Company 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.
Why watch Rare Disease Gene Therapy
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 Gene Therapy news
The latest feed items from companies currently grouped under Rare Disease Gene Therapy.
- 01
- 02
- 03
CRNXVertex to Acquire Crinetics Pharmaceuticals | Crinetics
Source: Crinetics Pharmaceuticals
- 04
BCRXBioCryst Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: BioCryst Pharmaceuticals
- 05
CRNXCrinetics Pharmaceuticals to Report Second Quarter 2026 Financial Results on August 4, 2026 | Crinetics
Source: Crinetics Pharmaceuticals
- 06
SRPTSarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Source: Sarepta Therapeutics
- 07
SRPTSarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®
Source: Sarepta Therapeutics
- 08
- 09
REGNRegeneron to Report Second Quarter 2026 Financial and Operating Results and Host Conference Call and Webcast
Source: Regeneron Pharmaceuticals
- 10
REGNCemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG)
Source: Regeneron Pharmaceuticals
- 11
- 12
RCKTRocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million
Source: Rocket Pharmaceuticals
- 13
REGNRegeneron to Highlight Progress Across Its Metabolic Disease, Ophthalmology and Rare Disease Pipelines at ADA and ENDO
Source: Regeneron Pharmaceuticals
- 14
SRPTSarepta Therapeutics to Participate in Upcoming Investor Conferences
Source: Sarepta Therapeutics
- 15
- 16
- 17
- 18
REGNOtarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss
Source: Regeneron Pharmaceuticals
- 19
- 20
Companies in Rare Disease Gene Therapy
16 companies tagged with Rare Disease Gene Therapy.
Frequently Asked Questions
What does the Rare Disease Gene Therapy sector include?
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.
How many companies are currently tagged with Rare Disease Gene Therapy?
16 companies are currently grouped under Rare Disease Gene Therapy on BioPharmSignal.
Which companies are most visible in the Rare Disease Gene Therapy group right now?
AMGN, BCRX, CRNX, EDIT are among the companies currently shown on this sector page.
How can I watch news for Rare Disease Gene Therapy?
Use the Watch button on this page to subscribe to Rare Disease Gene Therapy headlines and related company updates through your BioPharmSignal saved filters.
What kinds of updates usually appear for Rare Disease Gene Therapy?
The livefeed typically surfaces company press releases, clinical readouts, FDA and regulatory updates, partnerships, financings, and commercialization developments tied to companies in Rare Disease Gene Therapy.