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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

CompanyPrevious Quarter Report DateNext Expected Report DateLast Quarter Earnings Move %Earnings Move %Intra-Quarter Move %Since Last Report %Market capWeight
Krystal Biotech, Inc.KRYS
05/04/2026 (BMO)05/04/2026 (BMO)2.47%6.97%-6.09%3.07%$8.65B14.2857
Scholar Rock Holding CorporationSRRK
05/07/2026 (BMO)05/07/2026 (BMO)9.08%2.72%-0.67%-1.72%$5.70B14.2857
Soleno Therapeutics, Inc.SLNO
05/07/2026 (AMC)05/07/2026 (AMC)-4.73%0.02%35.53%0.11%$2.76B14.2857
Ultragenyx Pharmaceutical Inc.RARE
05/05/2026 (AMC)05/05/2026 (AMC)-12.68%4.51%21.88%-13.42%$2.21B14.2857
Sarepta Therapeutics, Inc.SRPT
05/06/2026 (AMC)05/06/2026 (AMC)-11.65%-4.54%37.41%-20.00%$1.76B14.2857
uniQure N.V.QURE
05/05/2026 (BMO)05/05/2026 (BMO)-42.23%10.35%129.01%8.19%$1.52B14.2857
Intellia Therapeutics, Inc.NTLA
05/11/2026 (BMO)05/11/2026 (BMO)2.68%-0.71%2.25%-14.65%$1.34B14.2857
Average-8.15%2.76%31.33%-5.49%$3.42B14.2857
Median-4.73%2.72%21.88%-1.72%$2.21B14.2857
Std Dev15.73%4.65%43.02%9.74%$2.54B0.0%
Min-42.23%-4.54%-6.09%-20.00%$1.34B14.2857
Max9.08%10.35%129.01%8.19%$8.65B14.2857
% Positive Tickers42.86%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%.

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