Alnylam Pharmaceuticals Inc

Alnylam, a leader in RNA interference (RNAi) therapeutics, will utilize the AGD dataset to inform development of 'gene silencing' medicines

Members leverage AI-powered dataset to accelerate pharma R&D, and ensure that disease-impacting discoveries benefit all people

SAN DIEGO, Sept. 18, 2025 /PRNewswire/ -- Illumina, Inc. (NASDAQ: ILMN), and Nashville Biosciences, LLC (NashBio), today announced Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) (Alnylam) as a member of the Alliance for Genomic Discovery (AGD). Alnylam, a pioneer in RNA interference (RNAi) therapeutics, will utilize the AGD clinical genomic database to derive real-world insights and accelerate drug target discovery.

"We welcome Alnylam to the AGD as our members continue to leverage data that fuels innovation in novel therapeutics to improve lives of patients everywhere," said Todd Christian, senior vice president of Services, Arrays and Genomic Access at Illumina. "Alnylam's decision to join the alliance speaks to the immense value of this resource that combines powerful scale and depth of sequencing with clinical data quality and diverse ancestral representation to advance the critical work of our biopharma partners."

Alnylam is the ninth member of the alliance, joining AbbVie, Amgen, AstraZeneca, Bayer, Bristol Myers Squibb, GSK, Merck, and Novo Nordisk. Alnylam's partnership will further strengthen the AGD clinical genomic database, enabling additional investment to sequence and analyze a new set of 31,250 whole-genomes to be added to the alliance's already groundbreaking 250,000 whole-genome cohort.

RNAi therapeutics function by "silencing" genes that cause or contribute to disease, working upstream of traditional treatment approaches. The AGD's dataset will provide a valuable resource for Alnylam scientists who hunt for new therapeutic targets.

"We are excited to join the Alliance for Genomic Discovery and contribute to the expansion of this important dataset, while simultaneously mining it for insights," said Paul Nioi, senior vice president of Research at Alnylam. "We rely on human genetics to guide us as we discover and develop RNAi therapeutics with the potential to change medicine. The AGD dataset will bolster our ability to identify therapeutic targets that are relevant to patients from many different backgrounds."

As of March 2025, the AGD completed a major milestone in developing the most comprehensive clinical genomic dataset to date. Making this cohort fully representative—by linking analyzed, ancestrally diverse genomic data with informative longitudinal clinical phenotyping—has been a core priority from the start, both to ensure that discoveries benefit all people, and to uncover insights that are often missed in more homogeneous datasets.

The alliance's pace of sequencing, rapid and secure data analysis, and generation of readily usable insights with DRAGEN™ and Illumina Connected Analytics have surpassed previous large-scale genomic efforts, which typically took three to five years to reach 100,000 sequenced genomes. Large-scale aggregation with DRAGEN Iterative gVCF Genotyper further enhances variant calling accuracy and consistency across diverse populations, enabling deeper insights into rare and complex genetic traits. 

The AGD dataset is already proving valuable to alliance members. Novel therapeutic targets, particularly in autoimmune and neurodegenerative diseases, have been identified or validated, with reports published or underway.

"We are thrilled that Alnylam has chosen to join the AGD and help us expand this unique dataset for all members," said Leeland Ekstrom, chief executive officer of NashBio. "Additional data scale, as well as other planned extensions, such as adding proteomics and extracting more clinical phenotypes from the unstructured parts of the linked electronic health records, will accelerate the pace and types of innovation possible with this resource."

This growth in the alliance's membership coincides with its plans to add multiomic layers to its dataset, which will facilitate faster and more efficient target discovery and therapy development. Illumina and NashBio are actively expanding the AGD network to continue building upon current successes, accelerating therapeutic discovery, and creating new standards for clinical R&D pace, cost efficiency, and efficacy. 

Use of forward-looking statements

This release may contain forward-looking statements that involve risks and uncertainties. Among the important factors to which our business is subject that could cause actual results to differ materially from those in any forward-looking statements are: (i) challenges inherent in researching, developing and launching new technologies; (ii) our and our partners' ability to deploy new products, services, and applications, and to expand the markets for genomics-related products and services; and (iii) the challenges associated with multiparty collaborations, including our reliance on the performance of such partners, together with other factors detailed in our filings with the Securities and Exchange Commission, including our most recent filings on Forms 10-K and 10-Q, or in information disclosed in public conference calls, the date and time of which are released beforehand. We undertake no obligation, and do not intend, to update these forward-looking statements, to review or confirm analysts' expectations, or to provide interim reports or updates on the progress of the current quarter.

About Illumina

Illumina is improving human health by unlocking the power of the genome. Our focus on innovation has established us as a global leader in DNA sequencing and array-based technologies, serving customers in the research, clinical, and applied markets. Our products are used for applications in the life sciences, oncology, reproductive health, agriculture, and other emerging segments. To learn more, visit illumina.com and connect with us on X, Facebook, LinkedIn, Instagram, TikTok, and YouTube.

About NashBio

Nashville Biosciences LLC (NashBio), a wholly owned, for-profit subsidiary of Vanderbilt University Medical Center (VUMC), was created to make complex healthcare data easy to use for a wide range of life science research and development applications. Leveraging Vanderbilt University innovation, NashBio harnesses extensive real-world genomics and other longitudinal multi-modal datasets, along with powerful bioinformatics tools, to build and deliver a wide range of data products and services. NashBio works with clients in biotech, pharma, diagnostics, medical devices, and other life sciences domains to support their most critical use cases. We believe smarter data enables better outcomes for our clients and ultimately for patients. For more information, please visit NashBio.com, connect with us on LinkedIn or follow us on X at @NashvilleBio.

Contacts 

Investors:
Illumina Investor Relations
858-291-6421
IR@illumina.com

Media:
Christine Douglass
PR@illumina.com

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SOURCE Illumina, Inc.

OXLUMO has been shown to significantly reduce urinary oxalate, which drives the progression of PH1 Disease¹

MISSISSAUGA, ON, July 2, 2025 /CNW/ - Alnylam Canada ULC is pleased to announce that OXLUMO^® (lumasiran) is now funded across Canada through both public and private plans. OXLUMO is an RNA interference (RNAi) therapeutic administered via subcutaneous injection, indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients.¹

OXLUMO is the first therapy for PH1 approved in Canada, and among the first medications to be included on the common list of new drugs for rare diseases, as part of the Government of Canada's National Strategy for Drugs for Rare Diseases. The common list was developed to help patients with rare diseases have access to treatments as early as possible.

PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction.² Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body. Oxalate overproduction results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis (renal deposition of calcium oxalate crystals), progression to kidney failure, and systemic organ dysfunction.²

"Funding OXLUMO through the National Strategy for Drugs for Rare Diseases provides a new treatment option for people diagnosed with this debilitating, genetic disease – many of whom are infants and children," said Colleen Coxson, Country General Manager, Alnylam Canada ULC. "I want to congratulate the Canadian government for prioritizing access to therapies for those living with rare conditions, as there are often very limited options for patients."

There are several types of primary hyperoxaluria (PH), however, PH1 is the most common and the most severe form, accounting for 70 to 80 per cent of all PH cases.³ PH1 affects approximately four individuals per million, with some regions – such as the Middle East and North Africa – having a higher genetic prevalence.⁴ Symptom onset ranges from early infancy to sixty years of age, with the median age being four to six years.⁴ The remainder of affected cases present in adulthood with 20 to 50 per cent presenting late stages of chronic kidney disease when diagnosed.⁴  

"This funding decision marks a major step forward in the management of hyperoxaluria type 1, offering hope to patients of all ages living with this ultra-rare genetic condition," said Dr. Vladimir Belostotsky, Division Head for Pediatric Nephrology at McMaster Children's Hospital. "The medication has been shown to effectively lower urine oxalate levels, reducing the burden that leads to severe clinical symptoms."

The positive recommendation for reimbursement was supported by results of the ILLUMINATE clinical studies, including ILLUMINATE-A: a randomized, double-blind, placebo-controlled clinical study in patients six years and older with PH1, ILLUMINATE-B: a single-arm clinical study in patients less than six years of age with PH1¹ and ILLUMINATE-C: a single-arm trial in patients of all ages with advanced PH1, including patients on dialysis.⁵ The ILLUMINATE-A study showed that OXLUMO met its primary endpoint, evidenced by a 53 per cent mean reduction in urinary oxalate, and a 65 per cent mean reduction in urinary oxalate relative to baseline.¹ In ILLUMINATE-B, OXLUMO demonstrated a 72 per cent mean reduction in spot urinary oxalate:creatinine ratio from baseline to month six (averaged from months three to six), meeting its primary endpoint.¹In ILLUMINATE-C, OXLUMO met its primary endpoint, demonstrating a 33% least squares (LS) mean reduction in plasma oxalate (POx) levels in patients not on dialysis (Cohort A) and a 42% LS mean reduction in POx levels  in patients on hemodialysis (Cohort B) from baseline to month six.⁶

About OXLUMO^® (lumasiran)¹

OXLUMO^® (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in the liver to deplete the production of the glycolate oxidase (GO) enzyme. By silencing HAO1 and depleting the GO enzyme through RNA interference, OXLUMO in turn reduces the amount of oxalate that is produced. This process helps address the root cause of the rare, genetic disease since an overproduction of urinary and plasma oxalate levels is the underlying cause of PH1.

About Primary Hyperoxaluria Type 1 (PH1)

PH1 is an ultra-rare genetic disease that is characterized by oxalate overproduction in the liver, causing renal damage. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones. PH1 is associated with a progressive decline in kidney function, which exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and deposition of oxalate in bones, eyes, skin, and heart, leading to severe illness and death. Management options to date have been limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). These measures only delay the inevitable progression to kidney failure and the need for intensive dialysis as a bridge to a dual or sequential liver/kidney transplant. Other impacts of the disease include: infants often fail to thrive, meaning they are weak and not growing or developing at a normal rate.⁷ Affected children frequently face developmental challenges, with social barriers and the requirement of accommodations to be made at school to meet their special medical needs.⁶

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.5 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 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. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018.

SOURCE Alnylam Pharmaceuticals, Inc.