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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.

  1. 01

    RCKTRocket Pharmaceuticals Highlights FDA-Aligned Pivotal Phase 2 Trial and Updated Clinical Findings for RP-A501 Following Danon Disease Investor Webinar

    Source: Rocket Pharmaceuticals

  2. 02

    RAREUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million

    Source: Ultragenyx Pharmaceutical

  3. 03

    ARWRArrowhead Pharmaceuticals Granted Entry in the FDA STAR Pilot Program for Plozasiran in Severe Hypertriglyceridemia

    Source: Arrowhead Pharmaceuticals

  4. 04

    RCKTRocket Pharmaceuticals Secures Strategic Credit Facility for Up to $150 Million from Hercules Capital

    Source: Rocket Pharmaceuticals

  5. 05

    CAPRCapricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress

    Source: Capricor Therapeutics

  6. 06

    BCRXBioCryst Announces Formation of Scientific Advisory Board to Drive Rare Disease Pipeline Innovation and Growth

    Source: BioCryst Pharmaceuticals

  7. 07

    PHVSReal-World Analysis Highlighting HAE Disease Experience and Gaps in Treatment Satisfaction Published in Advances in Therapy

    Source: Pharvaris

  8. 08

    BIIBBiogen’s Litifilimab Demonstrates Rapid and Durable Efficacy in New 52-Week Phase 2 Data from Ongoing Phase 2/3 AMETHYST Study, Reinforcing Its Potential as a First-in-Class Therapy for Cutaneous Lupus Erythematosus

    Source: Biogen

  9. 09

    ABEOAbeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

    Source: Abeona Therapeutics

  10. 10

    RAREUltragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)

    Source: Ultragenyx Pharmaceutical

  11. 11

    RHHBYRoche presents new data at EURETINA, highlighting Vabysmo’s real-world effectiveness and safety across major retinal conditions

    Source: Roche Holdings AG

  12. 12

    RHHBYRoche’s giredestrant combination significantly improved progression-free survival in ER-positive advanced breast cancer in phase III evERA data published in The New England Journal of Medicine

    Source: Roche Holdings AG

  13. 13

    RHHBYRoche’s fenebrutinib is the first BTK inhibitor to receive U.S. FDA filing acceptance in both relapsing and primary progressive multiple sclerosis

    Source: Roche Holdings AG

  14. 14

    ARWRArrowhead Pharmaceuticals to Host a Webinar with Interim Clinical Data from a Phase 1/2a Study of ARO-MAPT for the Treatment of Alzheimer’s Disease and Other Tauopathies

    Source: Arrowhead Pharmaceuticals

  15. 15

    ARWRArrowhead Pharmaceuticals to Host a Webinar with Interim Clinical Data from a Phase 1/2a Study of ARO-MAPT for the Treatment of Alzheimer’s Disease and Other Tauopathies

    Source: Arrowhead Pharmaceuticals

  16. 16

    PHARPharming announces CEO transition

    Source: Pharming Group

  17. 17

    ETONEton Pharmaceuticals Announces U.S. Commercial Availability of IMPAVIDO® (miltefosine) Capsules

    Source: Eton Pharmaceuticals

  18. 18

    PTCTPTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

    Source: PTC Therapeutics, Inc.

  19. 19

    PHARPharming announces U.S. FDA acceptance and Priority Review of sNDA for lower doses of Joenja® to treat children with APDS

    Source: Pharming Group

  20. 20

    MGTXMeiraGTx Appoints Accomplished Healthcare Portfolio Manager Alan M. Sebulsky to Board of Directors

    Source: MeiraGTx

View all latest news

Companies in Rare Disease

46 companies tagged with Rare Disease.

ABEO

Abeona Therapeutics

ACAD

ACADIA Pharmaceuticals Inc.

AGLE

Aeglea Biotherapeutics

AGIO

Agios Pharmaceuticals, Inc.

ALNY

Alnylam Pharmaceuticals, Inc.

AMGN

Amgen Inc.

APLS

Apellis Pharmaceuticals

ARWR

Arrowhead Pharmaceuticals

AZN

AstraZeneca PLC

AVRO

AVROBIO

BCRX

BioCryst Pharmaceuticals

BDRX

Biodexa Pharmaceuticals

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.