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.
- 01
BIIBBiogen Completes Acquisition of RayThera Inc.
Source: Biogen
- 02
- 03
ABEOAbeona Therapeutics® to Host Second Quarter 2026 Financial Results Conference Call on August 13, 2026
Source: Abeona Therapeutics
- 04
- 05
EDITEditas Medicine Announces Second Quarter 2026 Results and Business Updates
Source: Editas Medicine
- 06
BCRXBioCryst Reports Second Quarter 2026 Financial Results
Source: BioCryst Pharmaceuticals
- 07
- 08
- 09
RAREUltragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Source: Ultragenyx Pharmaceutical
- 10
ARWRArrowhead Pharmaceuticals Reports Fiscal 2026 Third Quarter Results
Source: Arrowhead Pharmaceuticals
- 11
ARWRArrowhead Pharmaceuticals Acquires FDA Priority Review Voucher
Source: Arrowhead Pharmaceuticals
- 12
ABEOCMS Grants New Technology Add-On Payment (NTAP) Status for ZEVASKYN®
Source: Abeona Therapeutics
- 13
ARWRArrowhead Pharmaceuticals Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Source: Arrowhead Pharmaceuticals
- 14
CRNXCrinetics Pharmaceuticals Reports Second Quarter 2026 Financial Results and Provides Business Update
Source: Crinetics Pharmaceuticals
- 15
ABEOAbeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: Abeona Therapeutics
- 16
- 17
KRYSKrystal Biotech Announces Second Quarter 2026 Financial and Operating Results
Source: Krystal Biotech
- 18
ETONEton Pharmaceuticals to Report Second Quarter 2026 Financial Results on Thursday, August 13, 2026
Source: Eton Pharmaceuticals
- 19
PTCTPTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results
Source: PTC Therapeutics, Inc.
- 20
CAPRCapricor Therapeutics Provides Update on FDA Advisory Committee Meeting for Deramiocel
Source: Capricor Therapeutics
Companies in Rare Disease
45 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?
45 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.