---
title: "Gene Therapy 13Fs: Vertex-CRISPR, BioMarin, Sarepta, Beam, Editas"
type: learn
slug: gene-therapy-13f-vrtx-bmrn-decoder
canonical_url: https://13finsight.com/learn/gene-therapy-13f-vrtx-bmrn-decoder
published_at: 2026-05-16T18:41:42.773Z
updated_at: 2026-05-16T18:41:46.507Z
author: Sarah Mitchell
author_title: Education Editor
author_url: https://13finsight.com/authors/sarah-mitchell
word_count: 727
locale: en
source: 13F Insight
---

# Gene Therapy 13Fs: Vertex-CRISPR, BioMarin, Sarepta, Beam, Editas

> Vertex-CRISPR Therapeutics Casgevy, BioMarin Roctavian, Sarepta Elevidys, plus Beam Therapeutics, Editas Medicine, Intellia Therapeutics, and Verve Therapeutics anchor US-traded gene therapy 13F positioning. Multi-year emerging AAV gene therapy adoption, CRISPR gene editing, plus emerging emerging in-vivo gene editing drive distinctive institutional patterns.

US-traded gene therapy equities form a distinctive emerging biotech corner of institutional 13F positioning. Vertex Pharmaceuticals (VRTX, Casgevy with CRISPR Therapeutics), CRISPR Therapeutics (CRSP), BioMarin Pharmaceutical (BMRN, Roctavian), Sarepta Therapeutics (SRPT, Elevidys), Beam Therapeutics (BEAM, base editing), Editas Medicine (EDIT, CRISPR), Intellia Therapeutics (NTLA, in-vivo CRISPR), plus Verve Therapeutics (VERV, in-vivo base editing) anchor the cohort. Multi-year emerging AAV gene therapy adoption, CRISPR gene editing, plus emerging emerging in-vivo gene editing drive distinctive institutional positioning. Reading gene therapy 13F positioning requires understanding the technology framework plus the multi-year clinical dynamics.The gene therapy business modelGene therapy companies operate four primary economic engines:AAV gene therapy adoption. Multi-year emerging AAV (adeno-associated virus) gene therapy adoption drives BioMarin plus Sarepta plus emerging emerging emerging Novartis Zolgensma plus emerging emerging Pfizer-Sangamo plus emerging emerging Spark Therapeutics (Roche) operator economics. Multi-year emerging AAV serotype selection plus emerging emerging emerging emerging immunogenicity dynamics plus emerging emerging emerging emerging manufacturing complexity plus emerging emerging emerging emerging premium pricing ($2-3M+ per patient).CRISPR gene editing. Multi-year emerging CRISPR gene editing drives Vertex-CRISPR plus emerging emerging Editas plus emerging emerging Beam plus emerging emerging Intellia plus emerging emerging Verve plus emerging emerging Tessera operator dynamics. Multi-year emerging Vertex-CRISPR Casgevy (sickle cell disease plus beta-thalassemia, FDA approved 2023 first CRISPR therapy at $2.2M per patient) drives multi-year emerging CRISPR commercialization. Multi-year emerging emerging Vertex commercialization plus 50/50 economics CRISPR Therapeutics.In-vivo gene editing. Multi-year emerging in-vivo gene editing drives multi-year emerging operator strategic positioning. Multi-year emerging Intellia NTLA-2001 transthyretin amyloidosis plus emerging emerging Verve VERVE-101 PCSK9 plus emerging emerging Beam BEAM-301 glycogen storage disease plus emerging emerging Prime Editing plus emerging emerging Tessera emerging emerging emerging epigenome editing drive multi-year emerging in-vivo gene editing. Multi-year emerging in-vivo (delivered systemically vs ex-vivo) drives broader patient access.Cell therapy adoption emerging. Multi-year emerging cell therapy adoption drives Gilead-Kite plus emerging emerging Novartis plus emerging emerging Bristol-Myers Squibb plus emerging emerging Johnson & Johnson-Legend Biotech plus emerging emerging 2seventy bio operator dynamics. Multi-year emerging CAR-T (Yescarta, Tecartus, Carvykti, Breyanzi, Abecma) plus emerging emerging T cell engagers plus emerging emerging allogeneic cell therapy plus emerging emerging emerging emerging Adicet plus emerging emerging emerging emerging Allogene drive multi-year cell therapy.Major US-traded gene therapy namesVertex Pharmaceuticals (VRTX)Diversified cystic fibrosis plus emerging emerging Casgevy (CRISPR Therapeutics partnered sickle cell disease plus beta-thalassemia, FDA approved 2023) plus emerging emerging emerging Suzetrigine non-opioid pain plus emerging emerging Type 1 diabetes Zimislecel plus emerging emerging IgA nephropathy povetacicept plus emerging emerging Reshma Kewalramani CEO leadership.CRISPR Therapeutics (CRSP)Pure-play CRISPR plus emerging emerging Vertex-partnered Casgevy plus emerging emerging in-vivo CRISPR pipeline plus emerging emerging Samarth Kulkarni CEO leadership.BioMarin Pharmaceutical (BMRN)Diversified rare disease plus emerging emerging Roctavian (hemophilia A AAV gene therapy) plus emerging emerging Voxzogo achondroplasia plus emerging emerging Alexander Hardy CEO leadership.Sarepta Therapeutics (SRPT)Duchenne muscular dystrophy (DMD) focus plus emerging emerging Elevidys gene therapy (AAV) plus emerging emerging Doug Ingram CEO leadership.Beam Therapeutics (BEAM)Base editing pioneer plus emerging emerging ex-vivo plus in-vivo base editing plus emerging emerging BEAM-101 sickle cell disease plus emerging emerging John Evans CEO leadership.Editas Medicine (EDIT)CRISPR plus emerging emerging EDIT-301 sickle cell disease plus beta-thalassemia plus emerging emerging Gilead Kite-partnered alpha-beta TCR CAR-T plus emerging emerging Gilmore O'Neill CEO leadership.Intellia Therapeutics (NTLA)In-vivo CRISPR pioneer plus emerging emerging NTLA-2001 (transthyretin amyloidosis Phase 3) plus emerging emerging NTLA-2002 hereditary angioedema plus emerging emerging John Leonard CEO leadership.Verve Therapeutics (VERV)In-vivo base editing plus emerging emerging VERVE-102 PCSK9 plus emerging emerging Sekar Kathiresan CEO leadership.How institutional managers position around gene therapyThree patterns appear across smart-money 13Fs:Pattern 1: Vertex-Casgevy concentrationVRTX, CRSP-concentrated growth manager positions reflect Casgevy plus emerging emerging CRISPR partnership plus emerging emerging CRISPR commercialization thesis.Pattern 2: AAV gene therapy positioningSRPT, BMRN-concentrated growth manager positions reflect AAV gene therapy plus emerging emerging Elevidys plus Roctavian commercialization thesis.Pattern 3: In-vivo editing positioningNTLA, VERV-concentrated growth manager positions reflect in-vivo gene editing plus emerging emerging NTLA-2001 plus VERVE-102 thesis.How to read gene therapy 13F positioningThree rules apply:Rule 1: Identify platform exposureAAV vs CRISPR vs base editing vs in-vivo have distinct dynamics.Rule 2: Watch clinical readoutsMulti-year clinical trial readouts drive operator pipeline.Rule 3: Cross-check payer reimbursementMulti-year payer reimbursement drives operator commercialization.What gene therapy positioning signalsCasgevy conviction. Concentrated VRTX, CRSP positions signal Casgevy plus CRISPR thesis.AAV conviction. Concentrated SRPT, BMRN positions signal AAV gene therapy thesis.In-vivo conviction. Concentrated NTLA, VERV positions signal in-vivo editing thesis.For real-time tracking of gene therapy 13F activity, see the institutional signals feed.

## FAQ

### What are the major US-traded gene therapy companies?

Eight major US-traded gene therapy: (1) Vertex Pharmaceuticals (VRTX) — Casgevy with CRISPR; (2) CRISPR Therapeutics (CRSP); (3) BioMarin Pharmaceutical (BMRN) — Roctavian; (4) Sarepta Therapeutics (SRPT) — Elevidys; (5) Beam Therapeutics (BEAM) — base editing; (6) Editas Medicine (EDIT) — CRISPR; (7) Intellia Therapeutics (NTLA) — in-vivo CRISPR; (8) Verve Therapeutics (VERV) — in-vivo base editing.

### How does AAV gene therapy work?

AAV (adeno-associated virus) gene therapy drives BioMarin plus Sarepta plus Novartis Zolgensma plus Pfizer-Sangamo plus Spark Therapeutics (Roche) operator economics. AAV serotype selection plus immunogenicity dynamics plus manufacturing complexity plus premium pricing ($2-3M+ per patient) drive operator dynamics. Multi-year emerging AAV gene therapy approvals plus emerging emerging payer reimbursement plus emerging emerging multi-year durability data drive multi-year adoption.

### How does CRISPR gene editing work?

CRISPR gene editing drives Vertex-CRISPR plus Editas plus Beam plus Intellia plus Verve operator dynamics. Vertex-CRISPR Casgevy (sickle cell disease plus beta-thalassemia, FDA approved 2023 first CRISPR therapy at $2.2M per patient) drives CRISPR commercialization. Multi-year Vertex commercialization plus 50/50 economics CRISPR Therapeutics drives multi-year operator economics. Multi-year emerging ex-vivo (cells edited outside body) vs in-vivo CRISPR drives operator differentiation.

### How does in-vivo gene editing work?

In-vivo gene editing drives operator strategic positioning. Intellia NTLA-2001 transthyretin amyloidosis plus Verve VERVE-101 PCSK9 plus Beam BEAM-301 glycogen storage disease plus Prime Editing plus Tessera epigenome editing drive in-vivo gene editing. In-vivo (delivered systemically via lipid nanoparticle, AAV, or other delivery) vs ex-vivo drives broader patient access. Multi-year emerging in-vivo CRISPR Phase 3 readouts drive multi-year adoption.

### How does cell therapy adoption work?

Cell therapy adoption drives Gilead-Kite plus Novartis plus Bristol-Myers Squibb plus Johnson & Johnson-Legend Biotech plus 2seventy bio operator dynamics. CAR-T (Yescarta, Tecartus, Carvykti, Breyanzi, Abecma) plus T cell engagers plus allogeneic cell therapy plus Adicet plus Allogene drive cell therapy. Multi-year emerging autologous CAR-T (patient-specific cells) plus emerging allogeneic (off-the-shelf donor cells) drive multi-year operator economics.

### What signals gene therapy cycle inflections?

Four signals: (1) clinical trial readouts plus emerging FDA approval calendar; (2) Casgevy commercialization plus emerging payer reimbursement; (3) in-vivo gene editing progression plus emerging Phase 3 readouts; (4) M&A activity plus emerging emerging deal pipeline. Concentrated 13F changes around these signals reveal manager cycle reading.

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Source: 13F Insight — https://13finsight.com/learn/gene-therapy-13f-vrtx-bmrn-decoder
Author: Sarah Mitchell — https://13finsight.com/authors/sarah-mitchell
Last updated: 2026-05-16T18:41:46.507Z