---
title: "Gene Therapy 13Fs: CRISPR, Intellia, Beam, Editas, Sarepta"
type: learn
slug: gene-therapy-13f-crsp-ntla-vrtx-decoder
canonical_url: https://13finsight.com/learn/gene-therapy-13f-crsp-ntla-vrtx-decoder
published_at: 2026-05-16T05:49:03.149Z
updated_at: 2026-05-16T05:49:07.022Z
author: Sarah Mitchell
author_title: Education Editor
author_url: https://13finsight.com/authors/sarah-mitchell
word_count: 412
locale: en
source: 13F Insight
---

# Gene Therapy 13Fs: CRISPR, Intellia, Beam, Editas, Sarepta

> CRISPR Therapeutics, Intellia Therapeutics, Beam Therapeutics, Editas Medicine, and Sarepta Therapeutics anchor US gene therapy 13F positioning. Clinical milestone cycles, manufacturing scaling, reimbursement framework development, and ongoing M&A drive distinctive institutional patterns.

US gene therapy equities form a distinctive innovation-driven specialty-biotech corner of institutional 13F positioning. CRISPR Therapeutics (CRSP), Intellia Therapeutics (NTLA), Beam Therapeutics (BEAM), Editas Medicine (EDIT), and Sarepta Therapeutics (SRPT) anchor the cohort. Multi-year clinical milestone cycles, manufacturing scaling challenges, reimbursement framework development, and ongoing M&A activity drive distinctive institutional patterns. Reading gene therapy 13F positioning requires understanding the clinical-milestone framework plus the multi-year manufacturing and reimbursement cycle dynamics.The gene therapy business modelGene therapy faces four primary economic drivers:Clinical milestone cycles. Multi-year clinical trial timelines plus FDA approval pathways drive long-cycle franchise economics. Multi-year Phase 1/2 plus pivotal Phase 3 readouts produce dramatic equity revaluation.Manufacturing scaling. Gene therapy manufacturing complexity (viral vectors, autologous cell therapy, plus emerging in vivo delivery) drives capacity scaling plus emerging emerging cost reduction challenges.Reimbursement framework. Multi-year reimbursement framework development for one-time gene therapy treatments (multi-million dollar pricing) plus emerging outcomes-based pricing affects commercial economics.M&A activity. Multi-year big pharma M&A activity (Vertex-CRISPR partnership for CASGEVY, Pfizer's Sangamo plus Beam partnerships, plus emerging acquisitions) drives valuation dynamics.Major US gene therapy namesCRISPR Therapeutics (CRSP)Joint developer with Vertex Pharmaceuticals of CASGEVY (first FDA-approved CRISPR therapy for sickle cell plus beta thalassemia). Multi-year pipeline expansion plus emerging in vivo plus immuno-oncology applications.Intellia Therapeutics (NTLA)In vivo CRISPR therapy leader with NTLA-2002 (hereditary angioedema) plus NTLA-2001 (transthyretin amyloidosis, in partnership with Regeneron). Multi-year clinical pipeline.Beam Therapeutics (BEAM)Base editing technology leader with BEAM-101 (sickle cell), BEAM-201 (acute lymphoblastic leukemia), plus emerging pipeline. Multi-year operational scaling.Editas Medicine (EDIT)CRISPR/Cas9 plus Cas12a gene editing focus. Multi-year operational restructuring plus emerging in vivo applications.Sarepta Therapeutics (SRPT)Duchenne muscular dystrophy (DMD) franchise (Elevidys gene therapy, plus exon-skipping antisense therapies) plus emerging limb-girdle muscular dystrophy pipeline.How institutional managers position around gene therapyThree patterns:Pattern 1: CASGEVY-validation concentrationCRSP-concentrated growth manager positions reflect CASGEVY FDA approval plus expanding pipeline thesis.Pattern 2: In-vivo CRISPR positioningNTLA-concentrated growth manager positions reflect in vivo CRISPR therapy thesis.Pattern 3: Commercial-franchise positioningSRPT-concentrated active manager positions reflect Elevidys plus DMD franchise commercial thesis.How to read gene therapy 13F positioningThree rules:Rule 1: Identify technology platformCRISPR/Cas9 vs base editing vs prime editing vs viral vector have distinct economics.Rule 2: Watch clinical milestonesPhase 3 readouts plus FDA approvals drive operator equity revaluation.Rule 3: Cross-check M&A activityBig pharma M&A drives valuation step changes.What gene therapy positioning signalsCASGEVY-validation conviction. Concentrated CRSP positions signal CASGEVY plus pipeline thesis.In-vivo CRISPR conviction. Concentrated NTLA positions signal in vivo CRISPR thesis.Commercial-franchise conviction. Concentrated SRPT positions signal Elevidys franchise thesis.For real-time tracking of gene therapy 13F activity, see the institutional signals feed.

## FAQ

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

Five major US gene therapy: (1) CRISPR Therapeutics (CRSP) — CASGEVY developer with Vertex for sickle cell plus beta thalassemia; (2) Intellia Therapeutics (NTLA) — in vivo CRISPR with NTLA-2002 plus NTLA-2001 with Regeneron; (3) Beam Therapeutics (BEAM) — base editing leader; (4) Editas Medicine (EDIT) — CRISPR/Cas9 plus Cas12a; (5) Sarepta Therapeutics (SRPT) — Duchenne muscular dystrophy franchise with Elevidys gene therapy.

### How does CRISPR therapy work?

CRISPR/Cas9 gene editing technology enables targeted modification of DNA sequences. CASGEVY (Vertex Pharmaceuticals plus CRISPR Therapeutics, FDA approved December 2023) treats sickle cell disease plus beta thalassemia through ex vivo editing of patient hematopoietic stem cells. Multi-year emerging in vivo CRISPR therapy (Intellia, Editas) plus base editing (Beam) plus prime editing platforms expand applications. Reading platform progress drives positioning.

### What is the gene therapy manufacturing challenge?

Gene therapy manufacturing complexity drives capacity scaling challenges. Viral vector manufacturing (AAV, lentivirus) requires specialized facilities. Autologous cell therapy requires patient-specific manufacturing plus complex logistics. Multi-year emerging in vivo delivery (LNP plus emerging delivery systems) attempts to reduce manufacturing complexity. Multi-year emerging cost reduction plus capacity scaling drives long-cycle economics. Reading manufacturing capacity drives positioning.

### How does gene therapy reimbursement work?

Multi-year reimbursement framework development for one-time gene therapy treatments (typically $2-4M+ per patient pricing) drives commercial economics. Multi-year emerging outcomes-based pricing arrangements plus extended payment plans plus CMS demonstration projects drive reimbursement evolution. Multi-year commercial uptake limited by reimbursement plus treatment center infrastructure plus patient identification. Reading reimbursement coverage drives positioning.

### What is Sarepta's Elevidys franchise?

Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec) gene therapy treats Duchenne muscular dystrophy (DMD) through AAV-delivered functional dystrophin gene. FDA approved June 2023 with subsequent label expansions. Multi-year commercial scaling plus emerging limb-girdle muscular dystrophy pipeline drives franchise. Multi-year DMD population identification plus treatment center expansion drives commercial trajectory. Concentrated SRPT positions reflect Elevidys franchise thesis.

### What signals gene therapy cycle inflections?

Four signals: (1) Phase 3 clinical readouts plus FDA approval milestones; (2) manufacturing capacity expansion plus emerging cost reduction; (3) reimbursement framework development plus commercial uptake; (4) big pharma M&A activity. Concentrated 13F changes around these signals reveal manager cycle reading.

---

Source: 13F Insight — https://13finsight.com/learn/gene-therapy-13f-crsp-ntla-vrtx-decoder
Author: Sarah Mitchell — https://13finsight.com/authors/sarah-mitchell
Last updated: 2026-05-16T05:49:07.022Z