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Rare Diseases '25: Gene Therapy & Genetic Med
7 tickers · $23.94B total market cap
Group: Rare Diseases
Gene therapy is entering a commercial breakthrough phase for rare diseases, backed by regulatory flexibility, a Medicare out-of-pocket revolution, and maturing delivery science. Investors are positioned to benefit from both binary upside and increased durability of rare disease franchises.
Rare Diseases '25: Gene Therapy & Genetic Med tracks 7 publicly listed stocks in this theme: KRYS, NTLA, QURE, RARE, SLNO, SRPT, SRRK. See the constituent table for details.
Thesis update: 2025-04-01
Constituents
Build 20260520T081747
| Company | Previous Quarter Report Date | Next Expected Report Date | Last Quarter Earnings Move % | Earnings Move % | Intra-Quarter Move % | Since Last Report % | Market cap | Weight |
|---|---|---|---|---|---|---|---|---|
Krystal Biotech, Inc.KRYS | 05/04/2026 (BMO) | 05/04/2026 (BMO) | 2.47% | 6.97% | -6.09% | 3.07% | $8.65B | 14.2857 |
Scholar Rock Holding CorporationSRRK | 05/07/2026 (BMO) | 05/07/2026 (BMO) | 9.08% | 2.72% | -0.67% | -1.72% | $5.70B | 14.2857 |
Soleno Therapeutics, Inc.SLNO | 05/07/2026 (AMC) | 05/07/2026 (AMC) | -4.73% | 0.02% | 35.53% | 0.11% | $2.76B | 14.2857 |
Ultragenyx Pharmaceutical Inc.RARE | 05/05/2026 (AMC) | 05/05/2026 (AMC) | -12.68% | 4.51% | 21.88% | -13.42% | $2.21B | 14.2857 |
Sarepta Therapeutics, Inc.SRPT | 05/06/2026 (AMC) | 05/06/2026 (AMC) | -11.65% | -4.54% | 37.41% | -20.00% | $1.76B | 14.2857 |
uniQure N.V.QURE | 05/05/2026 (BMO) | 05/05/2026 (BMO) | -42.23% | 10.35% | 129.01% | 8.19% | $1.52B | 14.2857 |
Intellia Therapeutics, Inc.NTLA | 05/11/2026 (BMO) | 05/11/2026 (BMO) | 2.68% | -0.71% | 2.25% | -14.65% | $1.34B | 14.2857 |
| Average | -8.15% | 2.76% | 31.33% | -5.49% | $3.42B | 14.2857 | ||
| Median | -4.73% | 2.72% | 21.88% | -1.72% | $2.21B | 14.2857 | ||
| Std Dev | 15.73% | 4.65% | 43.02% | 9.74% | $2.54B | 0.0% | ||
| Min | -42.23% | -4.54% | -6.09% | -20.00% | $1.34B | 14.2857 | ||
| Max | 9.08% | 10.35% | 129.01% | 8.19% | $8.65B | 14.2857 | ||
| % Positive Tickers | 42.86% | 71.43% | 71.43% | 42.86% |
* Provisional value: before LstRpt% reaches its 2-day post-report lock window (BMO/AMC adjusted), EarningsPerf is calculated from current vs pre-report and then locks to final LstRpt%.
