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CAM-101
CAM-101 is a gene therapy that delivers a functional copy of the CDKN1C gene to treat Beckwith-Wiedemann syndrome and related imprinting disorders.
CAM-101 is a gene therapy that delivers a functional copy of the CDKN1C gene to treat Beckwith-Wiedemann syndrome and related imprinting disorders. Used for Beckwith-Wiedemann syndrome, CDKN1C-related imprinting disorders.
At a glance
| Generic name | CAM-101 |
|---|---|
| Also known as | Elate Ocular, FD hPL |
| Sponsor | Cambium Bio Limited |
| Drug class | Gene therapy (AAV-based) |
| Target | CDKN1C gene / p57 protein |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disorders / Oncology |
| Phase | Phase 3 |
Mechanism of action
CAM-101 uses an adeno-associated virus (AAV) vector to introduce a working CDKN1C gene into patients with loss-of-function mutations or epigenetic defects affecting this tumor suppressor gene. CDKN1C encodes p57, a cyclin-dependent kinase inhibitor critical for growth regulation and genomic imprinting. By restoring functional p57 expression, the therapy aims to normalize cell growth control and reduce overgrowth and cancer predisposition associated with imprinting disorders.
Approved indications
- Beckwith-Wiedemann syndrome
- CDKN1C-related imprinting disorders
Common side effects
- Injection site reactions
- Transient liver enzyme elevation
- Immune responses to AAV vector
Key clinical trials
- Phase III Study of Topical Fibrinogen-Depleted Human Platelet Lysate Compared to Placebo for Moderate to Severe Dry Eye (PHASE3)
- Topical Fibrinogen-Depleted Human Platelet Lysate in Patients With Dry Eye Secondary to Graft vs. Host Disease (PHASE1, PHASE2)
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 |