# 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: 11

## Recent Sector News

- 2026-09-03 12:00 EST — KRYS — [Krystal Biotech to Present at 2026 Cantor Global Healthcare Conference](https://biopharmsignal.com/news/KRYS/krystal-biotech-to-present-at-2026-cantor-global-healthcare-conference-568c7dd8) — Source: Krystal Biotech
- 2026-09-03 11:00 EST — RCKT — [Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-to-participate-in-upcoming-investor-conferences-bbefd532) — Source: Rocket Pharmaceuticals
- 2026-09-02 11:30 EST — ABEO — [Abeona Therapeutics® Announces Participation in Upcoming Investor Conferences](https://biopharmsignal.com/news/ABEO/abeona-therapeutics-announces-participation-in-upcoming-investor-conferences-017a6b95) — Source: Abeona Therapeutics
- 2026-09-01 13:00 EST — EDIT — [Editas Medicine to Participate in Upcoming Investor Conferences](https://biopharmsignal.com/news/EDIT/editas-medicine-to-participate-in-upcoming-investor-conferences-0428bd41) — Source: Editas Medicine
- 2026-09-01 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-000ee332) — Source: Abeona Therapeutics
- 2026-08-13 12:00 EST — MGTX — [MeiraGTx Reports Second Quarter 2026 Financial and Operational Results](https://biopharmsignal.com/news/MGTX/meiragtx-reports-second-quarter-2026-financial-and-operational-results-7f83916b) — Source: MeiraGTx
- 2026-08-13 11:30 EST — ABEO — [Abeona Therapeutics® Reports Second Quarter 2026 Results and Provides Business Update](https://biopharmsignal.com/news/ABEO/abeona-therapeutics-reports-second-quarter-2026-results-and-provides-business-up-5235c160) — Source: Abeona Therapeutics
- 2026-08-13 05:00 EST — PHAR — [Pharming announces commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients with APDS aged 4 and older](https://biopharmsignal.com/news/PHAR/pharming-announces-commercial-launch-of-joenja-leniolisib-in-japan-for-adult-and-15d022ed) — Source: Pharming Group
- 2026-08-10 20:00 EST — RCKT — [Rocket Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Progress](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-reports-second-quarter-2026-financial-results-and-highlig-f6cb5dae) — Source: Rocket Pharmaceuticals
- 2026-08-06 20:30 EST — MGTX — [MeiraGTx Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/MGTX/meiragtx-announces-inducement-grant-under-nasdaq-listing-rule-5635-c-4-3ee9d0a1) — Source: MeiraGTx
- 2026-08-06 11:30 EST — ABEO — [Abeona Therapeutics® to Host Second Quarter 2026 Financial Results Conference Call on August 13, 2026](https://biopharmsignal.com/news/ABEO/abeona-therapeutics-to-host-second-quarter-2026-financial-results-conference-cal-9a5111ba) — Source: Abeona Therapeutics
- 2026-08-05 20:01 EST — EDIT — [Editas Medicine Announces Second Quarter 2026 Results and Business Updates](https://biopharmsignal.com/news/EDIT/editas-medicine-announces-second-quarter-2026-results-and-business-updates-733cacb7) — Source: Editas Medicine
- 2026-08-04 20:15 EST — EDIT — [Editas Medicine Strengthens Board of Directors with Appointment of Patrick T. Ellinor, M.D., Ph.D.](https://biopharmsignal.com/news/EDIT/editas-medicine-strengthens-board-of-directors-with-appointment-of-patrick-t-ell-517ace14) — Source: Editas Medicine
- 2026-08-04 11:30 EST — ABEO — [CMS Grants New Technology Add-On Payment (NTAP) Status for ZEVASKYN®](https://biopharmsignal.com/news/ABEO/cms-grants-new-technology-add-on-payment-ntap-status-for-zevaskyn-300aae33) — Source: Abeona Therapeutics
- 2026-08-04 06:00 EST — PHAR — [Pharming Group to participate in Canaccord Genuity 46th Annual Growth Conference](https://biopharmsignal.com/news/PHAR/pharming-group-to-participate-in-canaccord-genuity-46th-annual-growth-conference-89bea5c1) — Source: Pharming Group
- 2026-08-03 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-bd27d591) — Source: Abeona Therapeutics
- 2026-08-03 11:00 EST — RCKT — [Rocket Pharmaceuticals Announces Positive Clinical Safety Update from Initial Three Patients Treated with RP-A501 Under Modified Phase 2 Protocol for Danon Disease](https://biopharmsignal.com/news/RCKT/rocket-pharmaceuticals-announces-positive-clinical-safety-update-from-initial-th-979c2d7f) — Source: Rocket Pharmaceuticals
- 2026-08-03 11:00 EST — KRYS — [Krystal Biotech Announces Second Quarter 2026 Financial and Operating Results](https://biopharmsignal.com/news/KRYS/krystal-biotech-announces-second-quarter-2026-financial-and-operating-results-6dbba7e1) — Source: Krystal Biotech
- 2026-07-30 05:00 EST — PHAR — [Pharming reports second quarter and first half 2026 financial results and revises year-end guidance; strong Joenja momentum and near-term clinical readouts to support broader therapeutic use](https://biopharmsignal.com/news/PHAR/pharming-reports-second-quarter-and-first-half-2026-financial-results-and-revise-5218552c) — Source: Pharming Group
- 2026-07-23 21:00 EST — KRYS — [Krystal Biotech to Report Second Quarter 2026 Financial Results on August 3, 2026](https://biopharmsignal.com/news/KRYS/krystal-biotech-to-report-second-quarter-2026-financial-results-on-august-3-2026-2f96b902) — Source: Krystal Biotech

## Companies

- [ABEO](https://biopharmsignal.com/company/ABEO) — Abeona Therapeutics
- [AVRO](https://biopharmsignal.com/company/AVRO) — AVROBIO
- [BMRN](https://biopharmsignal.com/company/BMRN) — BioMarin Pharmaceutical
- [CCBS](https://biopharmsignal.com/company/CCBS) — Castle Creek Biosciences
- [EDIT](https://biopharmsignal.com/company/EDIT) — Editas Medicine
- [KRYS](https://biopharmsignal.com/company/KRYS) — Krystal Biotech
- [MGTX](https://biopharmsignal.com/company/MGTX) — MeiraGTx
- [PHAR](https://biopharmsignal.com/company/PHAR) — Pharming Group
- [RGNX](https://biopharmsignal.com/company/RGNX) — REGENXBIO Inc.
- [RCKT](https://biopharmsignal.com/company/RCKT) — Rocket Pharmaceuticals
- [QURE](https://biopharmsignal.com/company/QURE) — uniQure N.V.