AIT-101
Amyotrophic Lateral Sclerosis (ALS)
PreclinicalOn Hold
Key Facts
Indication
Amyotrophic Lateral Sclerosis (ALS)
Phase
Preclinical
Status
On Hold
Company
About OrphAI Therapeutics
OrphAI Therapeutics is a private, preclinical to Phase 2 biotech founded in 2020 and headquartered in New Haven, Connecticut. The company is advancing a focused pipeline led by LAM-001, a candidate in Phase 2 for Bronchiolitis Obliterans Syndrome (BOS) and earlier stages for pulmonary hypertension-associated disorders, alongside earlier-stage oncology and neurology programs. Utilizing an AI/ML-driven discovery platform, OrphAI aims to develop life-saving drugs for niche, underserved patient populations, operating as a pre-revenue entity with a lean, experienced leadership team.
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| Drug | Company | Phase |
|---|---|---|
| NPT520-34 | Neuropore Therapies | Pre-clinical/Phase 1 |
| NUZ-001 | Neurizon Therapeutics | Phase 2/3 (Platform Trial) |
| CNM-Au8 | Clene | Phase 2/3 |
| Masitinib | AB Science | Phase 3 |
| Undisclosed Program | Zhimeng Biopharma | Clinical |
| SLS-005 | Seelos Therapeutics | Platform Trial |
| PMN442 | ProMIS Neurosciences | Preclinical |
| nAV-101 | nVector | Pre-clinical |
| ALTA-808 | Alteron Therapeutics | Preclinical |
| GM6 | Genervon Biopharmaceuticals | Phase 2 |
| Lou Gehrig’s disease | Genclis | Discovery |
| Treg-directed therapy for ALS | Iltoo Pharma | Pre-clinical |