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.

$6.3B
ASO Market 2026
Roots Analysis est.
~8%
CAGR Growth
2026–2035 projection
10+
FDA-Approved ASOs
20 total oligo drugs (FDA/EMA)
200+
ASOs in Development
Clinical & preclinical pipeline

Sources: Roots Analysis, Antisense Oligonucleotides Market (Dec 2025); FDA/EMA approval records as of Mar 2024.

New to ASOs?Find out what they are →

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.

The Science Behind What We Do

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.

Expert Methodology

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.

1

Analyzes molecular targets through multiple biological dimensions

2

Identifies optimal intervention points based on mechanistic criteria

3

Designs optimized antisense sequences using expert biological rules

4

Evaluates candidates for efficacy and safety parameters

5

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 Story

Our 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

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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
Perfect for feasibility studies and pilot projects
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MOST POPULAR

Standard Design

Comprehensive ASO development

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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
Solid foundation to get your project going
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Advanced Consulting

End-to-end project partnership

Get in Touch

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
Best chances for your project to jumpstart into results
Schedule Consultation

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 Hub

RNA Structure Viewer

Predict RNA secondary structure and accessibility across a region. Overlay candidate ASO binding sites with accessibility metrics and ΔG.

View Structure

Isoform Explorer

Compare isoforms side-by-side. Visualize exon inclusion patterns, CDS/UTR differences, and isoform-specific target windows for ASOs.

Explore Isoforms

One 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 — Founder

Jose Stigliano, PhD

Founder & Chief Scientific Officer

RNA TherapeuticsSplicingEpigeneticsCell (Cover)SMACSHL
PhD, Kornblihtt Lab — UBA/CONICET
BSc Molecular Biology — University of Dundee
Collaborated with Adrian Krainer (Spinraza)
Industry: Biotechnology & molecular engineering

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.

SAMPLE DELIVERABLE

What 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

ASOwalker™Sample

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

Strong Kozak

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

#SequenceLengthPositionScoreRisk
#1NNNNNNNNNNNNNNNNNNN18nt-12██.██LOW
#2NNNNNNNNNNNNNNNNNNN18nt-12██.██LOW
#3NNNNNNNNNNNNNNNNNNN18nt-12██.██LOW
#4NNNNNNNNNNNNNNNNNNN18nt-12██.██LOW
#5NNNNNNNNNNNNNNNNNNN18nt-12██.██LOW
Sequences & scores available in full report

5 ranked candidates with full sequences, thermodynamic data, and synthesis-ready formats

Multi-Layer Scoring

Each candidate is scored across multiple evidence layers:

Biophysical and thermodynamic evaluation
RNA structure and target context
Safety and liability screening
Mechanism-of-action alignment
Layer 1
██%
Layer 2
██%
Layer 3
██%
Proprietary scoring

Off-Target Screening

Transcriptome-wide specificity analysis for each candidate:

Transcriptome-wide sequence specificity search
Risk stratification of potential off-targets
Biological context assessment
Clear risk classification per candidate

Low

Med

High

Risk data in full report

Chemistry & Synthesis-Ready Output

Recommended chemistry:

2'-MOEPS backboneFully modifiedSteric blocker

Output formats included:

IDTDharmaconASCIIBenchling
ASO-1/52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/...
ASO-2/52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/...
ASO-3/52MOErN/*/i2MOErN/*/i2MOErN/*/i2MOErN/...
Vendor sequences in full report

Full Report Includes

Complete ASO sequences with rationale for each
Thermodynamic binding data (Tm, ΔG, accessibility)
Evidence-based candidate ranking with explanations
Transcriptome-wide off-target risk report
Vendor-ready sequences (IDT, Dharmacon, etc.)
Chemistry optimization recommendations
Experimental validation strategy
Scientific rationale document
This preview shows the structure and gene context of a real report. Sequences, scores, and proprietary analysis are provided exclusively to clients.
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Frequently Asked Questions

Everything you need to know about our ASO design services

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