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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KRYSKrystal Biotech to Present at 2026 Cantor Global Healthcare Conference
Source: Krystal Biotech
- 03
ARWRArrowhead Pharmaceuticals to Participate in Upcoming September 2026 Investor Conferences
Source: Arrowhead Pharmaceuticals
- 04
RCKTRocket Pharmaceuticals to Participate in Upcoming Investor Conferences
Source: Rocket Pharmaceuticals
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RAREUltragenyx Announces Phase 3 Aspire results in Angelman Syndrome
Source: Ultragenyx Pharmaceutical
- 08
ABEOAbeona Therapeutics® Announces Participation in Upcoming Investor Conferences
Source: Abeona Therapeutics
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EDITEditas Medicine to Participate in Upcoming Investor Conferences
Source: Editas Medicine
- 11
ABEOAbeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: Abeona Therapeutics
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BCRXBioCryst to Present at Upcoming Investor Conference
Source: BioCryst Pharmaceuticals
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PTCTPTC Therapeutics to Participate in Upcoming Investor Conferences
Source: PTC Therapeutics, Inc.
- 17
ETONEton Pharmaceuticals to Participate at 2026 Wells Fargo Healthcare Conference
Source: Eton Pharmaceuticals
- 18
RAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Source: Ultragenyx Pharmaceutical
- 19
CAPRCapricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA
Source: Capricor Therapeutics
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Companies in Rare Disease
46 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?
46 companies are currently grouped under Rare Disease on BioPharmSignal.
Which companies are most visible in the Rare Disease group right now?
ABEO, ACAD, AGLE, AGIO 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.