Company Sector
Rare Disease
Rare Disease groups companies developing therapies for smaller patient populations with high unmet need. These businesses are often built around genetically defined disorders, orphan indications, and targeted commercial strategies with highly specialized clinical programs.
Why watch Rare Disease
This page combines a company index, a watch action, and the newest relevant feed items in one place so readers can monitor a biotech theme without scanning each company one by one.
Latest Rare Disease news
The latest feed items from companies currently grouped under Rare Disease.
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CRNXVertex to Acquire Crinetics Pharmaceuticals | Crinetics
Source: Crinetics Pharmaceuticals
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BCRXBioCryst Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: BioCryst Pharmaceuticals
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CRNXCrinetics Pharmaceuticals to Report Second Quarter 2026 Financial Results on August 4, 2026 | Crinetics
Source: Crinetics Pharmaceuticals
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SRPTSarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: Sarepta Therapeutics
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SRPTSarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®
Source: Sarepta Therapeutics
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REGNRegeneron to Report Second Quarter 2026 Financial and Operating Results and Host Conference Call and Webcast
Source: Regeneron Pharmaceuticals
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REGNCemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG)
Source: Regeneron Pharmaceuticals
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RCKTRocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million
Source: Rocket Pharmaceuticals
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REGNRegeneron to Highlight Progress Across Its Metabolic Disease, Ophthalmology and Rare Disease Pipelines at ADA and ENDO
Source: Regeneron Pharmaceuticals
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SRPTSarepta Therapeutics to Participate in Upcoming Investor Conferences
Source: Sarepta Therapeutics
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REGNOtarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss
Source: Regeneron Pharmaceuticals
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Companies in Rare Disease
16 companies tagged with Rare Disease.
Frequently Asked Questions
What does the Rare Disease sector include?
Rare Disease groups companies developing therapies for smaller patient populations with high unmet need. These businesses are often built around genetically defined disorders, orphan indications, and targeted commercial strategies with highly specialized clinical programs.
How many companies are currently tagged with Rare Disease?
16 companies are currently grouped under Rare Disease on BioPharmSignal.
Which companies are most visible in the Rare Disease group right now?
AMGN, BCRX, CRNX, EDIT are among the companies currently shown on this sector page.
How can I watch news for Rare Disease?
Use the Watch button on this page to subscribe to Rare Disease headlines and related company updates through your BioPharmSignal saved filters.
What kinds of updates usually appear for Rare Disease?
The livefeed typically surfaces company press releases, clinical readouts, FDA and regulatory updates, partnerships, financings, and commercialization developments tied to companies in Rare Disease.