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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

    ABEOAbeona Therapeutics® Congratulates Ultragenyx on U.S. FDA Approval of FAYUVI™ Gene Therapy for Sanfilippo Syndrome Type A (MPS IIIA)

    Source: Abeona Therapeutics

  2. 02

    BIIBPhase 3 Pediatric EMPAVELI Data Published in Clinical Journal of the American Society of Nephrology Show Reduction in Proteinuria and Stabilized Kidney Function in Adolescents with C3G or Primary IC-MPGN

    Source: Biogen

  3. 03

    RAREUltragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

    Source: Ultragenyx Pharmaceutical

  4. 04

    CAPRCapricor Therapeutics to Present HOPE-3 and HOPE-3 Open-Label Extension Data at 2026 World Muscle Society Congress

    Source: Capricor Therapeutics

  5. 05

    ABEOAbeona Therapeutics® Announces Activation of University of Florida Health as Newest Qualified Treatment Center for ZEVASKYN®

    Source: Abeona Therapeutics

  6. 06

    BIIBLEQEMBI® Pen (Subcutaneous Formulation of LEQEMBI®) Approved in Japan for the Treatment of Early Alzheimer’s Disease

    Source: Biogen

  7. 07

    BDRXBiodexa Enters Into Warrant Exercise Transaction for $2.3 Million in Gross Proceeds

    Source: Biodexa Pharmaceuticals

  8. 08

    ARWRArrowhead Pharmaceuticals Announces Interim Topline Clinical Data for the First Dual-Functional RNAi Therapeutic for the Treatment of Mixed Hyperlipidemia

    Source: Arrowhead Pharmaceuticals

  9. 09

    RCKTRocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease

    Source: Rocket Pharmaceuticals

  10. 10

    BDRXInterim results for the six months ended June 30, 2026

    Source: Biodexa Pharmaceuticals

  11. 11

    PHARFDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.

    Source: Pharming Group

  12. 12

    EDITEditas Medicine Reports Inducement Grants to New Chief Medical Officer

    Source: Editas Medicine

  13. 13

    PHARPharming Group to participate in September investor conferences

    Source: Pharming Group

  14. 14

    TARAProtara Therapeutics to Present at the H.C. Wainwright 28th Annual Global Investment Conference

    Source: Protara Therapeutics

  15. 15

    EDITEditas Medicine Appoints Dan Ory, M.D., as Chief Medical Officer

    Source: Editas Medicine

  16. 16

    PHVSPharvaris Announces Positive Topline Data from CHAPTER-3 Pivotal Study of Deucrictibant XR for Prophylaxis of HAE Attacks

    Source: Pharvaris

  17. 17

    BIIBStoke Therapeutics and Biogen Present Long-Term Clinical Data that Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 16th European Epilepsy Congress (EEC)

    Source: Biogen

  18. 18

    KRYSKrystal Biotech to Present at 2026 Cantor Global Healthcare Conference

    Source: Krystal Biotech

  19. 19

    ARWRArrowhead Pharmaceuticals to Participate in Upcoming September 2026 Investor Conferences

    Source: Arrowhead Pharmaceuticals

  20. 20

    RCKTRocket Pharmaceuticals to Participate in Upcoming Investor Conferences

    Source: Rocket Pharmaceuticals

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

APLT

Applied Therapeutics

ARWR

Arrowhead Pharmaceuticals

AZN

AstraZeneca PLC

AVRO

AVROBIO

BCRX

BioCryst 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.