Rare & Genetic Disease
Companies whose principal business activity is classified as “Biotech - Rare & Genetic Disease”.
Scope: Disease-area basket spanning many modalities.
Leadership as of 2026-10-27: Lagging. Relative to SPY: 5 sessions +0.8pp, 21 sessions −3.6pp, 63 sessions −17.0pp. Participation 32%; 18 of 20 members eligible.
- 21-session excess -3.7pp
- 63-session excess -18.8pp (robust)
Economic family: Biotechnology
Member companies
- Agios Pharmaceuticals, Inc. (AGIO)
- Disc Medicine, Inc. (IRON)
- Travere Therapeutics, Inc. (TVTX)
- Sarepta Therapeutics, Inc. (SRPT)
- BridgeBio Pharma, Inc. (BBIO)
- Damora Therapeutics, Inc. (DMRA)
- BioCryst Pharmaceuticals, Inc. (BCRX)
- BioMarin Pharmaceutical Inc. (BMRN)
- Savara, Inc. (SVRA)
- Scholar Rock Holding Corporatio (SRRK)
- Rhythm Pharmaceuticals, Inc. (RYTM)
- Mirum Pharmaceuticals, Inc. (MIRM)
- Monopar Therapeutics Inc. (MNPR)
- PTC Therapeutics, Inc. (PTCT)
- Ultragenyx Pharmaceutical Inc. (RARE)
- Insmed Incorporated (INSM)
- Capricor Therapeutics, Inc. (CAPR)
- Hemab Therapeutics Holdings, In (COAG)
- Design Therapeutics, Inc. (DSGN)
- Palvella Therapeutics, Inc. (PVLA)
Related themes
- Large-Cap Biopharma: Leading — 21-session vs SPY +6.1pp, participation 75%
- Neuro / CNS: Neutral — 21-session vs SPY −0.2pp, participation 43%
- Molecular Diagnostics: Weakening — 21-session vs SPY +15.9pp, participation 80%
- Immunology & Inflammation: Lagging — 21-session vs SPY −3.6pp, participation 29%
- RNA Therapeutics: Lagging — 21-session vs SPY −4.2pp, participation 40%
- Oncology: Lagging — 21-session vs SPY −8.2pp, participation 32%
- Gene Therapy: Lagging — 21-session vs SPY −16.6pp, participation 0%
- Targeted Protein Degradation: Tracked (too few stocks for a leadership state) — 21-session vs SPY +2.4pp, participation 67%