ASOlutions™
Intelligent ASO Design Service
Your Solution for RNA Therapeutics Strategies
We deliver optimized, mechanism-based antisense oligonucleotide designs — driven by deep RNA biology expertise, critical thinking, and a comprehensive understanding of the genetic landscape.
Sources: Roots Analysis, Antisense Oligonucleotides Market (Dec 2025); FDA/EMA approval records as of Mar 2024.
Why ASOlutions™?
Expert-driven RNA biology that bridges the gap between research and therapeutic development
Deep Molecular Expertise
Founded by a PhD molecular biologist with hands-on experience in RNA splicing and gene therapy research.
Accelerated Design
What takes weeks of trial and error, we deliver in days. Our expert team rapidly identifies and designs optimized ASO candidates.
Precision Targeting
Every ASO design comes with comprehensive off-target analysis, thermodynamic predictions, and mechanistic rationale.
Risk Reduction
De-risk your pipeline early. Our expert analysis identifies the most promising candidates before synthesis.
Global Service
Fully virtual consultancy serving clients worldwide. Seamless collaboration across time zones.
Patient-Driven Mission
Rooted in collaboration with patient advocacy groups like FAME Argentina and CureSMA. We understand the urgency.
Spinraza Showed What's Possible
Imagine a disease that slowly steals a child's ability to move, swallow, and breathe. That's spinal muscular atrophy (SMA). Then scientists realized it wasn't only a "DNA problem" — it was an RNA problem. The body has a backup gene (SMN2) that could help, but it makes the wrong RNA message.
The breakthrough idea: don't replace the gene — fix the message.
Spinraza (nusinersen) is a short, engineered molecule that binds SMN2 RNA and forces the cell to include the missing piece. Children who would never sit up began reaching milestones. A once-devastating diagnosis became treatable. Spinraza proved a bigger point: RNA can be programmed.
Groundbreaking science built the foundation
The discovery of drugs like Spinraza was a triumph of deep biological insight and rigorous experimental work. Scientists systematically screened candidate molecules — tiling hundreds of ASOs across the target — and through careful experimentation identified the regions that mattered most. That pioneering effort created the knowledge base that expert-driven design builds on today.
Why now is a new era
Our team leverages the modern era of RNA evidence: comprehensive molecular datasets, structural biology insights, and an expanding track record of therapeutic successes and failures. But our work goes beyond therapeutics. We help researchers and teams build a smarter experimental plan before touching a pipette — prioritizing the most plausible intervention points and producing an explainable rationale for why a given strategy should work.
Building on decades of foundational research, we can now complement experimental screening with data-driven design.
Industry consultants who understand the challenges
ASOlutions wasn't born in an academic lab. It came from industry consultants who repeatedly saw the same challenge: "We know the target is interesting — but where do we intervene? Which molecular mechanisms matter? How do we justify strategies before committing resources?" We built what our clients desperately needed: expert RNA biology guidance for real-world development.
Our Expert Design Approach
We apply an RNA-first design methodology that integrates deep biological criteria to turn molecular evidence into rational intervention strategies. Think of it as taking the systematic approach that led to breakthrough ASO therapies — and making it repeatable, rigorous, and accessible to every team.
Analyzes molecular targets through multiple biological dimensions
Identifies optimal intervention points based on mechanistic criteria
Designs optimized antisense sequences using expert biological rules
Evaluates candidates for efficacy and safety parameters
Delivers ranked, scientifically justified designs for validation
Reduce synthesis costs
Fewer molecules to test — focused candidates, not fishing expeditions
Mechanistic rationale
Every candidate comes with scientific justification, not just a sequence
Repeatable methodology
Standardized expert workflow across targets and diseases — not a one-off project
Spinraza showed how RNA programming can change lives. ASOlutions exists to multiply that impact — by giving teams expert guidance to design ASO strategies from first principles and modern biological insights. And because the same design challenge exists in academic research and biotech R&D, our expertise serves both discovery and development.
We turn decades of RNA biology expertise into actionable ASO design strategies — for pharma, biotech, and academia — making research and translation faster, more cost-effective, and more mechanistically grounded.
Read the Full Scientific StoryOur Services
From expert in silico design to experimental validation—complete ASO development support
In Silico Design
Mechanism-based target selection, chemistry optimization tailored to your strategy and biological question, and design rationales ready for the bench
Basic Screening
Get started with ASO design
Tailored to your project scope
- Single target gene analysis
- One optimized ASO strategy
- Top 3 ASO candidates
- Off-target screening
- Design rationale report
- Vendor-ready sequences
Standard Design
Comprehensive ASO development
Tailored to your project scope
- Complete target gene analysis
- Full ASO candidate library
- Comprehensive off-target analysis
- Mechanism-based design rationale
- Chemistry modification recommendations
- Detailed design report
- Revision rounds available
- Vendor-ready sequences
- Standard protocol strategies
Advanced Consulting
End-to-end project partnership
Custom scope & partnership
- One biological question, multiple target genes
- Full candidate library with thermodynamic analysis
- Comprehensive off-target screening
- Tailored delivery strategy consultation
- Chemistry optimization & recommendations
- Synthesis partner coordination
- Scientific protocol design
- Priority support & revisions
Experimental Validation(Coming Soon)
Take your ASO candidates from expert design to experimental proof
In Vitro Validation
Coming Soon"Start your project with real, publication-ready results"
Cell-based ASO efficacy testing with knockdown quantification, dose-response curves, and toxicity assessment in relevant cell models.
- qPCR knockdown validation
- Western blot confirmation
- Cell viability assays
- IC50 determination
In Vivo Validation
Coming Soon"Ready to begin your path to clinical trials?"
Pre-clinical animal studies with tissue distribution analysis, pharmacokinetics, and efficacy evaluation in disease-relevant models.
- Tissue biodistribution
- PK/PD characterization
- Efficacy in disease models
- Safety & tolerability
Interested in validation services? Contact us to be notified when available.
Gene Intelligence Hub
Free tools to get started — explore genes, isoforms, and RNA structure to inform your ASO design
Examples: SMN2, ENSG00000172062, NM_000546
Gene Information Hub
Search any gene (HGNC / Ensembl / RefSeq) and view transcripts, exon–intron structure, UTRs, variants, and key annotations—optimized for ASO targeting.
Open HubRNA Structure Viewer
Predict RNA secondary structure and accessibility across a region. Overlay candidate ASO binding sites with accessibility metrics and ΔG.
View StructureIsoform Explorer
Compare isoforms side-by-side. Visualize exon inclusion patterns, CDS/UTR differences, and isoform-specific target windows for ASOs.
Explore IsoformsOne Team, Multiple Markets
The same fundamental problem — limited time and money, too many possible experiments — exists across academia, biotech, and pharma. Our experts turn "Where do we start?" into a ranked, mechanistically justified shortlist.
Same deep expertise → different outcomes. That's what makes us the go-to team for RNA science.
Academic Labs
Stronger hypotheses, faster iteration
Cleaner mechanistic stories, more publishable projects. Our experts help you justify targets before spending months at the bench.
- Target landscape + isoform map
- Regulatory feature hypothesis
- Ranked intervention sites
- Proposed validation experiments (RT-PCR, minigene, CLIP)
Biotech Startups
Fewer cycles, better early prioritization
Reduce synthesis/testing iterations and move faster to milestones. Data-driven candidate selection before committing resources.
- Full candidate ASO panel
- Off-target & liability screening
- Chemistry + delivery recommendations
- Milestone-oriented lead selection plan
Pharma & Patient Foundations
Scalable, cross-program design logic
Standardized target-to-lead workflows that scale across rare disease programs. Partner with patient communities to advance treatments.
- Continuous iterations + feedback loop
- IP strategy hooks (rationale, novelty, claims)
- Scale across multiple disease targets
- Patient advocacy partnership support
Meet the Founder
From a question that got laughed at to building the pipeline
A question that got laughed at
I grew up in Rome — Italian and half Argentinian — in an international school where biology felt like a language for patterns. In ninth grade, in 2010, we were studying ecosystems. I raised my hand and asked: could the human gut be considered an ecosystem, given how many different organisms live there together, competing and cooperating? The teacher pointed me out. The class laughed. Two years later, in August 2012, The Economist put the microbiome on its cover — framing humans as ecosystems of collaborating and competing microbes.
That was my first proof that "parallel thinking" can turn into public truth — and it's still how I build: connect mechanisms, systems, and evidence before the field catches up.
Scotland to Argentina: finding RNA as my home
I studied molecular biology at the University of Dundee in Scotland. In my third year, I heard about Alberto Kornblihtt in Argentina: a world-class RNA scientist and a rare kind of leader who defends public education and scientific institutions. I reached out, got an interview in London while he was traveling, took an eight-hour train from Dundee, and earned an internship in his lab.
From my first day there, I worked on antisense oligonucleotides and alternative splicing. I wasn't "introduced" to RNA later — RNA therapeutics shaped my scientific identity from the beginning.
SMA: science that answers a call
When I arrived in Alberto Kornblihtt's lab, I stepped into something bigger than a project. Alberto is one of the world's most respected RNA scientists — recipient of the 2025 Bunge and Born Prize in Biochemistry and Molecular Biology, and a Howard Hughes Medical Institute International Scholar for 15 years. What brought the lab into SMA research wasn't "market opportunity." It was urgency. Patient families — through Familias AME Argentina (FAME) — were desperate for options at a time when Spinraza still wasn't accessible in Argentina.
Alberto was cautious at first — he didn't want to promise what he couldn't deliver. But his decades of splicing biology gave him a path forward: instead of guessing, we would understand the mechanism. That journey led to a discovery we didn't expect — ASO treatment can reshape chromatin and transcription, not just RNA — and that work became a Cell paper and a foundation for improving therapeutic strategies.
Cell paper (Cover of the Issue): "Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy" — Marasco et al., 2022
Spinraza: FDA-approved Dec 2016. In Argentina, ANMAT authorized nusinersen March 2019.
Academia-grade mechanism. Industry-grade delivery.
During my PhD, our lab was also contracted by Argentine pharmaceutical companies to deliver RNA biology assays and structured reporting through a government-accredited high-level technical services program. It was my first training in "science that ships" — clear experimental plans, reproducible pipelines, and decision-ready results.
That experience is a direct ancestor of how ASOlutions operates today: evidence-first design, validation-minded planning, and reports that teams can act on.
Why I Founded ASOlutions
After my PhD, I went into industry — working in biotechnology across molecular biology and genome engineering. It taught me scale, constraints, and how decisions get made when time and money are real. But my heart stayed in RNA therapeutics, and I kept seeing the same bottleneck:
We now have enough accumulated knowledge to design smarter interventions — the question is how to put that knowledge to work before going to the bench.
ASOlutions is the consultancy I wished existed when I was a researcher. Expert-driven, rational ASO design that helps you choose where to intervene on RNA before you spend months at the bench — built by RNA scientists who understand the mechanism, for teams who want to move faster without losing rigor.
Scientific Advisors
Prof. Alberto Kornblihtt
Senior Scientific Advisor
UBA / CONICET
RNA Biology & Splicing
Dr. Martín García Sola
Bioinformatician
Bioinformatics & Computational Strategy
Dr. Juan Cristóbal Muñoz
Postdoc, University of Cambridge
ASO Therapeutics & Translational Research
Scientific Advisor roles; not directors or officers. Affiliations listed for identification purposes only.

Jose Stigliano, PhD
Founder & Chief Scientific Officer
Key Publications
Cell (Cover) — Chromatin effects of splicing-correcting ASOs in SMA
Marasco et al., 2022 — doi.org/10.1016/j.cell.2022.04.031 →Molecular Cell (under review) — ASO treatment reshapes 3D chromatin architecture through gene looping
Preprint: doi.org/10.1101/2025.10.10.681673v1 →Let's Connect
Ready to accelerate your ASO project? Get in touch for a free consultation.
Or reach us directly:
info@asolutions-therapeutics.comWhat You Receive
Preview of an ASOwalker™ design report. Gene context is shown in full — proprietary analysis and sequences are redacted.
ASOwalker™ Design Report
CNOT6L — Translation Blocking ASO Design
Gene & Transcript Context
Gene
CNOT6L
CCR4-NOT transcription complex subunit 6-like
Chromosome 4 · Protein coding
Primary Transcript
NM_001387838.1
mRNA · 4,218 nt
CDS: 196 → 1,764 · 14 exons
Isoforms Detected
5 transcript variants
NM_001387838, NM_144571, +3 others
All share exon 1 AUG context
Gene Function Summary
CNOT6L encodes a deadenylase component of the CCR4-NOT complex, which is the major cytoplasmic mRNA deadenylase in eukaryotes. It plays a role in mRNA turnover and translational regulation. The gene is broadly expressed across tissues with highest levels in testis, brain, and immune cells.
Translation Initiation Analysis
Primary AUG
Position 196 (CDS start)
Kozak context: gccAAUGG
AUG Codons Found
23
Total AUGs
8
In-frame
1
Strong Kozak
Targeting Window
AUG −30 → AUG +20
Centered on primary start codon, spanning Kozak context and early CDS
Exon Architecture
14 exons · AUG in exon 1 · Target region highlighted
Ranked ASO Candidates
| # | Sequence | Length | Position | Score | Risk |
|---|
NNNNNNNNNNNNNNNNNNN18nt-12██.██LOWNNNNNNNNNNNNNNNNNNN18nt-12██.██LOWNNNNNNNNNNNNNNNNNNN18nt-12██.██LOWNNNNNNNNNNNNNNNNNNN18nt-12██.██LOWNNNNNNNNNNNNNNNNNNN18nt-12██.██LOW5 ranked candidates with full sequences, thermodynamic data, and synthesis-ready formats
Multi-Layer Scoring
Each candidate is scored across multiple evidence layers:
Off-Target Screening
Transcriptome-wide specificity analysis for each candidate:
Low
Med
High
Chemistry & Synthesis-Ready Output
Recommended chemistry:
Output formats included:
/52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/.../52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/.../52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/...Full Report Includes
Frequently Asked Questions
Everything you need to know about our ASO design services
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