CRISPR Therapeutics

CRISPR Therapeutics

CRISPR Therapeutics (NASDAQ: CRSP) is a leading gene-editing company focused on developing transformative medicines using CRISPR/Cas9 gene-editing technology. Founded in 2013, the company is headquartered in Boston, Massachusetts, with operations in the US, UK, and Switzerland.

Overview

CRISPR Therapeutics

CRISPR Therapeutics (NASDAQ: CRSP) is a leading gene-editing company focused on developing transformative medicines using CRISPR/Cas9 gene-editing technology. Founded in 2013, the company is headquartered in Boston, Massachusetts, with operations in the US, UK, and Switzerland.

Overview

| Attribute | Details | [@casgevy2023]
|-----------|---------| [@pipeline2024]
| Founded | 2013 | [@vertex2015]
| Headquarters | Boston, Massachusetts, USA | [@sickle2023]
| IPO | NASDAQ: CRSP (2016) |
| Market Cap | ~$6B (2024) |
| Employees | ~500 |
| Website | crisprtx.com |

Technology Platform

CRISPR/Cas9 Gene Editing

CRISPR Therapeutics leverages CRISPR/Cas9 technology for precise gene editing:

• Cas9: Streptococcus pyogenes Cas9 nuclease
• Guide RNA: Custom RNA guides for target specificity
• Delivery: Lipid nanoparticles (LNPs), AAV, and viral vectors
• Applications: Gene knock-out, knock-in, and base editing

Strategic Focus

Pipeline

Clinical Programs

| Program | Indication | Modality | Status |
|---------|------------|----------|--------|
| CTX110 | B-cell lymphoma | Allogeneic CAR-T | Phase 1/2 |
| CTX120 | Multiple myeloma | Allogeneic CAR-T | Phase 1 |
| CTX130 | Solid tumors | Allogeneic CAR-T | Phase 1 |
| CTX130 | Renal cell carcinoma | Allogeneic CAR-T | Phase 1 |
| VCTX1 | Type 1 Diabetes | Regenerative | Preclinical |
| CTX110 | Sickle cell disease | Autologous ex vivo | Phase 1 |

Neurological Disease Programs

Alzheimer's Disease

• Target: Amyloid precursor protein (APP) gene editing
• Approach: Reduce Aβ production through gene knockout
• Status: Discovery stage

Parkinson's Disease

• Target: Alpha-synuclein (SNCA) gene silencing
• Approach: Reduce toxic protein aggregation
• Status: Discovery stage

Huntington's Disease

• Target: Mutant huntingtin (HTT) gene
• Approach: Allele-selective editing
• Status: Discovery stage

Clinical Progress

| Year | Milestone |
|------|-----------|
| 2013 | Company founded by Emmanuelle Charpentier |
| 2016 | IPO on NASDAQ |
| 2018 | First IND submission (CTX001 with Vertex) |
| 2020 | Positive Phase 1 data for CTX001 (sickle cell) |
| 2023 | FDA approval of Casgevy for sickle cell disease |
| 2024 | Multiple CAR-T programs in clinical trials |

Partnerships

Vertex Pharmaceuticals

• Focus: Hematopoietic stem cell therapies
• Programs: CTX001 for sickle cell and beta-thalassemia
• Deal: Up to $2.6B in milestone payments

ViaCyte

• Focus: Cell replacement therapy for diabetes
• Programs: Encapsulated cell devices with gene editing

Competitive Landscape

| Company | Technology | Focus |
|---------|------------|-------|
| Editas Medicine | CRISPR/Cas9 | In vivo editing |
| Intellia Therapeutics | CRISPR/Cas9 | LNP delivery |
| Caribou Biosciences | CRISPR/Cas9 | Off-the-shelf CAR-T |
| Beam Therapeutics | Base editing | Precise editing |

FDA Approval

Casgevy (exa-cel)

In December 2023, the FDA approved Casgevy (exagamglogene autotemcel), the first CRISPR-based therapy for sickle cell disease:

• Indication: Sickle cell disease
• Efficacy: 94% of patients achieved sustained VOC-free outcomes
• Manufacturer: CRISPR Therapeutics (partnered with Vertex)
• Price: $2.2M per treatment

NeuroWiki Cross-References

• CRISPR Gene Editing
• [Gene Therapy](/therapeutics/gene-therapy-neurodegeneration) Gene Therapy for Neurological Diseases
• Companies Index
• [Gene Therapy](/therapeutics/gene-therapy)
• [CRISPR-Cas9](/proteins/spr)
• [Alzheimer's Disease Pipeline Companies](/companies/alzheimers-disease-pipeline-companies)](/diseases/alzheimers-disease)
• [Parkinson's Disease Pipeline Companies](/companies/parkinsons-disease-pipeline-companies)](/genes/ar)
• [Companies Index](/diseases/mpan)

External Links

• [CRISPR Therapeutics Website](https://crisprtx.com)
• [CRISPR Therapeutics Pipeline](https://crisprtx.com/pipeline/)

References