# Gene Therapy Sector Canonical URL: https://biopharmsignal.com/sector/gene-therapy Gene Therapy includes companies delivering genetic material to correct, replace, silence, or modulate disease-driving biology. This sector page is useful for following businesses exposed to AAV, lentiviral, non-viral, or editing-enabled approaches across rare and broader diseases. Companies currently grouped under Gene Therapy: 40 ## 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 - 2026-10-06 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 - 2026-10-06 20:05 EST — CRBU — [Caribou Biosciences to Evaluate Strategic Alternatives](https://biopharmsignal.com/news/CRBU/caribou-biosciences-to-evaluate-strategic-alternatives-7064f17e) — Source: Caribou - 2026-10-06 14:00 EST — GSK — [ViiV Healthcare’s long-acting HIV treatment Cabenuva (cabotegravir + rilpivirine) meets primary endpoint of superior viral suppression vs. daily oral therapy in people with viremia](https://biopharmsignal.com/news/GSK/viiv-healthcare-s-long-acting-hiv-treatment-cabenuva-cabotegravir-rilpivirine-me-20d488d4) — Source: GSK plc - 2026-10-06 11:02 EST — OCGN — [Ocugen Appoints Industry Executive Jolanda Crombach General Manager of European Affiliate](https://biopharmsignal.com/news/OCGN/ocugen-appoints-industry-executive-jolanda-crombach-general-manager-of-european--12ef7410) — Source: Ocugen - 2026-10-02 20:01 EST — NTLA — [Intellia Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/NTLA/intellia-therapeutics-reports-inducement-grants-under-nasdaq-listing-rule-5635-c-c6e6fb53) — Source: Intellia - 2026-10-02 20:01 EST — NTLA — [Intellia Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/NTLA/intellia-therapeutics-reports-inducement-grants-under-nasdaq-listing-rule-5635-c-240d5c46) — Source: Intellia - 2026-10-02 14:15 EST — CRSP — [CRISPR Therapeutics to Present Clinical Data on Zugocabtagene Geleucel (Zugo-cel) in Autoimmune Disease at the American College of Rheumatology (ACR) Convergence 2026](https://biopharmsignal.com/news/CRSP/crispr-therapeutics-to-present-clinical-data-on-zugocabtagene-geleucel-zugo-cel--195d617d) — Source: CRISPR Therapeutics - 2026-10-02 12:00 EST — TSHA — [Taysha Gene Therapies Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/TSHA/taysha-gene-therapies-announces-inducement-grant-under-nasdaq-listing-rule-5635--18c0402b) — Source: Taysha Gene Therapies - 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 - 2026-10-01 12:00 EST — TSHA — [Taysha Gene Therapies Announces Multiple Encore Presentations Supporting TSHA-102 Rett Syndrome Clinical Program at the 55th Child Neurology Society Annual Meeting](https://biopharmsignal.com/news/TSHA/taysha-gene-therapies-announces-multiple-encore-presentations-supporting-tsha-10-88119405) — Source: Taysha Gene Therapies - 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 20:30 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--c621c21d) — Source: Sarepta Therapeutics - 2026-09-30 12:30 EST — SRPT — [Sarepta Therapeutics Presents New Data at 2026 World Muscle Society Annual Congress Demonstrating Safety and Clinically Meaningful Functional Benefit of ELEVIDYS (delandistrogene moxeparvovec) in Older Ambulatory Patients with Duchenne](https://biopharmsignal.com/news/SRPT/sarepta-therapeutics-presents-new-data-at-2026-world-muscle-society-annual-congr-237ee3f5) — Source: Sarepta Therapeutics - 2026-09-30 11:02 EST — OCGN — [Ocugen to Participate in Upcoming Scientific Conferences](https://biopharmsignal.com/news/OCGN/ocugen-to-participate-in-upcoming-scientific-conferences-7bd3a904) — Source: Ocugen - 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 11:02 EST — OCGN — [Ocugen to Participate in Upcoming October Investor and Industry Conferences](https://biopharmsignal.com/news/OCGN/ocugen-to-participate-in-upcoming-october-investor-and-industry-conferences-58af91b7) — Source: Ocugen - 2026-09-29 05:00 EST — PHAR — [Pharming announces CEO transition](https://biopharmsignal.com/news/PHAR/pharming-announces-ceo-transition-c241aabd) — Source: Pharming Group - 2026-09-25 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 ## Companies - [ABEO](https://biopharmsignal.com/company/ABEO) — Abeona Therapeutics - [ADVM](https://biopharmsignal.com/company/ADVM) — Adverum Biotechnologies - [AVRO](https://biopharmsignal.com/company/AVRO) — AVROBIO - [BAYRY](https://biopharmsignal.com/company/BAYRY) — Bayer AG - [BLFS](https://biopharmsignal.com/company/BLFS) — BioLife Solutions - [BMRN](https://biopharmsignal.com/company/BMRN) — BioMarin Pharmaceutical - [BLUE](https://biopharmsignal.com/company/BLUE) — bluebird bio, Inc. - [CRBU](https://biopharmsignal.com/company/CRBU) — Caribou - [CCBS](https://biopharmsignal.com/company/CCBS) — Castle Creek Biosciences - [CRSP](https://biopharmsignal.com/company/CRSP) — CRISPR Therapeutics - [EDIT](https://biopharmsignal.com/company/EDIT) — Editas Medicine - [GBIO](https://biopharmsignal.com/company/GBIO) — Generation Bio - [GNPX](https://biopharmsignal.com/company/GNPX) — Genprex - [GILD](https://biopharmsignal.com/company/GILD) — Gilead Sciences - [GRPH](https://biopharmsignal.com/company/GRPH) — Graphite Bio - [GSK](https://biopharmsignal.com/company/GSK) — GSK plc - [HNSA](https://biopharmsignal.com/company/HNSA) — Hansa Biopharma AB - [FIXX](https://biopharmsignal.com/company/FIXX) — Homology Medicines - [NTLA](https://biopharmsignal.com/company/NTLA) — Intellia - [KRYS](https://biopharmsignal.com/company/KRYS) — Krystal Biotech - [MXCT](https://biopharmsignal.com/company/MXCT) — MaxCyte - [MGTX](https://biopharmsignal.com/company/MGTX) — MeiraGTx - [MBIO](https://biopharmsignal.com/company/MBIO) — Mustang Bio - [NVS](https://biopharmsignal.com/company/NVS) — Novartis AG - [OCGN](https://biopharmsignal.com/company/OCGN) — Ocugen - [OMGA](https://biopharmsignal.com/company/OMGA) — Omega Therapeutics - [PASG](https://biopharmsignal.com/company/PASG) — Passage Bio - [PHAR](https://biopharmsignal.com/company/PHAR) — Pharming Group - [RGNX](https://biopharmsignal.com/company/RGNX) — REGENXBIO Inc. - [RHHBY](https://biopharmsignal.com/company/RHHBY) — Roche Holdings AG - [RCKT](https://biopharmsignal.com/company/RCKT) — Rocket Pharmaceuticals - [SANA](https://biopharmsignal.com/company/SANA) — Sana Biotechnology - [SGMO](https://biopharmsignal.com/company/SGMO) — Sangamo Therapeutics, Inc. - [SNY](https://biopharmsignal.com/company/SNY) — Sanofi - [SRPT](https://biopharmsignal.com/company/SRPT) — Sarepta Therapeutics - [TSHA](https://biopharmsignal.com/company/TSHA) — Taysha Gene Therapies - [QURE](https://biopharmsignal.com/company/QURE) — uniQure N.V. - [VRTX](https://biopharmsignal.com/company/VRTX) — Vertex Pharmaceuticals Incorporated - [VERV](https://biopharmsignal.com/company/VERV) — Verve Therapeutics - [VYGR](https://biopharmsignal.com/company/VYGR) — Voyager Therapeutics