Gene editing allows scientists to alter DNA sequences with unprecedented ease and precision, unlocking possibilities for transformative advances across healthcare, agriculture, biotechnology and more. At the forefront of this revolution is CRISPR – a technology enabling cutting and pasting within genetic code to silence, enhance or correct disease-causing abnormalities.
CRISPR has captured immense excitement and investment based on the promise of solving humanity‘s greatest challenges – from ending cancer to eradicating hunger. But transformative change does not happen overnight. It requires innovative companies equipped with the expertise and resources to translate groundbreaking science into solutions for the real world.
The good news? The CRISPR arena is filled with pioneering firms striving to deliver on gene editing‘s next-generation potential. Excitement is so great that analysts project the global CRISPR technology market ballooning to $6.28 billion by 2025.
In this comprehensive industry guide, we spotlight the top 10 CRISPR companies leading technology development based on total capital raised. We will analyze their focus areas, flagship programs, latest successes and future milestones that could reshape the biotechnology landscape.
What is CRISPR and Why it Matters
Before diving into the major players, it helps set context on what exactly CRISPR is and why researchers are so thrilled about capabilities that did not exist just a decade ago.
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It actually a naturally occurring bacterial defense system that scientists have now refashioned into a breakthrough genome editing tool.
Bacteria use CRISPR‘s basic components – guide RNAs and DNA-cutting enzymes like Cas9 – to seek out and destroy invading viruses. By delivering the Cas9 enzyme along with a guide RNA that matches a target DNA sequence, researchers hijack bacteria‘s CRISPR machinery to make ultra-precise cuts within the genome.
These controlled DNA breaks then activate the cell‘s own repair mechanisms. But here is the revolutionary part – scientists can use CRISPR to trick the repair process into introducing intentional changes they specify at the cut site. This allows correcting disease-causing mutations or inserting therapeutic genes with unprecedented precision, efficiency and scale.
| Gene Editing Approach | Precision | Efficiency | Ease of Use | Cost |
|---|---|---|---|---|
| Early Methods (Zinc Fingers, TALENS) | Low | Low | Difficult | High |
| CRISPR | High | High | Simple | Low |
Unlike early gene editing predecessors, CRISPR offers superior precision while also being faster, cheaper and simpler to employ. These strengths have researchers racing to uncover applications from reversing inherited disorders to supercharging crop yields.
But delivering this revolutionary technology requires trailblazing companies with specialized expertise and resources. Powering the era of genomic medicine are bold ventures focused on harnessing CRISPR‘s full disruptive potential across the biotechnology sphere.
The Pioneering CRISPR Companies Reshaping Biology
The most promising CRISPR tech firms have collectively raised over $4 billion to translate bleeding edge science into real world impact. We profile the top 10 leading the commercialization charge based on total capital raised through 2022.
1. CRISPR Therapeutics
- Year Founded: 2013
- Location: Zug, Switzerland
- Founders: Rodger Novak, Shaun Foy, Samarth Kulkarni, Matthew Porteus, Emmanuelle Charpentier
- Total Funding: $923 Million
The highest-funded CRISPR biotech firm, CRISPR Therapeutics focuses on leveraging CRISPR/Cas9 to develop transformative genetic medicines. With IP licensed from co-discoverer Emmanuelle Charpentier, it was an early mover establishing foundational CRISPR/Cas9 intellectual property. This has allowed building a pipeline now progressing through clinical trials.
Flagship program CTX001 applies CRISPR editing in blood stem cells to functionally cure rare blood disorders like sickle cell and beta thalassemia. This approach showed strong efficacy in early trials, with patients free of transfusion requirements or sickle crises a year later. With a recent $900M deal with Nike, CRISPR Therapeutics is advancing a portfolio of CRISPR medicines targeting major unmet needs.
2. Intellia Therapeutics
- Year Founded: 2014
- Location: Cambridge, MA
- Founders: Nessan Bermingham, Rachel Haurwitz, Matt Kane, John M. Leonard, J. Andre Hochheimer
- Total Funding: $730 Million
With foundational IP licensed from early CRISPR innovator Jennifer Doudna, Intellia Therapeutics is pioneering CRISPR/Cas9-based genomic medicines. It is developing treatments for genetic diseases including transthyretin amyloidosis, hepatitis B virus infections and autoimmune disease.
Recent phase 1 data for lead candidate NTLA-2001 showed the first successful in vivo CRISPR treatment safely lowered disease-causing protein by 87% after a single dose. This landmark demonstrates clinical applicability of direct body editing. With a robust delivery platform allowing tissue targeting, Intellia strives to extend applications addressing major therapeutic challenges.
3. Editas Medicine
- Year Founded: 2013
- Location: Cambridge, MA
- Founders: David R. Liu, Feng Zhang, George Church, J. Keith Joung
- Total Funding: $580 Million
Editas Medicine is translating CRISPR gene editing systems into medicines for eye conditions, blood disorders like sickle cell anemia and cancers. With exclusive access to foundational Cas9 IP, Editas has built patent protection across complementary CRISPR systems like Cas12a.
The company has multiple clinical programs underway targeting inherited retinal diseases. Lead candidate EDIT-101 applies in vivo CRISPR treatment directly in the body to reverse blindness from a specific genetic mutation. Preclinical data shows this experimental medicine restored vision by enabling light detection again in photoreceptor cells. Editas strives to extend gene editing‘s curative potential by addressing root causes of debilitating diseases.
4. Caribou Biosciences
- Year Founded: 2011
- Location: Berkeley, CA
- Founders: Jennifer Doudna, Martin Jinek, James Berger, Rachel Haurwitz
- Total Funding: $418 Million
Co-founded by CRISPR pioneer Jennifer Doudna, Caribou Biosciences has an IP estate covering foundational CRISPR-Cas9 technologies and innovations enabling flexible editing. It provides researchers critical tools like ready-to-use Cas9 reagents that scientists globally employ to modify genes underpinning biology and disease.
Splitting focus between tools and treatments, Caribou has also spun off several CRISPR therapeutics ventures. One example is Intellia Therapeutics, which Caribou co-founded to translate CRISPR into clinical medicines. Caribou itself has preclinical programs pursuing antivirals and engineered cell therapies benefiting from its ability to enhance specificity of genetic corrections.
5. Beam Therapeutics
- Year Founded: 2017
- Location: Cambridge, MA
- Founders: Feng Zhang, David R. Liu, John Evans
- Total Funding: $370 Million
Founded by CRISPR pioneers Feng Zhang and David Liu, Beam Therapeutics leverages next-generation CRISPR editing approaches called base editing. This targets misspellings in DNA without making cuts, offering tailored treatment of genetic diseases. Compared to traditional cut-and-paste CRISPR, base editing minimizes genome changes with the goal of safer, more precise therapeutics.
Beam is applying base editing to treat sickle cell disease, directly correcting the single-letter mutation underlying symptoms. It also has programs pursuing heart diseases and an approach using CRISPR activators to amplify beneficial genes. With a crack team specializing in CRISPR advancement, Beam aims to push editing capabilities further into more refined, readily controllable treatments.
6. Prime Medicine
- Year Founded: 2020
- Location: Cambridge, MA
- Founders: Keith Gottesdiener, David R. Liu, Jeffrey Smith, Jennifer Doudna
- Total Funding: $315 Million
Formed by CRISPR pioneers Jennifer Doudna and David Liu, Prime Medicine is built around Liu‘s latest innovation – Prime Editing. This next-gen CRISPR strategy enables rewriting nearly any type of genetic variation without double-stranded breaks, offering unprecedented editing precision suited for therapies.
Prime Medicine has exclusive license to foundational Prime Editing IP out the gate, including covering human therapeutic use from Editas Medicine. It has research underway targeting sickle cell, cancer and rare diseases. With configurable editors possible by tweaking guide RNAs, Prime Editing provides extensive tailoring forCONTEXT and PRIME seek to advance Prime Editing capabilities to directly correct more disease targets.
| Comparing CRISPR Strategies | Method | Precision | Efficiency | Safety |
|---|---|---|---|---|
| Standard CRISPR | Double strand DNA breaks | Medium | High | Risk of unwanted changes |
| Base Editing | Directly swap letters | Higher | Medium | More changes minimized |
| Prime Editing | Direct new sequence writing | Highest | Medium | Least changes needed |
Advanced CRISPR approaches refine specificity and controllability for therapeutic use where minimizing genome changes is critical. With vast flexibility plugging desired sequences into guide RNAs, Prime Medicine has tremendous potential to erase root causes of disease by rewriting DNA glitches.
7. Mammoth Biosciences
- Year Founded: 2017
- Location: San Francisco, CA
- Founders: Jennifer Doudna, Janice Chen, Lucas Harrington, Trevor Martin
- Total Funding: $195 Million
Initially pursuing CRISPR-powered disease detection, Mammoth Biosciences leverages CRISPR‘s intrinsic DNA searching ability to rapidly test patient samples for viruses. It has an experimental platform using Cas13 that can signal present of SARS-CoV-2 virus in just 20 minutes.
Expanding beyond diagnostics, Mammoth is also developing next-gen CRISPR cell and gene therapies. Its portfolio includes CAR-T treatments with enhanced targeting and signaling as well as ways to insert safer control switches. Mammoth ultimately aims to expand CRISPR use through maximizing programmability of cell and immune behavior.
8. Poseida Therapeutics
- Year Founded: 2015
- Location: San Diego, CA
- Founders: Eric Ostertag, Matthew Osborne, Mark Gergen, Martin Amberg, Joseph Villa
- Total Funding: $174 Million
Poseida Therapeutics applies CRISPR and gene editing strategies to engineer transformative cell and gene therapies targeting cancer. The company genetically enhances immune cells ex vivo by knocking out inhibitory pathways and outfitting with synthetic receptors detecting solid tumors. This approach aims to create allogenic CAR-T products more potent and with less side effects than early generation options.
Poseida‘s autologous pipeline also applies CRISPR to insert safer control switches minimizing serious adverse effects. With in-house capabilities combined across CRISPR, gene insertion and delivery – Poseida strives to push the boundaries of genetic engineering for bringing next-gen, off-the-shelf cell medicines to patients.
9. Precision BioSciences
- Year Founded: 2006
- Location: Durham, NC
- Founders: Derek Jantz, Jeff Smith, Matthew Kane
- Total Funding: $135 Million
Initially focused on early genome editing options, Precision BioSciences pivoted to the CRISPR arena through developing ARCUS – a proprietary nuclease technology complementary to Cas9. By optimizing efficacy and specificity, ARCUS offers expanded targeting range and minimized off-target effects.
Precision is pursuing in vivo treatments applying gene correction guided by ARCUS to counter diseases driven by single faulty genes – like primary hyperoxaluria and hemophilia. It also has allogenic CAR-T therapies undergoing testing against liquid and solid tumors that integrate ARCUS to maximize cancer cell destruction. Precision ultimately aims to extend the reach of genetic medicines through therapeutic insight combined with manufacturing excellence.
10. Inscripta
- Founded: 2015
- Location: Boulder, CO
- Founders: Kevin Ness, Mitch Fernandez, Ryan Gill
- Total Funding: $118 Million
Providing researchers critical tools for unlocking experimentation, Inscripta has an automated benchtop platform allowing on-demand design and generation of thousands of custom CRISPR guides. This unprecedented scale empowers targeted genetic screens investigating roles of gene families.
Inscripta also produces various CRISPR libraries for screening and directly sells instruments like the Onyx Digital PCR device tightly integrating editing workflows with genomic analysis. The company has even recently begun direct commercialization of CRISPR-modified cell lines for drug discovery pursuits across major disease areas. By powering precision genome editing, Inscripta strives to accelerate breakthroughs across biomedicine.
The Outlook: More Innovation Fueling the CRISPR Revolution
These profiled CRISPR companies are collectively pursuing hundreds of high potential programs striving to finally make good on genome editing‘s long-running promise. Though risks around feasibility, safety and ethics linger – progress unlocking precision treatment of once untreatable conditions inspires confidence we sit on the cusp of a healthcare revolution.
Bolstered by over $4 billion in capital, the pioneers highlighted here will continue charting the course for realizing this immense potential. We anticipate much more to come as next-generation CRISPR technologies like base and prime editing mature through the drug development cycle. Passionate teams specializing across biomedicine, agriculture and industrial biotech ensure CRISPR is poised to fast emerge as perhaps this century‘s most transformative basic science innovation.
