PacBio Announces Plans for Collaboration With n-Lorem Foundation and EspeRare to Advance Precision Therapies for Rare Genetic Diseases

Rhea-AI Summary

PacBio (NASDAQ: PACB) announced on Jan 12, 2026 plans to pursue a strategic collaboration with the n-Lorem Foundation and EspeRare to apply HiFi long‑read whole‑genome sequencing to support individualized antisense oligonucleotide (ASO) therapies for ultra‑rare genetic diseases. PacBio will donate sequencing reagents and provide scientific resources to enable variant phasing, structural variant detection, repeat expansion resolution, and regulatory‑grade molecular characterization to inform ASO target identification, design feasibility, and regulatory documentation. The collaboration aims to reduce sequential tests, accelerate target validation, and strengthen end‑to‑end genomics‑driven therapy development for n‑of‑1 patients.

News Market Reaction

+3.61%

17 alerts

+3.61% News Effect

+6.9% Peak in 26 hr 9 min

+$23M Valuation Impact

$658M Market Cap

0.6x Rel. Volume

On the day this news was published, PACB gained 3.61%, reflecting a moderate positive market reaction. Argus tracked a peak move of +6.9% during that session. Our momentum scanner triggered 17 alerts that day, indicating notable trading interest and price volatility. This price movement added approximately $23M to the company's valuation, bringing the market cap to $658M at that time.

Data tracked by StockTitan Argus on the day of publication.

Market Reality Check

Price: $2.21 Vol: Volume 11,395,578 is 1.67...

high vol

$2.21 Last Close

Volume Volume 11,395,578 is 1.67x the 20-day average, indicating elevated trading activity ahead of this partnership news. high

Technical Shares at $1.94 were trading above the 200-day MA of $1.49, suggesting some prior recovery despite recent weakness.

Peers on Argus

PACB was down 4.9% while peers were mixed: DCTH and SRDX slightly up, RXST down ...

1 Up

PACB was down 4.9% while peers were mixed: DCTH and SRDX slightly up, RXST down 8.11%, SENS and CTKB modestly lower. Momentum scans only flagged RXST earlier with a strong upside move, reinforcing a stock-specific backdrop for PACB.

Common Catalyst Some peers (SENS, CTKB) issued preliminary revenue updates, pointing to an earnings-season backdrop, while PACB’s announcement is a strategic collaboration in rare-disease genomics.

Historical Context

5 past events · Latest: Jan 08 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 08	Technology update	Positive	-4.2%	Launch of CiFi long-read 3C method with chromosome-scale assemblies.
Dec 30	Investor conference	Neutral	+1.2%	Planned presentation at J.P. Morgan Healthcare Conference with webcast.
Nov 28	Investor conference	Neutral	-2.9%	Participation in Piper Sandler healthcare conference fireside chat.
Nov 13	Multiple conferences	Neutral	-1.2%	Schedule of three investor conferences and webcasted fireside chats.
Nov 05	Earnings update	Neutral	-4.3%	Q3 2025 results with slight revenue decline but margin and loss improvements.

Pattern Detected

Recent PACB news often saw muted-to-negative next-day moves even on seemingly positive technology or partnership updates.

Recent Company History

Over the last few months, PacBio has focused on technology advances, investor outreach, and cost discipline. On Nov 5, 2025, Q3 results showed $38.4M revenue, improved non-GAAP gross margin of 42%, and reduced operating expenses, yet the stock fell 4.25%. Subsequent conference announcements in November had small negative or modest positive reactions. The Jan 8, 2026 CiFi long-read method update, a clear technology milestone, also saw a 4.23% decline, underscoring a pattern of cautious trading around news.

Market Pulse Summary

This announcement details a proposed collaboration with n-Lorem and EspeRare to apply PacBio’s HiFi ...

Analysis

This announcement details a proposed collaboration with n-Lorem and EspeRare to apply PacBio’s HiFi long-read whole-genome sequencing to individualized ASO therapies for ultra-rare diseases. It extends PacBio’s role from research genomics into precision-therapy development and clinical implementation. In context of prior partnerships and technology launches, key watchpoints include concrete project throughput, evidence that donated sequencing builds durable demand, and how such programs intersect with PacBio’s broader path to profitability.

Key Terms

antisense oligonucleotide (aso) therapies, long-read whole genome sequencing, variant phasing, structural variation, +4 more

8 terms

antisense oligonucleotide (aso) therapies medical

"individualized antisense oligonucleotide (ASO) therapies for ultra-rare genetic diseases"

Antisense oligonucleotide (ASO) therapies are short, lab-made strands of genetic material designed to stick to a specific RNA message inside cells and change how a gene is read—reducing, correcting, or blocking production of a harmful protein. They matter to investors because ASOs offer highly targeted ways to treat diseases that conventional drugs often cannot reach; successful programs can drive large revenue opportunities but also carry significant scientific, regulatory, and manufacturing risks that affect company value.

long-read whole genome sequencing technical

"position long-read whole-genome sequencing as enabling infrastructure to support"

Long-read whole genome sequencing is a method that reads very long stretches of an organism’s DNA end-to-end, rather than piecing together many short snippets, producing a more complete and clearer map of the entire genetic code. Investors care because it can reveal complex or previously hidden genetic changes that improve diagnostics, drug target discovery and clinical decision-making, creating commercial opportunities for companies that develop the technology or use it in healthcare.

variant phasing medical

"including variant phasing, structural variation, repeat expansions, and regulatory"

Variant phasing is the process of determining whether two or more genetic changes sit on the same copy of a person’s chromosome or on opposite copies, analogous to checking whether two marks are on the same shoelace or on different laces. For investors, phasing matters because it changes how a genetic finding affects disease risk, diagnostic accuracy, drug targeting, and which patients are eligible for therapies or trials, thereby influencing the value of diagnostics and therapeutic programs.

structural variation medical

"including variant phasing, structural variation, repeat expansions, and regulatory"

Large-scale changes to a genome that add, remove, flip or move chunks of DNA—such as deletions, duplications, inversions or translocations—altering the ‘layout’ of genetic material much like rearranging, tearing or copying pages in a book. Investors track structural variation because these changes can cause or predict disease, affect how patients respond to therapies, enable or undermine diagnostic tests, and therefore influence a biotech asset’s clinical value, regulatory prospects and commercial potential.

repeat expansions medical

"including variant phasing, structural variation, repeat expansions, and regulatory"

Repeat expansions are mutations where a short piece of DNA is copied too many times in a row, like a word in a sentence being repeated so often it breaks the meaning. These runaway repeats can disrupt how a gene works and cause inherited diseases. For investors, repeat expansions matter because they define clear diagnostic tests, drug targets, and patient populations and therefore influence the value and risk of therapies, diagnostics, and regulatory approval paths.

regulatory-grade molecular characterization regulatory

"ASO design feasibility, and regulatory-grade molecular characterization, strengthening"

A rigorous, validated set of laboratory tests and data that describe the detailed molecular makeup of a biological sample—such as DNA sequences, proteins, or biomarkers—produced to meet the quality and documentation standards required by health regulators. Think of it as a certified blueprint that regulators can trust; for investors, it matters because regulatory-grade results reduce approval risk, support product claims and market access, and make scientific evidence more defensible in reviews and reimbursement decisions.

n-of-1 therapeutic models medical

"As individualized and n-of-1 therapeutic models gain momentum across the biopharma"

A treatment approach that develops and tests a therapy for a single patient, using that person as the whole “trial” and adjusting the therapy until it works. Think of it like tailoring a suit to one person rather than making off-the-rack sizes. For investors, these models can speed proof-of-concept and open premium, highly personalized markets but also pose scaling, regulatory and reimbursement uncertainties that affect commercial value.

genomic architecture medical

"the first step is to understand the genomic architecture of each patient"

Genomic architecture is the overall layout and relationship of genes and other important DNA elements in an organism’s genome — like a blueprint showing where parts are located and how they’re connected. For investors, it matters because this layout influences how easily a disease can be understood, diagnosed, or treated; clearer architectures make it more likely that drugs or tests can be developed predictably, reducing scientific risk and affecting a biotech company’s value.

AI-generated analysis. Not financial advice.

The planned collaboration highlights the role of complete genome resolution in scaling n-of-1 therapeutic models

MENLO PARK, Calif., Jan. 12, 2026 (GLOBE NEWSWIRE) -- PacBio (NASDAQ: PACB), developer of the world's most advanced sequencing technologies, today announced plans to pursue a strategic collaboration with the n-Lorem Foundation and EspeRare that we believe will position long-read whole-genome sequencing as enabling infrastructure to support the development of individualized antisense oligonucleotide (ASO) therapies for ultra-rare genetic diseases.

Under the proposed collaboration, PacBio will provide its scientific expertise, and the collaboration will utilize PacBio’s long-read whole genome sequencing technology, to study the design, feasibility assessment, and molecular characterization of ASO therapies – to help extend the role of long-read genomics for those who may lack effective treatment options.

ASO therapies require a complete understanding of the genomic architecture, including variant phasing, structural variation, repeat expansions, and regulatory alterations that are frequently missed or unresolved by standard sequencing approaches. By integrating long-read whole-genome sequencing earlier in the therapeutic development process, the proposed collaboration will aim to reduce the need for multiple sequential tests, accelerate target validation, and improve confidence in ASO design.

Under the proposed collaboration, PacBio will donate sequencing reagents and provide expert scientific resources to support long-read genome analysis for research to further development of therapeutic treatments for individuals with rare diseases. These efforts are intended to directly inform target identification, ASO design feasibility, and regulatory-grade molecular characterization, strengthening the end-to-end pipeline from genomic discovery to enabling development of therapeutic interventions.

“Rare disease therapies demand a level of genomic resolution that simply isn’t achievable with fragmented testing approaches,” said Christian Henry. “By working with n-Lorem and EspeRare under the proposed collaboration, we aim to establish HiFi long-read whole genome sequencing as the preferred platform for precision therapeutic development in rare disease, where understanding the full complexity of the genome is essential to designing safe and effective interventions.”

n-Lorem Foundation is a nonprofit organization dedicated to discovering, developing, and providing free, individualized ASO therapies for patients with rare genetic diseases, often under patient-specific regulatory pathways. The EspeRare foundation works to unlock and advance translational infrastructure, partnerships, and patient access to novel therapeutics across rare disease communities. Together, the organizations represent a complementary ecosystem spanning patient identification, molecular characterization, therapy development, and clinical implementation.

“n-Lorem was founded to discover, develop, manufacture and provide experimental ASO treatments to patients with the rarest genetic mutations for free, for life. To bring genetic medicines to patients with extremely rare genetic diseases, the first step is to understand the genomic architecture of each patient,” said Stanley T. Crooke, M.D., Ph.D., Founder, chairman and CEO, n-Lorem Foundation. “PacBio’s generous donation of long-read sequencing for many patients each year will enhance our ability to meet the extraordinary demand from the nano-rare community. It exemplifies the heart of the therapeutic industry, and it adds to the generosity of so many companies in our industry that have stepped up to help n-Lorem do what seemed impossible six years ago.”

“PacBio’s long-read sequencing technology enables the level of genomic understanding required to guide care in rare diseases,” said Caroline Kant, Executive Director of EspeRare. “Through the proposed collaboration we will be able to translate complex genomic information into therapies tailored to each patient’s biology, bringing groundbreaking treatments to families who urgently need them.”

As individualized and n-of-1 therapeutic models gain momentum across the biopharma industry, PacBio aims to play a central role in supporting scalable, genomics-driven therapy development, where complete and accurate genome characterization is foundational to developing treatment options.

The proposed collaboration reflects PacBio’s broader commitment to advancing human health through technologies that enable deeper biological insight and support the development of potential actionable outcomes.

About PacBio
PacBio (NASDAQ: PACB) is a premier life science technology company that designs, develops, and manufactures advanced sequencing solutions to help scientists and clinical researchers resolve genetically complex problems. Our products and technologies, which include our HiFi long-read sequencing, address solutions across a broad set of research applications including human germline sequencing, plant and animal sciences, infectious disease and microbiology, oncology, and other emerging applications. For more information, please visit www.pacb.com and follow @PacBio.  

PacBio products are provided for Research Use Only. Not for use in diagnostic procedures.  

About n-Lorem Foundation
n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem received over 380 applications for treatment with more than 200 nano-rare patients approved and more than 30 patients on treatment. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.

To learn more about n-Lorem’s mission visit us at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to nano-rare patients in need.

About EspeRare
EspeRare is a nonprofit organization dedicated to advancing therapies for patients with rare diseases through strategic partnerships, translational infrastructure, and patient-centered programs. After a decade of removing drug development barriers that prevent promising science from reaching historically overlooked patients, EspeRare has become a pioneer in prenatal and personalized therapies for rare diseases. Combining biotech agility with a mission-driven model, EspeRare bridges the translational “valley of death” by aligning patient communities, academics, commercial partners, and regulators to advance treatments and support equitable access. Based in Geneva, it leverages its position at the crossroads of science, health diplomacy and the life science industry to structure collaborations that unlock innovative therapies and maximize global patient impact.

Forward Looking Statements
This press release may contain “forward-looking statements” within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the U.S. Private Securities Litigation Reform Act of 1995. All statements other than statements of historical fact are forward-looking statements, including statements relating to: the uses, advantages, or quality or performance of, or benefits or expected benefits of using, PacBio products or technologies, including in connection with the proposed collaboration; entering into the proposed collaboration; outcomes, results, developments and impacts of, and the positioning of PacBio products in connection with or as a result of, the proposed collaboration, such as reducing the need for multiple tests, acceleration of target validation, and improved confidence in therapeutic development and design, resulting from the use of PacBio products; use of PacBio products to translate genomic information into individually-tailored; and other future events. You should not place undue reliance on forward-looking statements because they are subject to assumptions, risks, and uncertainties and could cause actual outcomes and results to differ materially from currently anticipated results, including, challenges in negotiating and entering into collaboration arrangements with multiple parties and risk that PacBio may be unable to agree to terms on the proposed collaboration; challenges inherent in generating, validating and accelerating discoveries in connection with, and supporting the potential development of, individually-tailored genomics-related therapies; potential product performance and quality issues; regulatory issues related to therapeutic development; and third-party claims alleging infringement of patents and proprietary rights or seeking to invalidate PacBio's patents or proprietary rights, among others. Additional factors that could materially affect actual results can be found in PacBio's most recent filings with the Securities and Exchange Commission, including PacBio's most recent reports on Forms 8-K, 10-K, and 10-Q, and include those listed under the caption "Risk Factors." These forward-looking statements are based on current expectations and speak only as of the date hereof; except as required by law, PacBio disclaims any obligation to revise or update these forward-looking statements to reflect events or circumstances in the future, even if new information becomes available.

Contacts 

Investors: 

Jim Gibson: jamesgibson@pacb.com or ir@pacificbiosciences.com 

Media: 

pr@pacificbiosciences.com 

FAQ

What did PacBio (PACB) announce on January 12, 2026 about rare disease collaborations?

PacBio announced plans to pursue a strategic collaboration with n‑Lorem Foundation and EspeRare to apply HiFi long‑read whole‑genome sequencing to support individualized ASO therapy development.

What will PacBio provide under the planned collaboration with n‑Lorem and EspeRare?

PacBio will donate sequencing reagents and provide expert scientific resources to support long‑read genome analysis and molecular characterization for ASO development.

How will PacBio’s long‑read whole‑genome sequencing help ASO therapy design?

Long‑read sequencing improves detection of variant phasing, structural variants, repeat expansions, and regulatory alterations, increasing confidence in ASO target identification and design feasibility.

Who are the partners in the collaboration with PacBio and what are their roles?

n‑Lorem Foundation focuses on discovering and providing individualized ASO therapies; EspeRare advances translational infrastructure and patient access; PacBio supplies sequencing technology and expertise.

Does the announcement include financial guidance or commercial terms for PACB shareholders?

No financial guidance or commercial terms were provided; the announcement describes donated reagents and scientific support to advance research and therapeutic development.

What is the expected impact of the PacBio collaboration on n‑of‑1 therapeutic development timelines?

The collaboration aims to reduce the need for multiple sequential tests and accelerate target validation by integrating long‑read whole‑genome sequencing earlier in the development process.