# Rare Disease Gene Therapy Sector

Canonical URL: https://biopharmsignal.com/sector/rare-disease-gene-therapy

Rare Disease Gene Therapy is a company sector tag on BioPharmSignal. This page groups public biotech and healthcare companies associated with Rare Disease Gene Therapy, making it easier to scan related businesses, compare peers, and follow companies exposed to the same modality, disease area, or development theme.

Companies currently grouped under Rare Disease Gene Therapy: 16

## Recent Sector News

- 2026-07-09 15:43 EST — CRNX — [Crinetics and Leading Patient Groups Launch ‘Everyday Advocacy’ Initiative to Empower People Across Endocrine Disease Communities | Crinetics](https://biopharmsignal.com/news/CRNX/crinetics-and-leading-patient-groups-launch-everyday-advocacy-initiative-to-empo-2503d5d7) — Source: Crinetics Pharmaceuticals
- 2026-07-09 15:43 EST — CRNX — [Crinetics Presents Full Results From Phase 2 Trial of Atumelnant in Congenital Adrenal Hyperplasia (CAH) in Oral Presentation at ENDO 2026 | Crinetics](https://biopharmsignal.com/news/CRNX/crinetics-presents-full-results-from-phase-2-trial-of-atumelnant-in-congenital-a-cc021e52) — Source: Crinetics Pharmaceuticals
- 2026-07-06 20:10 EST — CRNX — [Vertex to Acquire Crinetics Pharmaceuticals | Crinetics](https://biopharmsignal.com/news/CRNX/vertex-to-acquire-crinetics-pharmaceuticals-crinetics-29e39d07) — Source: Crinetics Pharmaceuticals
- 2026-07-02 11:00 EST — BCRX — [BioCryst Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/BCRX/biocryst-reports-inducement-grants-under-nasdaq-listing-rule-5635-c-4-afb4cc8d) — Source: BioCryst Pharmaceuticals
- 2026-06-30 21:53 EST — CRNX — [Crinetics Pharmaceuticals to Report Second Quarter 2026 Financial Results on August 4, 2026 | Crinetics](https://biopharmsignal.com/news/CRNX/crinetics-pharmaceuticals-to-report-second-quarter-2026-financial-results-on-aug-6cad0f8c) — Source: Crinetics Pharmaceuticals
- 2026-06-30 20:25 EST — SRPT — [Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/SRPT/sarepta-therapeutics-announces-inducement-grants-under-nasdaq-listing-rule-5635--c021b8ab) — Source: Sarepta Therapeutics
- 2026-06-30 12:30 EST — SRPT — [Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®](https://biopharmsignal.com/news/SRPT/sarepta-announces-fda-acceptance-of-sndas-for-amondys-45-and-vyondys-53-40aea7f9) — Source: Sarepta Therapeutics
- 2026-06-29 11:00 EST — BCRX — [BioCryst Sharpens Scientific Focus on External Innovation with Wind Down of Internal Discovery Programs and Closure of Birmingham Research Facility](https://biopharmsignal.com/news/BCRX/biocryst-sharpens-scientific-focus-on-external-innovation-with-wind-down-of-inte-83b84c41) — Source: BioCryst Pharmaceuticals
- 2026-06-25 20:05 EST — REGN — [Regeneron to Report Second Quarter 2026 Financial and Operating Results and Host Conference Call and Webcast](https://biopharmsignal.com/news/REGN/regeneron-to-report-second-quarter-2026-financial-and-operating-results-and-host-c2e371f6) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — REGN — [Cemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG)](https://biopharmsignal.com/news/REGN/cemdisiran-regulatory-submissions-accepted-for-review-by-fda-and-ema-for-the-treatment-of-generalized-myasthenia-gravis-gmg) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — BCRX — [BioCryst Presents New Clinical Data and Real-World Evidence Demonstrating Impact of HAE Portfolio at the 2026 European Academy of Allergy and Clinical Immunology Annual Meeting](https://biopharmsignal.com/news/BCRX/biocryst-presents-new-clinical-data-and-real-world-evidence-demonstrating-impact-of-hae-portfolio-at-the-2026-european-academy-of-allergy-and-clinical-immunology-annual-meeting) — Source: BioCryst Pharmaceuticals
- 2026-06-25 13:46 EST — RCKT — [Rocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-announces-closing-of-sale-of-rare-pediatric-disease-priority-review-voucher-for-180-million) — Source: Rocket Pharmaceuticals
- 2026-06-25 13:46 EST — REGN — [Regeneron to Highlight Progress Across Its Metabolic Disease, Ophthalmology and Rare Disease Pipelines at ADA and ENDO](https://biopharmsignal.com/news/REGN/regeneron-to-highlight-progress-across-its-metabolic-disease-ophthalmology-and-rare-disease-pipelines-at-ada-and-endo) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — SRPT — [Sarepta Therapeutics to Participate in Upcoming Investor Conferences](https://biopharmsignal.com/news/SRPT/sarepta-therapeutics-to-participate-in-upcoming-investor-conferences) — Source: Sarepta Therapeutics
- 2026-06-25 13:46 EST — RLYB — [Rallybio Corporation and Avenzo Therapeutics Announce Merger Agreement to Advance Next-Generation Oncology Therapies and $215 Million Concurrent Private Placement](https://biopharmsignal.com/news/RLYB/rallybio-corporation-and-avenzo-therapeutics-announce-merger-agreement-to-advance-next-generation-oncology-therapies-and-215-million-concurrent-private-placement) — Source: Rallybio
- 2026-06-25 13:46 EST — REGN — [Regeneron’s Ebola Antibody Recommended by World Health Organization for Investigational Use in Response to Current Bundibugyo Ebolavirus Outbreak](https://biopharmsignal.com/news/REGN/regeneron-s-ebola-antibody-recommended-by-world-health-organization-for-investigational-use-in-response-to-current-bundibugyo-ebolavirus-outbreak) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — BCRX — [BioCryst to Present New HAE Data from ORLADEYO® (berotralstat) and Navenibart at the 2026 European Academy of Allergy and Clinical Immunology Annual Meeting](https://biopharmsignal.com/news/BCRX/biocryst-to-present-new-hae-data-from-orladeyor-berotralstat-and-navenibart-at-the-2026-european-academy-of-allergy-and-clinical-immunology-annual-meeting) — Source: BioCryst Pharmaceuticals
- 2026-06-25 13:46 EST — REGN — [Otarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss](https://biopharmsignal.com/news/REGN/otarmenitm-lunsotogene-parvec-receives-ema-filing-acceptance-for-genetic-hearing-loss) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — REGN — [Lynozyfic® (linvoseltamab) Monotherapy Demonstrates Deep and Rapid Responses in All Treated Patients with Second-Line-Plus Systemic Amyloid Light Chain Amyloidosis](https://biopharmsignal.com/news/REGN/lynozyficr-linvoseltamab-monotherapy-demonstrates-deep-and-rapid-responses-in-all-treated-patients-with-second-line-plus-systemic-amyloid-light-chain-amyloidosis) — Source: Regeneron Pharmaceuticals
- 2026-06-25 13:46 EST — REGN — [Regeneron Announces Strategic Collaboration with Parabilis Medicines to Advance Novel Antibody-Helicon™ Conjugates Across Multiple Therapeutic Areas](https://biopharmsignal.com/news/REGN/regeneron-announces-strategic-collaboration-with-parabilis-medicines-to-advance-novel-antibody-helicontm-conjugates-across-multiple-therapeutic-areas) — Source: Regeneron Pharmaceuticals

## Companies

- [AMGN](https://biopharmsignal.com/company/AMGN) — Amgen Inc.
- [BCRX](https://biopharmsignal.com/company/BCRX) — BioCryst Pharmaceuticals
- [CRNX](https://biopharmsignal.com/company/CRNX) — Crinetics Pharmaceuticals
- [EDIT](https://biopharmsignal.com/company/EDIT) — Editas Medicine
- [ETON](https://biopharmsignal.com/company/ETON) — Eton Pharmaceuticals
- [GILD](https://biopharmsignal.com/company/GILD) — Gilead Sciences
- [MRNA](https://biopharmsignal.com/company/MRNA) — Moderna, Inc.
- [RLYB](https://biopharmsignal.com/company/RLYB) — Rallybio
- [REGN](https://biopharmsignal.com/company/REGN) — Regeneron Pharmaceuticals
- [RCKT](https://biopharmsignal.com/company/RCKT) — Rocket Pharmaceuticals
- [SNY](https://biopharmsignal.com/company/SNY) — Sanofi
- [SRPT](https://biopharmsignal.com/company/SRPT) — Sarepta Therapeutics
- [SEEL](https://biopharmsignal.com/company/SEEL) — Seelos Therapeutics, Inc.
- [SLNO](https://biopharmsignal.com/company/SLNO) — Soleno Therapeutics
- [TVTX](https://biopharmsignal.com/company/TVTX) — Travere Therapeutics
- [RARE](https://biopharmsignal.com/company/RARE) — Ultragenyx Pharmaceutical