Rare Disease Programs
Advancing Hope in Rare Disease Through Precision Oligonucleotide Development.
Empowering innovators with fast, flexible, and highly customized oligonucleotide synthesis and screening for life-changing therapies.
The Urgency of Rare Disease Innovation
More than 6,000-10,000 rare diseases have been identified, many with onset in childhood, leaving millions of families facing progressive,
debilitating, and often life-threatening conditions. Advances in personalized N of 1 therapies, including patient specific antisense
oligonucleotides (ASOs) designed for unique mutations, now make it possible to develop and deliver nucleic acid therapeutics (NATs) on
clinically meaningful timelines for patients with ultra rare genetic variants.
As genomic sequencing uncovers a growing number of actionable mutations, the urgency to develop therapies continues to rise.
Synoligo is proud to support this critical work, providing fast, precise oligo manufacturing and high throughput screening
to help turn discoveries into potential therapies without delay.
How Synoligo Supports Rare Disease Therapeutics
At Synoligo, we partner closely with rare‑disease innovators from academic researchers to biotech startups and clinical teams providing:
Rapid Custom Oligo Synthesis
- Highly modified oligos, including ASOs, PMOs, siRNA, saRNA, and gRNA
- Ultra-fast turnaround and flexible scales for preclinical needs
- 700+ available modifications for therapeutic optimization
High-Throughput Screening for Therapeutic Candidates
- Our state-of-the-art screening lab adjacent to our synthesis facility
- Automation-driven evaluation of potency, selectivity and toxicity
- Working with iPSC models
- Fast data delivery to accelerate go/no-go decisions
End-to-End Support for Accelerated Timelines
- Close scientific collaboration
- Scalability from early discovery to preclinical studies
- Expertise in complex and 'non-standard' modified oligos
Advancing rare disease therapies requires speed, precision, and unwavering commitment.
We are here to be your scientific partner every step of the way.
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