Last reviewed · How we verify
MDR-101
MDR-101 is a gene therapy that delivers a functional copy of the ABCA3 gene to restore surfactant production in patients with ABCA3-deficient interstitial lung disease.
MDR-101 is a gene therapy that delivers a functional copy of the ABCA3 gene to restore surfactant production in patients with ABCA3-deficient interstitial lung disease. Used for ABCA3-deficient interstitial lung disease.
At a glance
| Generic name | MDR-101 |
|---|---|
| Sponsor | Medeor Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | ABCA3 gene |
| Modality | Biologic |
| Therapeutic area | Pulmonology / Rare Genetic Lung Disease |
| Phase | Phase 3 |
Mechanism of action
ABCA3 mutations cause defective surfactant metabolism, leading to progressive lung disease. MDR-101 uses an adeno-associated viral (AAV) vector to deliver a working ABCA3 gene directly to lung cells, enabling them to produce functional ABCA3 protein and restore normal surfactant homeostasis. This addresses the underlying genetic defect rather than treating symptoms.
Approved indications
- ABCA3-deficient interstitial lung disease
Common side effects
- Transaminitis (elevated liver enzymes)
- Immune response to AAV vector
- Respiratory adverse events
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- MDR-101 CI brief — competitive landscape report
- MDR-101 updates RSS · CI watch RSS
- Medeor Therapeutics, Inc. portfolio CI