CRISPR technology’s next wave: Ten companies to watch in 2025 

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a gene-editing technology that allows scientists to modify DNA with unprecedented precision. Discovered in the early 2010s, CRISPR technology leverages a natural defense mechanism used by bacteria to protect against viral infections. The system uses a guide RNA to direct the Cas9 enzyme to a specific location in the genome, where it creates a double-strand break. This break can then be repaired by the cell’s natural mechanisms, allowing for the addition, deletion, or modification of genetic material. CRISPR companies are seeing more and more success in the clinic and the market is growing. 

CRISPR has rapidly become one of the most powerful tools in genetic engineering, enabling precise changes to the DNA. Its applications are not limited to medicine, which is the focus of this article, as it also allows the creation of crops with selected traits in agriculture for instance. 

In recent years, the field of CRISPR technology has improved, and different forms of the technology are now being leveraged by biotech companies. Prime editing and base editing are innovative CRISPR-related technologies aiming to improve the versatility and precision of therapies. 

After CRISPR Therapeutics and Vertex Pharmaceuticals’ collaborative success leading to CASGEVY’s approval by the U.S. Food and Drug Administration (FDA) and Editas Medicine’s promising efforts to treat blindness, here are ten companies keeping the CRISPR field dynamic. 

Beam Therapeutics 

• Founded: 2017 
• Headquarters: Cambridge, Massachusetts, U.S. 
• CRISPR focus: Base editing (single-nucleotide edits without double-strand breaks) 

Beam Therapeutics is one of the leading names in the gene-editing space due to its early and deep commitment to base editing, a variant of CRISPR that enables the replacement of one DNA base with another without cutting both strands of the DNA. This approach aims to reduce the risk of unwanted insertions or deletions (indels) and off-target damage relative to traditional CRISPR-Cas9 editing. 

The company’s flagship program is BEAM-101, targeting sickle cell disease and beta-thalassemia. The idea is to edit hematopoietic stem cells ex vivo to mimic a benign hemoglobin variant or to upregulate fetal hemoglobin, to reduce or eliminate the pathological effects of the mutant hemoglobin in red blood cells. In addition, Beam is advancing programs in liver and or metabolic-related areas. For example, BEAM-302 in alpha-1 antitrypsin deficiency (AATD) is under investigation. 

On the funding and partnerships front, Beam went public in February 2020, raising about $207 million, and in 2025, it raised $500 million more via direct financing. It has also secured Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for BEAM-101 in the treatment of sickle cell disease. 

Recent developments are encouraging. In mid-2025, Beam announced new data from the BEACON phase 1/2 trial of BEAM-101, with 17 treated patients showing durable increases in fetal hemoglobin (HbF), reductions in sickle hemoglobin (HbS), fast engraftment, normalized hemolysis markers, and no vaso-occlusive crises (VOCs) after engraftment to date. 

Caribou Biosciences 

• Founded: 2011 
• Headquarters: Berkeley, California, U.S. 
• CRISPR focus: Genome editing for allogeneic cell therapies 

Caribou positions itself as a next-generation CRISPR company with a specific focus on allogeneic cell therapies. Its core technological distinguishing feature is its chRDNA (CRISPR hybrid RNA–DNA) platform. This approach is intended to support more complex editing while preserving genomic integrity and reducing unwanted off-target effects or chromosomal damage. 

Because its therapies are not patient-specific, Caribou must engineer cells to avoid immune rejection. To make its therapies compatible across patients, Caribou edits donor cells so they are less likely to be recognized as foreign by the immune system. This includes removing signals that would normally trigger rejection and adding protective ones that help the cells persist. 

Its leading clinical candidates include: 

• CB-010 (anti-CD19 CAR-T) in phase 1 trial targeting relapsed/refractory B cell non-Hodgkin lymphoma. 

• CB-011 (anti-BCMA CAR-T) in phase 1 trial for relapsed/refractory multiple myeloma. 

In 2025, the company announced a strategic pipeline prioritization, narrowing its focus to CB-010 and CB-011 and discontinuing or deprioritizing some other programs to conserve resources and extend its runway. It also reported cutting 32% of its workforce to streamline operations. 

Chroma Medicine / nChroma Bio 

• Founded: 2021 (merged with Nvelops Therapeutics to become nChroma Bio in 2024) 
• Headquarters: Boston, Massachusetts, U.S. 
• CRISPR focus: Epigenetic editing 

Chroma Medicine (now merged into nChroma Bio) approaches gene editing from a different angle: rather than cutting DNA, it works at the epigenetic level, modifying how genes are expressed. Its platform essentially couples programmable DNA-binding elements with effector domains that write or erase methylation marks, or otherwise adjust chromatin states, turning genes on or off or tuning their levels. 

Because it doesn’t change the underlying genetic code, Chroma argues it avoids risks associated with DNA breaks and allows safer multiplex regulation. 

One of its showcase preclinical experiments demonstrated simultaneous epigenetic modulation of three genes in primary human T cells. The result: durable silencing without detectable insertions and deletions or chromosomal rearrangements. 

Chroma launched with $125 million in financing in late 2021 to build its epigenetic editing platform, and in 2023, it secured a $135 million series B led by Google Ventures. In December 2024, Chroma merged with Nvelop Therapeutics, a company specializing in non-viral delivery vehicles, to form nChroma Bio, combining Chroma’s epigenetic editors with Nvelop’s delivery tech. The merged company announced $75 million of new funding attached to the deal, and a lead program: CRMA-1001, a liver-targeted epigenetic therapy intended for hepatitis B / D treatment.  

Eligo Bioscience 

• Founded: 2014 
• Headquarters: Paris, France 
• CRISPR focus: Microbiome editing 

Eligo Bioscience is a French company founded in 2014. The company focuses on precision gene editing of the microbiome to treat diseases driven by bacterial genes. Eligo Bioscience leverages its proprietary Gene Editing of the Microbiome (GEM) platform to develop therapies that target and modify specific bacterial populations. In 2023, Eligo Bioscience raised $30 million in a series B funding led by Sanofi Ventures. 

The company’s GEM platform uses engineered bacteriophages to deliver CRISPR-Cas systems directly to specific bacteria within the microbiome. This approach allows for the precise elimination of pathogenic bacteria or the correction of harmful bacterial genes without disrupting the overall balance of the microbiome. By targeting bacterial genes in vivo, Eligo’s technology aims to address various diseases associated with microbiome dysbiosis, including antibiotic-resistant infections and chronic diseases. 

Unlike broad-spectrum antibiotics, which indiscriminately kill bacteria and disrupt the microbiome, Eligo’s technology selectively targets pathogenic bacteria or genes within the microbiome. This precision reduces collateral damage to beneficial bacteria, which helps maintain a healthy microbiome. 

Beyond GEM, Eligo also has two other modalities in its pipeline: 

• SSAM (Sequence-Specific Antimicrobials): Here, the CRISPR payload acts more like a bactericidal agent, introducing double-strand breaks in targeted bacteria’s DNA to kill them, but only in those carrying the target sequence. 

• FAME (Function Addition to the Microbiome): With this modality, the vector carries a gene cassette that allows a bacterium to express a therapeutic function. 

Intellia Therapeutics

• Founded: 2014 
• Headquarters: Cambridge, Massachusetts, U.S. 
• CRISPR focus: In vivo gene editing 

Intellia is one of the most advanced companies working on in vivo CRISPR therapies. Its strategy centers on delivering CRISPR/Cas9 machinery systemically into patients, usually with lipid nanoparticles, to knock down disease-causing genes directly inside organs such as the liver. 

The company’s first-in-human CRISPR program was nexiguran ziclumeran (nex-z, formerly NTLA-2001) for transthyretin amyloidosis (ATTR). Nex-z was the first systemic in vivo CRISPR therapy to be tested in people back in 2021. Phase 1 results shared last week have shown that a single dose can lead to over 90% reductions in transthyretin protein levels, with effects lasting for years and accompanied by stabilization or improvement of disease symptoms. 

But Intellia’s most advanced program in the clinic is lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002), which targets hereditary angioedema (HAE). This candidate is already in a phase 3 trial, with the goal of permanently reducing the frequency of painful swelling attacks in HAE patients. The company completed enrollment in 2025 and expects to file for regulatory approval as early as 2026. 

On the business side, Intellia has long collaborated with Regeneron and holds a broad in vivo editing pipeline. Like Caribou Biosciences, from which it licenses some CRISPR/Cas9 IP, the company announced a reorganization, focusing its resources on nex-z and lonvo-z while scaling back earlier programs, including workforce reductions of about 27%. 

Mammoth Biosciences 

• Founded: 2017 
• Headquarters: Brisbane, California, U.S. 
• CRISPR focus: Ultra-small CRISPR systems (Cas14, CasΦ) 

Mammoth Biosciences is best known for its work on compact CRISPR nucleases, particularly Cas14 and CasΦ, which are significantly smaller than the standard Cas9. The company’s bet is that these smaller nucleases will make it easier to deliver gene-editing tools into a wider range of tissues, a critical hurdle for moving beyond liver-targeted therapies. 

The company originally gained visibility through its CRISPR-based diagnostics platform, DETECTR, which helped establish its commercial footing. In recent years, however, Mammoth has shifted more decisively toward therapeutics, positioning itself as a key innovator in in vivo editing. Unlike some of its peers, Mammoth has not singled out one advanced lead candidate. Instead, it has concentrated on building partnerships that combine its nucleases with external expertise in delivery technologies. 

A major example came in 2024, when Mammoth entered a collaboration with Regeneron. The deal, valued at around $100 million in upfront equity and cash, gave Regeneron access to Mammoth’s compact CRISPR systems, while Mammoth gained Regeneron’s experience in developing delivery platforms such as viral vectors and antibody conjugates. The goal of the collaboration is to create editing therapies that can reach tissues outside the liver, where many genetic diseases remain untreatable. 

Mammoth’s fundraising history is also quite impressive. The company achieved unicorn status in 2021 after raising $150 million in a series D round, adding to earlier financings.  

Prime Medicine 

• Founded: 2019 
• Headquarters: Cambridge, Massachusetts, U.S. 
• CRISPR focus: Prime editing 

Prime Medicine is pioneering prime editing, a CRISPR-derived technique designed to rewrite DNA sequences more precisely than traditional Cas9. Instead of cutting both DNA strands, prime editing makes a single-strand nick and uses a reverse transcriptase fused to a Cas protein to integrate a corrected sequence. The goal is to repair mutations with fewer errors or collateral changes. 

The company’s lead program, PM359, targets the p47phox form of chronic granulomatous disease, a rare inherited immunodeficiency caused by mutations in the NCF1 gene. In May 2025, Prime reported the first human data showing that prime editing can correct the underlying defect and restore immune function. The first patient in the trial achieved 58% neutrophil activity by day 15 and 66% by day 30. Engraftment was rapid, with neutrophils returning by day 14 and platelets by day 19, and there were no serious adverse events linked to the therapy. Prime described these results as the first clinical proof that prime editing can be both effective and safe in people. 

Even as it showcased these breakthrough findings, the company announced a strategic shift: it will consolidate from a broader pipeline of nearly 18 programs to just four core areas, and cut about 25% of its workforce. 

The market has responded positively. Over the past six months, Prime’s share price has more than doubled, including a 22% jump in a single week following the release of the data. Major backers such as Google Ventures increased their holdings, and ARK Invest added shares as well.  

Scribe Therapeutics 

• Founded: 2018 
• Headquarters: Alameda, California, U.S. 
• CRISPR focus: Engineered CRISPR systems for therapeutic use 

Scribe Therapeutics is built around the idea of designing new CRISPR enzymes from the ground up rather than relying on Cas9 or Cas12. Its platform, known as XE or “X-Editor,” produces enzymes optimized for precision, potency, and easier delivery into the body. Alongside this, its ELXR technology applies CRISPR tools for epigenetic silencing, switching off genes without permanently altering the DNA sequence. 

The company’s leading program, STX-1150, targets PCSK9, a gene involved in regulating cholesterol. By silencing PCSK9 in the liver, the therapy aims to reduce LDL cholesterol levels durably, providing a new way to treat cardiovascular disease. A second program, STX-1400, is designed to lower triglycerides by knocking down APOC3, another key lipid regulator. In preclinical studies, Scribe has reported strong reductions in both cholesterol and triglyceride levels, including durable effects in nonhuman primates. 

Scribe has attracted major partners such as Sanofi, which is collaborating on CRISPR-engineered NK cell therapies for cancer, and Prevail Therapeutics, a Lilly subsidiary developing neurological and neuromuscular therapies.  

Recent updates show both progress and challenges. Scribe has unveiled preclinical data on its CasXE-based therapy for durably lowering Lp(a), STX-1200, which achieved over 90% knockdown, while also reducing its workforce by about 20% to streamline operations as it prepares for its first clinical trials.  

Tessera Therapeutics 

• Founded: 2018 
• Headquarters: Somerville, Massachusetts, U.S. 
• CRISPR focus: Gene Writing 

Tessera calls what it does “Gene Writing,” a next-gen approach aimed at going beyond the limitations of gene editing tools like CRISPR. Instead of cutting DNA and relying on cell repair mechanisms, Tessera engineers systems derived from mobile genetic elements (MGEs) to insert, replace, or rewrite DNA sequences in a more controlled way. Its toolkit includes RNA Gene Writers, DNA Gene Writers, and Gene Rewriters, each tuned for different types of edits, from single-base substitutions to insertion of full genes. 

These writers use mechanisms such as target-primed reverse transcription, where the system nicks a DNA strand and uses an RNA template to synthesize a new DNA sequence at the target site. This approach has the potential advantage of avoiding double-strand DNA breaks and less reliance on cellular repair pathways, which are often error-prone. 

In terms of disease areas, Tessera has disclosed promising preclinical results in alpha-1 antitrypsin deficiency (AATD). In multiple mouse models carrying human AATD mutations, its RNA Gene Writers achieved corrections in whole liver tissues at around 49 to 55% at relatively low doses. Those correction levels reportedly exceeded what you’d see in asymptomatic heterozygotes, and they translated into increases in functional (wild-type) A1AT protein in serum. Tessera is also exploring other liver targets, PKU (phenylketonuria), sickle cell disease, and even in vivo engineering of CAR T cells. 

On the financing and strategic side, Tessera has attracted large funding rounds. It raised more than $300 million in its series C in 2022 though it still operates mostly in the preclinical and exploratory domain. 

Verve Therapeutics 

• Founded: 2018  
• Headquarters: Cambridge, Massachusetts, U.S. 
• CRISPR focus: In vivo base editing 

Verve Therapeutics is among the pioneers aiming to bring gene editing to cardiovascular disease, a large, population-level target rather than a rare genetic disorder. Its strategy relies on in vivo base editing in the liver to permanently silence genes associated with cholesterol and lipid risk. 

Its lead program, VERVE-102, is designed to edit the PCSK9 gene in liver cells. PCSK9 is a well-validated target for lowering LDL cholesterol. Verve delivers mRNA encoding a base editor and a guide RNA via a custom GalNAc-LNP delivery system. The idea is that a single infusion will produce a precise base change that inactivates the PCSK9 gene, leading to lifelong reduction in LDL levels. Verve describes this as a “single-course” therapy. 

In April 2025, Verve announced initial data from its phase 1b trial of VERVE-102. The treatment was well-tolerated, with no treatment-related serious adverse events. At the highest dose cohort, patients showed LDL-C reductions of 53%, with a maximum observed reduction of 69%. Earlier, the company had paused enrollment of an earlier version of the therapy (VERVE-101) after a patient experienced elevated liver enzymes and lowered platelet counts; Verve says it improved the delivery vehicle (LNP) in VERVE-102 to address that. 

Verve’s story has also seen turbulence. Its collaboration with Vertex, which focused on gene editing for liver disease, was terminated in February 2025, with rights reverting to Verve.  

The company also gained major attention in mid-2025 when Eli Lilly announced a deal to acquire it for approximately $1 billion. Under the deal, the milestone payments are tied to progress, such as dosing a patient in a phase 3 trial of VERVE-102. 

CRISPR technology’s growth driven by both progress and pressure 

The CRISPR field has never been more dynamic. The global market was valued at nearly $3.8 billion in 2024 and is projected to nearly double by 2029 to reach $7.5 billion. Companies like Vertex and CRISPR Therapeutics have shown that CRISPR-based medicines can reach patients, and newer entrants are pushing the technology into broader disease areas. 

Yet behind the headlines of clinical progress and billion-dollar partnerships lies a harsher reality. Several leading CRISPR companies, including Intellia, Prime, and Scribe, have all cut staff or pared down their pipelines in the past year. These moves aren’t necessarily signs of failure but they reflect the brutal economics of drug development. Editing tools may be powerful, but they are costly to develop, and investors now expect programs to focus on clear, near-term value rather than extensive portfolios. In that sense, CRISPR is maturing and companies are transitioning from platform plays built on technological promise to leaner organizations judged on clinical milestones. 

On top of economic pressure, the field still faces significant hurdles. Manufacturing and delivery remain costly bottlenecks. At the same time, entirely new modalities, from epigenetic editing to “gene writing” platforms like those at Chroma Medicine and Tessera Therapeutics, are emerging. 

The result is a CRISPR landscape that feels both triumphant and precarious. The technology is proving itself in the clinic, but survival in this space requires discipline. The companies that succeed will likely be those that pair scientific innovation with the hard choices needed to sustain momentum in an unforgiving biotech market. 

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