# Ascidian Therapeutics

> Ascidian Therapeutics is a clinical-stage biotechnology company developing RNA exon editors that replace mutated sections of genetic messages without directly editing DNA. Its lead investigational candidate, ACDN-01, is in the Phase 1/2 STELLAR study for Stargardt disease and other ABCA4 retinopathies; Roche and Lilly have licensed its technology for selected neurological and kidney-disease targets.

- **Founded:** 2020
- **Headquarters:** Boston, United States
- **Founders:** Michael Ehlers (Founder and founding CEO; board chair as of July 2026)
- **Products:** RNA exon-editing platform, ACDN-01, STARPATH, MSH3 splice modulation research
- **Notable:** Launched publicly in October 2022 with $50 million in Series A financing., Obtained FDA IND clearance for ACDN-01 in 2024 and began Phase 1/2 clinical development., Named to the 2024 Endpoints 11 list.

## Products & services

- **RNA exon-editing platform** — Discovery platform using computational RNA design, high-throughput molecular biology and sequencing to create synthetic RNA constructs that replace multiple contiguous exons through pre-mRNA trans-splicing. Licensed on a target-specific basis to partners.
- **ACDN-01** — Investigational single-AAV RNA exon editor for Stargardt disease and other ABCA4 retinopathies, delivered by one subretinal injection in the Phase 1/2 STELLAR study. Not an approved medicine.
- **STARPATH** — Observational prescreening study providing genetic testing and retinal imaging to assess potential eligibility for future ACDN-01 trials; it does not administer treatment.
- **MSH3 splice modulation research** — Preclinical research into repeat expansion disorders, discussed at the 2025 Huntington’s Disease Therapeutics Conference and ASGCT 2026.

## Achievements

- Launched publicly in October 2022 with $50 million in Series A financing.
- Obtained FDA IND clearance for ACDN-01 in 2024 and began Phase 1/2 clinical development.
- Named to the 2024 Endpoints 11 list.
- Completed STELLAR adult dose escalation in May 2026 and expanded to pediatric enrollment.
- Presented first-in-human ACDN-01 safety data at ASGCT in May 2026.
- Secured target-specific research collaborations with Roche and Lilly.

## Latest updates

- **2026-07** — Daniel Rosan appointed president while retaining CFO and CBO roles; founding CEO Michael Ehlers stepped down as CEO and remained board chair during the CEO succession process.
- **2026-06** — Lilly collaboration announced for undisclosed inherited kidney disease targets.
- **2026-05** — Completed adult STELLAR dose escalation, expanded pediatric enrollment and presented first-in-human safety data at ASGCT.
- **2026-01** — Announced ACDN-01 manufacturing partnership with Forge Biologics.

## Links

- Website: https://ascidian-tx.com/
- LinkedIn: https://www.linkedin.com/company/ascidian-therapeutics
- Twitter/X: https://twitter.com/AscidianTx
- Bluesky: https://bsky.app/profile/ascidiantx.bsky.social

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Profile page: https://yespress.io/ascidian-therapeutics
Published by YesPress — https://yespress.io
Last updated: 2026-10-04