Ascidian Therapeutics
Clinical-stage biotechnology company developing RNA editing therapeutics for inherited retinal diseases and other genetic conditions, using proprietary ADAR-mediated RNA editing technology.
Notes
Ascidian Therapeutics is a clinical-stage biotechnology company pioneering the development of RNA editing therapeutics for genetic diseases. The company's proprietary technology harnesses endogenous ADAR (adenosine deaminase acting on RNA) enzymes to correct disease-causing mutations at the RNA level without permanently altering the genome.
Ascidian's lead program targets Stargardt disease, an inherited retinal dystrophy that causes progressive vision loss. By correcting the mutation at the RNA level, the company aims to restore functional protein production while avoiding the permanent changes associated with DNA editing.
The company's RNA editing platform offers potential advantages over DNA-based gene therapy approaches, including reversibility, use of endogenous cellular machinery, and applicability to larger genes that are challenging for traditional gene therapy vectors.
Team
- David Locke - Chief Executive Officer
- LinkedIn: linkedin.com/in/davidlocke
- John Mattison, M.D. - Chief Medical Officer
- Joshua Rosenthal, Ph.D. - Scientific Founder
- Marine Biological Laboratory researcher
Additional Research Findings
- Lead program for Stargardt disease (inherited retinal dystrophy)
- ADAR-mediated RNA editing platform
- Clinical-stage company
- Focus on inherited retinal diseases
- RNA editing approach (not DNA editing)
- Boston, Massachusetts headquarters
- Founded 2020
- Raised significant Series B funding
Sources
Investors
| Name | Location | Type | Stages | Portfolio |
|---|---|---|---|---|
| Apple Tree Partners | New York, USA | biotech-focused | series-aseries-b+2 | 23 |