CERC-801
Phase 1- Indications
- PGM1 Deficiency
Company intelligence / Profile preview
Ichorion Therapeutics
New York, New York, United Statespitchbook.com ↗
- Ownership
- Private
- Development stage
- Phase 1
01
Overview
Ichorion developed a pipeline of genetically-targeted therapies for rare pediatric and orphan metabolic disorders. At acquisition, its lead program targeted DGUOK for mitochondrial diseases and was in preclinical development. Two programs had received Rare Pediatric Disease Designation and were eligible for Priority Review Vouchers upon approval. The company’s approach included leveraging the 505(b)(2) regulatory pathway to potentially expedite development timelines
- Therapeutic areas
- Rare Diseases
- Modalities
- Small molecule drugs
- Industry
- Biotech
02
Drug pipeline
7 assets6 more assets in the full pipeline.
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Partnerships
No major external partnerships disclosed prior to acquisition; now part of Cerecor/Avalo’s portfolio.
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