Intellia Therapeutics (NASDAQ: NTLA) is a clinical-stage biotechnology company leading the development of CRISPR/Cas9 technologies for gene editing and gene therapy. Founded in 2014 as a spinout from Novartis, Intellia is headquartered in Cambridge, Massachusetts.
| Attribute | Details | [1]
|-----------|---------| [2]
| Founded | 2014 | [3]
| Headquarters | Cambridge, Massachusetts, USA | [4]
| IPO | NASDAQ: NTLA (2018) |
| Market Cap | ~$4B (2024) |
| Employees | ~600 |
| Website | intelliatx.com |
Intellia specializes in lipid nanoparticle (LNP) delivery for in vivo gene editing:
| Program | Indication | Target | Status |
|---|---|---|---|
| NTLA-2001 | Transthyretin (ATTR) amyloidosis | TTR | Phase 3 |
| NTLA-2002 | Hereditary angioedema | KLKB1 | Phase 1/2 |
| NTLA-3001 | Alpha-1 antitrypsin deficiency | SERPINA1 | Phase 1 (IND cleared) |
Intellia's Parkinson's disease program targeting GBA1 is particularly relevant for autonomic dysfunction:
GBA1 and Autonomic Symptoms
Heterozygous GBA1 mutations are the most common genetic risk factor for PD and are associated with:
Gene Therapy Approach
Intellia's CRISPR-based approach offers potential benefits:
Relevant Mechanisms
| Target | Autonomic Relevance |
|---|---|
| GBA1 | Lysosomal dysfunction, alpha-synuclein accumulation |
| Additional risk genes | May be identified for autonomic-specific targeting |
| Year | Milestone |
|---|---|
| 2014 | Founded as Novartis spinout |
| 2018 | IPO on NASDAQ |
| 2020 | First in vivo CRISPR data in humans (NTLA-2001) |
| 2021 | Positive Phase 1 data for NTLA-2001 |
| 2023 | Regeneron partnership ($400M upfront) |
| 2024 | Phase 3 enrollment for NTLA-2001 |
| Company | Technology | Key Differentiator |
|---|---|---|
| CRISPR Therapeutics | Ex vivo editing | First FDA-approved CRISPR therapy |
| Editas Medicine | In vivo CRISPR | CRISPR/Cas12a |
| Beam Therapeutics | Base editing | Precision editing |
| Intellia | LNP delivery | In vivo systemic delivery |
NTLA-2001 is the first systemically delivered CRISPR gene-editing therapy to show clinical activity:
CRISPR Gene Editing
Gene Therapy Lipid Nanoparticles
Companies Index
NTLA-2001 Clinical Data (2021). 2001. ↩︎
Regeneron Partnership (2023). 2023. ↩︎
Pipeline Overview (2024). 2024. ↩︎
In Vivo CRISPR Delivery (2020). 2020. ↩︎