Aptadir Therapeutics
Italy · Biotech · Healthtech
RNA therapeutics designed to restore expression of silenced genes
RNA molecules that target DNA methylation
Aptadir’s DiR platform is based on DNMT-interacting RNAs. Its aptaDiR molecules bind and inhibit DNMT1, while captaDiR fuses the RNA inhibitor to a gene-specific sequence to target DNA methylation at an individual locus. The approach aims to reactivate genes that have been silenced in disease, rather than broadly altering methylation across the genome.
A pipeline led by Fragile X syndrome
The company’s lead investigational program, CAP1-FMR1, is being developed for Fragile X syndrome, where the FMR1 gene is silenced. Aptadir’s pipeline also covers other rare genetic disorders and cancers. Investor and company materials describe the company as preclinical; these are development-stage therapeutics, not approved or clinically available treatments.
- Platform
- DNMT-interacting RNAs (DiRs)
- Lead program
- CAP1-FMR1 for Fragile X syndrome
- Stage
- Preclinical
Funding history
1 round · 1 with disclosed amounts| Announced | Round | Amount | Investors |
|---|---|---|---|
| Seed | €40M | 4BIO Capital — LeadAngelini Ventures — ParticipantBio Lombardia Fund — ParticipantCDP Venture Capital — ParticipantCE-Ventures — ParticipantClub degli Investitori — ParticipantEXTEND Initiative — ParticipantIndaco Bio — ParticipantItalian Angels for Biotech — ParticipantKerna Ventures — ParticipantXgen Ventures — Participant |