# Rare Disease Sector

Canonical URL: https://biopharmsignal.com/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.

Companies currently grouped under Rare Disease: 47

## Recent Sector News

- 2026-10-07 13:36 EST — RCKT — [Rocket Pharmaceuticals Highlights FDA-Aligned Pivotal Phase 2 Trial and Updated Clinical Findings for RP-A501 Following Danon Disease Investor Webinar](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-highlights-fda-aligned-pivotal-phase-2-trial-and-updated--fc3517ad) — Source: Rocket Pharmaceuticals
- 2026-10-07 12:30 EST — RARE — [Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million](https://biopharmsignal.com/news/RARE/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review--e9651c18) — Source: Ultragenyx Pharmaceutical
- 2026-10-07 11:30 EST — ARWR — [Arrowhead Pharmaceuticals Granted Entry in the FDA STAR Pilot Program for Plozasiran in Severe Hypertriglyceridemia](https://biopharmsignal.com/news/ARWR/arrowhead-pharmaceuticals-granted-entry-in-the-fda-star-pilot-program-for-plozas-a339837f) — Source: Arrowhead Pharmaceuticals
- 2026-10-06 20:12 EST — RCKT — [Rocket Pharmaceuticals Secures Strategic Credit Facility for Up to $150 Million from Hercules Capital](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-secures-strategic-credit-facility-for-up-to-150-million-f-92e85216) — Source: Rocket Pharmaceuticals
- 2026-10-05 13:00 EST — CAPR — [Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress](https://biopharmsignal.com/news/CAPR/capricor-therapeutics-presents-positive-hope-3-open-label-extension-data-on-uppe-661c1be7) — Source: Capricor Therapeutics
- 2026-10-05 11:00 EST — BCRX — [BioCryst Announces Formation of Scientific Advisory Board to Drive Rare Disease Pipeline Innovation and Growth](https://biopharmsignal.com/news/BCRX/biocryst-announces-formation-of-scientific-advisory-board-to-drive-rare-disease--16991f5e) — Source: BioCryst Pharmaceuticals
- 2026-10-05 10:50 EST — PHVS — [Real-World Analysis Highlighting HAE Disease Experience and Gaps in Treatment Satisfaction Published in Advances in Therapy](https://biopharmsignal.com/news/PHVS/real-world-analysis-highlighting-hae-disease-experience-and-gaps-in-treatment-sa-30d14e71) — Source: Pharvaris
- 2026-10-02 15:20 EST — BIIB — [Biogen’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](https://biopharmsignal.com/news/BIIB/biogen-s-litifilimab-demonstrates-rapid-and-durable-efficacy-in-new-52-week-phas-bb06832e) — Source: Biogen
- 2026-10-02 11:30 EST — ABEO — [Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/ABEO/abeona-therapeutics-announces-new-employee-inducement-grants-under-nasdaq-listin-7f2926a6) — Source: Abeona Therapeutics
- 2026-10-02 06:00 EST — RARE — [Ultragenyx 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)](https://biopharmsignal.com/news/RARE/ultragenyx-announces-marketing-authorisation-application-maa-submission-to-the-e-1ad2a236) — Source: Ultragenyx Pharmaceutical
- 2026-10-01 06:00 EST — RHHBY — [Roche presents new data at EURETINA, highlighting Vabysmo’s real-world effectiveness and safety across major retinal conditions](https://biopharmsignal.com/news/RHHBY/roche-presents-new-data-at-euretina-highlighting-vabysmo-s-real-world-effectiven-85eb6858) — Source: Roche Holdings AG
- 2026-10-01 05:00 EST — RHHBY — [Roche’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](https://biopharmsignal.com/news/RHHBY/roche-s-giredestrant-combination-significantly-improved-progression-free-surviva-366d7079) — Source: Roche Holdings AG
- 2026-09-30 05:00 EST — RHHBY — [Roche’s fenebrutinib is the first BTK inhibitor to receive U.S. FDA filing acceptance in both relapsing and primary progressive multiple sclerosis](https://biopharmsignal.com/news/RHHBY/roche-s-fenebrutinib-is-the-first-btk-inhibitor-to-receive-u-s-fda-filing-accept-58ab83d7) — Source: Roche Holdings AG
- 2026-09-29 11:30 EST — ARWR — [Arrowhead 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](https://biopharmsignal.com/news/ARWR/arrowhead-pharmaceuticals-to-host-a-webinar-with-interim-clinical-data-from-a-ph-3d5934b8) — Source: Arrowhead Pharmaceuticals
- 2026-09-29 11:30 EST — ARWR — [Arrowhead 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](https://biopharmsignal.com/news/ARWR/arrowhead-pharmaceuticals-to-host-a-webinar-with-interim-clinical-data-from-a-ph-d53ad7a1) — Source: Arrowhead Pharmaceuticals
- 2026-09-29 05:00 EST — PHAR — [Pharming announces CEO transition](https://biopharmsignal.com/news/PHAR/pharming-announces-ceo-transition-c241aabd) — Source: Pharming Group
- 2026-09-28 10:50 EST — ETON — [Eton Pharmaceuticals Announces U.S. Commercial Availability of IMPAVIDO® (miltefosine) Capsules](https://biopharmsignal.com/news/ETON/eton-pharmaceuticals-announces-u-s-commercial-availability-of-impavido-miltefosi-a43e7fef) — Source: Eton Pharmaceuticals
- 2026-09-25 20:30 EST — PTCT — [PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/PTCT/ptc-therapeutics-reports-inducement-grants-under-nasdaq-listing-rule-5635-c-4-21999412) — Source: PTC Therapeutics, Inc.
- 2026-09-25 14:30 EST — PHAR — [Pharming announces U.S. FDA acceptance and Priority Review of sNDA for lower doses of Joenja® to treat children with APDS](https://biopharmsignal.com/news/PHAR/pharming-announces-u-s-fda-acceptance-and-priority-review-of-snda-for-lower-dose-4039ea3c) — Source: Pharming Group
- 2026-09-23 12:00 EST — MGTX — [MeiraGTx Appoints Accomplished Healthcare Portfolio Manager Alan M. Sebulsky to Board of Directors](https://biopharmsignal.com/news/MGTX/meiragtx-appoints-accomplished-healthcare-portfolio-manager-alan-m-sebulsky-to-b-7e549edb) — Source: MeiraGTx

## Companies

- [ABEO](https://biopharmsignal.com/company/ABEO) — Abeona Therapeutics
- [ACAD](https://biopharmsignal.com/company/ACAD) — ACADIA Pharmaceuticals Inc.
- [AGLE](https://biopharmsignal.com/company/AGLE) — Aeglea Biotherapeutics
- [AGIO](https://biopharmsignal.com/company/AGIO) — Agios Pharmaceuticals, Inc.
- [ALNY](https://biopharmsignal.com/company/ALNY) — Alnylam Pharmaceuticals, Inc.
- [AMGN](https://biopharmsignal.com/company/AMGN) — Amgen Inc.
- [APLS](https://biopharmsignal.com/company/APLS) — Apellis Pharmaceuticals
- [APLT](https://biopharmsignal.com/company/APLT) — Applied Therapeutics
- [ARWR](https://biopharmsignal.com/company/ARWR) — Arrowhead Pharmaceuticals
- [AZN](https://biopharmsignal.com/company/AZN) — AstraZeneca PLC
- [AVRO](https://biopharmsignal.com/company/AVRO) — AVROBIO
- [BCRX](https://biopharmsignal.com/company/BCRX) — BioCryst Pharmaceuticals
- [BDRX](https://biopharmsignal.com/company/BDRX) — Biodexa Pharmaceuticals
- [BIIB](https://biopharmsignal.com/company/BIIB) — Biogen
- [BMRN](https://biopharmsignal.com/company/BMRN) — BioMarin Pharmaceutical
- [CALT](https://biopharmsignal.com/company/CALT) — Calliditas Therapeutics AB
- [CAPR](https://biopharmsignal.com/company/CAPR) — Capricor Therapeutics
- [CCBS](https://biopharmsignal.com/company/CCBS) — Castle Creek Biosciences
- [CRNX](https://biopharmsignal.com/company/CRNX) — Crinetics Pharmaceuticals
- [CYTH](https://biopharmsignal.com/company/CYTH) — Cyclo Therapeutics
- [EDIT](https://biopharmsignal.com/company/EDIT) — Editas Medicine
- [EGTX](https://biopharmsignal.com/company/EGTX) — Egetis
- [ELOX](https://biopharmsignal.com/company/ELOX) — Eloxx
- [ETON](https://biopharmsignal.com/company/ETON) — Eton Pharmaceuticals
- [FRLN](https://biopharmsignal.com/company/FRLN) — Freeline Therapeutics
- [KRYS](https://biopharmsignal.com/company/KRYS) — Krystal Biotech
- [MGTX](https://biopharmsignal.com/company/MGTX) — MeiraGTx
- [MNPR](https://biopharmsignal.com/company/MNPR) — Monopar
- [OMER](https://biopharmsignal.com/company/OMER) — Omeros
- [ORPH](https://biopharmsignal.com/company/ORPH) — Orphazyme
- [PFE](https://biopharmsignal.com/company/PFE) — Pfizer, Inc.
- [PHAR](https://biopharmsignal.com/company/PHAR) — Pharming Group
- [PHVS](https://biopharmsignal.com/company/PHVS) — Pharvaris
- [TARA](https://biopharmsignal.com/company/TARA) — Protara Therapeutics
- [PTCT](https://biopharmsignal.com/company/PTCT) — PTC Therapeutics, Inc.
- [QBIO](https://biopharmsignal.com/company/QBIO) — Q BioMed
- [RLYB](https://biopharmsignal.com/company/RLYB) — Rallybio
- [RGNX](https://biopharmsignal.com/company/RGNX) — REGENXBIO Inc.
- [RGLS](https://biopharmsignal.com/company/RGLS) — Regulus Therapeutics
- [RHHBY](https://biopharmsignal.com/company/RHHBY) — Roche Holdings AG
- [RCKT](https://biopharmsignal.com/company/RCKT) — Rocket Pharmaceuticals
- [SEEL](https://biopharmsignal.com/company/SEEL) — Seelos Therapeutics, Inc.
- [SLNO](https://biopharmsignal.com/company/SLNO) — Soleno Therapeutics
- [MITO](https://biopharmsignal.com/company/MITO) — Stealth BioTherapeutics
- [TVTX](https://biopharmsignal.com/company/TVTX) — Travere Therapeutics
- [RARE](https://biopharmsignal.com/company/RARE) — Ultragenyx Pharmaceutical
- [QURE](https://biopharmsignal.com/company/QURE) — uniQure N.V.