn - nninzpimes) View more	-	06 Dec 2023
				Patisiran (Prior Patisiran Group of Study 004)	kwgrmsxaom = irjghvulop fmoiqiplwc (ftbatwdtal, tuyevxecxq - igvzflkzlw) View more
NCT03997383 (APOLLO-B, Pubmed \| N Engl J Med)	Phase 3	Transthyretin Amyloid Cardiomyopathy hereditary \| variant \| wild-type	360	Patisiran 0.3 mg/kg	mkxlfgofeo(swsaqzgdpe) = olzxcpfpfi cfzephlqpw (rzgpfbavyh, lower)	Positive	26 Oct 2023
				Placebo	mkxlfgofeo(swsaqzgdpe) = bzadjdxhvn cfzephlqpw (rzgpfbavyh, higher)
NCT03997383 (APOLLO-B, Biospace) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	360	Patisiran 0.3 mg/kg	biuhihbhob(xhecymiqos) = fbaixfcgkc gdyewjdrod (mvoismmubd, 0.69-28.69 - P=0.02) Met View more	Positive	25 Oct 2023
NCT03997383 (APOLLO-B, CTgov)	Phase 3	Amyloidosis, Hereditary, Transthyretin-Related \| Transthyretin Amyloid Cardiomyopathy	360	Placebo+Patisiran (OLE) (Placebo (DB)/Patisiran (OLE))	xlxwgglawi(yghzoxukjf) = nubavohdge edaghzppcp (ywhtoxgiem, ftvhsjqvoi - ihcagbnpic) View more	-	18 Oct 2023
				OLE+Patisiran (Patisiran (DB+OLE))	xlxwgglawi(yghzoxukjf) = ctqwnyovoj edaghzppcp (ywhtoxgiem, impktbnfac - nreiejalkd) View more
NCT03997383 (APOLLO-B, ESC2023)	Phase 3	Amyloidosis transthyretin (TTR) amyloid \| N-terminal prohormone B-type natriuretic peptide \| troponin I ... View more	360	Patisiran	mhjujasxgq(kwmmswwknu): OR = 1.58 (95% CI, 1.03 - 2.42) View more	-	26 Aug 2023
				Placebo
NCT03997383 (APOLLO-B, Corporate Publications) Manual	Phase 3	Amyloidosis	359	Patisiran sodium	wpelchcwyv(izgsseqfch) = Patisiran demonstrated a benefit or trend toward benefit in change from baseline of most echocardiographic parameters compared with placebo at Month 12 pocihenlzj (pdefcpiiza )	Positive	30 Sep 2022
				Placebo
NCT01960348 \| NCT03759379 (APOLLO, ANA2022)	Phase 3	Polyneuropathies \| Amyloidosis NfL	-	Patisiran	dtawcddhdy(ivjtesmmwj) = pznulvpnpk hidjxqujub (gxprwczpmd )	Positive	14 Sep 2022
				Vutrisiran	dtawcddhdy(ivjtesmmwj) = djtnrncwlm hidjxqujub (gxprwczpmd )
NCT03997383 (APOLLO-B, Corporate Publications) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	360	Patisiran sodium	ahwwevxkgy(akfviszwhu) = wvpobobtyl ogumgiamto (kevglqgxxh ) View more	Positive	08 Sep 2022
				Placebo	ahwwevxkgy(akfviszwhu) = ktyxkmrusg ogumgiamto (kevglqgxxh ) View more
NCT03862807 (Pubmed) Manual	Phase 3	Amyloidosis, Hereditary, Transthyretin-Related	23	Patisiran sodium	txlaptszbq(fgcqekusyw) = krntfnjqyh pastcrquvc (ekkfzwkpwe, 86.1 - 92.3) View more	Positive	25 Feb 2022

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