The Gene Editing Revolution — Technology, Companies, and Outlook (2025), Part II
CRISPR: A Breakthrough on Par with Antibiotics and DNA — Are We One Step Away from Cures for Cancer and HIV?
Informational and analytical only — not investment advice.
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liquidity
Haven’t read the first part yet? You can find it here 📬
In the first part, we outlined the fundamentals of CRISPR-Cas9 and the pioneers who were the first to bring this technology to the public markets. Now, we take a look at the next wave of players and summarize the clinical results from 2023–2025.
The Landscape of the Gene Editing Industry – New Players
The story doesn’t end with the first pioneering companies. Soon after, new firms emerged — some focusing on therapies for additional diseases, and others advancing next-generation CRISPR technologies.
Beam Therapeutics — Base Editing: Less Cutting, More Meaning
Beam Therapeutics is a company founded in 2017 by scientists from MIT and Harvard, including the eminent chemist David Liu, the inventor of base editing. Beam’s mission is to apply base editors in therapy—that is, to modify single nucleotide pairs without cutting both strands of DNA. Base editors are fusion proteins composed of a “crippled” Cas nuclease (which binds DNA at a specified site but does not cleave it) and a base-modifying enzyme (e.g., a deaminase that converts one base into another). As a result, one can make, for example, a C→T or A→G substitution at a chosen site in the genome[8]. Although small, such a change can be sufficient to repair many disease-causing mutations. Crucially, no dangerous double-strand break is created, which reduces the risk of adverse effects (e.g., chromosomal rearrangements or oncogene activation)[8]. Beam Therapeutics is developing an entire platform of different editors (cytosine, adenine, etc.) to tailor them to various diseases.
Like other companies, Beam first targeted hemoglobin-related blood disorders. Its BEAM-101 program focuses on sickle cell disease—but instead of cutting the BCL11A gene as CRISPR Tx or Editas do, the base editor changes a single DNA letter to activate the fetal hemoglobin gene. The end goal is the same: raise HbF levels and eliminate disease symptoms[8][64]. In January 2024, Beam dosed the first patient with BEAM-101. In 2025, the company presented initial clinical data (sustained HbF increase with no new safety signals) and obtained RMAT status from the FDA for BEAM-101 (August 2025), which accelerates the regulatory pathway. It is worth noting that a competing company, Sangamo Therapeutics, is testing an analogous HbF-boosting method using zinc-finger nucleases—its earlier results were mixed. If Beam demonstrates a clear effect with minimal complications, it may outpace that older technology.
Beam is also active in oncology. In 2022, a team in the UK (Great Ormond Street Hospital) described a case of a teenage patient with refractory T-cell leukemia who received CAR-T lymphocytes modified using base editing. It was the first therapeutic use of base editing—thanks to it, the donor T cells were altered so that they would not attack each other or be rejected, while targeting the CD7 marker on leukemia cells [65][66]. The patient (Alyssa) achieved remission and was then able to undergo a definitive bone marrow transplant, which saved her life [65][67].. This case study demonstrated the power of base editing to create “enhanced” cells to fight cancer. Beam Therapeutics collaborates with these clinicians and, in parallel, has initiated its own clinical trial, BEAM-201—an allogeneic anti-CD7 CAR-T for T-cell leukemia (targeting CD7, as in Alyssa’s case). The product includes several edits (e.g., removal of TCRs so the cells do not attack the patient, and receptor edits to avoid rejection and self-destruction)[68]. The first preliminary ASH 2024 data for BEAM-201 indicated activity signals for this multiply edited, allogeneic anti-CD7 CAR-T—enrollment and updates continue in 2025.
Beam is also developing in vivo therapies. Here, however, instead of CRISPR-Cas9, it uses base editors delivered via LNPs. The company has had programs in dyslipidemias (lowering cholesterol by editing PCSK9 or ANGPTL3), but Verve Therapeutics has pulled ahead in this area (more on Verve below). Beam has, however, signed a major partnership with Pfizer—to apply base editors in the liver, muscle, and CNS. This implies potential in areas such as Duchenne muscular dystrophy or CNS diseases, though these are still in the preclinical stage.
Strengths of Beam: As a pioneer of base editing, the company possesses CRISPR 2.0 technology that can solve some of the pain points of the classical approach. Base editors enable correction of point mutations without introducing DNA breaks—an enormous advantage where a subtle fix suffices instead of cutting out a gene. They can also target genes where a cut could be lethal but a small change is enough (e.g., converting a pathogenic codon into a stop codon). Beam has strong scientific backing—rooted in Liu’s work—with key patents in the field. It is therefore a dominant player in base editing and a potential technology provider to others (several firms have taken licenses). In addition, Beam has strategically broadened its portfolio: it operates in genetic blood disorders, oncology, and cardiovascular indications (in collaboration with Verve), and it has a major deal with Pfizer—providing diversification. Financially, Beam amassed substantial funds during the boom (its IPO and follow-on offerings brought in hundreds of millions of dollars), which still serves as a cushion. If any program proves successful, Beam’s value could rise quickly.
Weaknesses of Beam: The biggest drawback is the lack of human efficacy data—Beam is only just beginning patient trials. Others already have therapies on the market or in advanced stages, while Beam must demonstrate in practice that base editing offers advantages beyond theory. There is also the safety question—although there is no double-strand break, base editors can cause unintended base changes within a certain DNA “window” (biochemical off-targets—they may alter similar sequences or cause mosaicism). Work is ongoing to improve enzyme specificity, but only clinical trials will show how clean these edits are. Furthermore, base editors are limited to certain substitutions (e.g., C→T), meaning not every mutation can be repaired with this method. In sickle cell disease the goal is not a specific mutation but gene silencing (which base editing achieves indirectly), so it fits here. In other diseases, however, the editor may be powerless if a more complex fix is required—prime editing (a competing technology) may be better in such cases.
In sum, Beam is valuable but still a “prove-it” company. The next 1–2 years with first patient results will be critical. If strong effects emerge (e.g., durable cures in additional SCD patients or an effective CAR-T in T-cell leukemia), investor attention and capital may shift toward Beam as the next genetics star.
Caribou – universal CAR-T
Caribou Biosciences is a Berkeley, California–based company co-founded by Jennifer Doudna in 2011 (initially as a research tool company) and later transformed into a therapeutics firm. Caribou stands out for its proprietary modifications to the CRISPR system—it developed its own chimeric guides (chRDNA) designed to increase editing precision and reduce off-target risk. The company has focused primarily on cancer immunotherapy, developing allogeneic (donor-derived) CAR-T cells similar to CRISPR Therapeutics, but leveraging its gene-editing technology to produce better cells.
Caribou’s lead candidate is CB-010—CAR-T lymphocytes targeting CD19 (expressed on B-cell lymphomas) with two edits: (1) deletion of the TRAC gene (the T-cell receptor) to prevent donor cells from attacking the recipient’s body, and (2) PD-1 knockout in the lymphocytes to prevent their “shutdown” by tumor signals [70]. This second edit is novel—PD-1is an inhibitory molecule often exploited by tumors to suppress immune attack. Standard autologous CAR-T cells can still succumb to this mechanism, but in Caribou’s product PD-1 “doesn’t work,” allowing the cells to remain active longer. Phase 1 results (16 treated patients with refractory non-Hodgkin lymphomas) were very promising: 15 of 16 patients responded to treatment, including 7 who achieved complete remission lasting ≥6 months [70][71]. In one patient, remission lasted over 24 months (two-year follow-up)[72]—the longest documented durability to date for an allogeneic CAR-T therapy. These data led the FDA to grant RMAT, Fast Track, and orphan drug designations to CB-010, which should accelerate its development [73][74]. Caribou announced plans to skip Phase 2 and proceed directly to a pivotal Phase 3, aiming to start by the end of 2024 [75]. Meanwhile, enrollment of ~30 additional patients continues in Phase 1 to select the optimal dose for Phase 3 [76]. It’s an aggressive but exciting strategy—if successful, CB-010 could become the first off-the-shelf CAR-T therapy for lymphomas, significantly simplifying and speeding up treatment (current autologous CAR-T requires individualized manufacturing over several weeks for each patient).
Caribou isn’t stopping at one product. In 2023 the company launched two additional Phase 1 trials: CB-011—a CAR-T targeting BCMA in multiple myeloma (here, in addition to standard edits, an MHC class I modification was added so the cells evade the recipient’s immune response) and CB-012—a CAR-T targeting CLL-1 on AML leukemia cells [77]. Both programs employ advanced gene edits and aim to improve outcomes in hard-to-treat cancers (myeloma and AML). Results will take time—initial safety readouts are expected in 2025.
Strengths of Caribou: The company has some of the best clinical results in off-the-shelf CAR-T—remissions in nearly half of patients at ≥6 months are comparable to autologous CAR-T and better than most experimental allogeneic CAR-T products, which often produced only short-lived responses [32][78]. The PD-1 edit appears to work, an important proof that multi-gene engineering can translate into better therapies. Caribou also has a strong IP position—its chRDNA(hybrid RNA-DNA guides) allow for more precise DNA cutting with a lower error risk, which can be licensed or provide a safety advantage. Its pipeline is thoughtfully sequenced: starting with lymphomas (easier CAR-T targets) to build know-how, then moving into increasingly difficult cancers. The company has also expressed ambitions beyond oncology, having signaled early work on in vivo editing for genetic diseases.
Weaknesses of Caribou: Like any cell-therapy company, it faces challenges in scaling manufacturing and controlling costs. Although allogeneic CAR-T is intended to be cheaper than individualized products, it still involves complex cell-engineering processes requiring standardization. Competition in immunotherapy is intense: other companies are developing universal CAR-T (e.g., Allogene Therapeutics using TALEN technology), as well as newer modalities like NK-cell or TCR-based therapies. Caribou must maintain a technological edge—planning a Phase 3 is encouraging, but the FDA could still request additional Phase 2 data if concerns arise. Long-term safety is also crucial in CAR-T: cytokine release syndrome, opportunistic infections, etc., can occur—Caribou has recorded such events (as in any immunotherapy), e.g., infections in some patients due to temporary immunosuppression [67][80]. These effects are serious but acceptable in life-threatening diseases. To surpass autologous CAR-T, Caribou must demonstrate at least comparable efficacy with better access/cost. Finally, financially Caribou is relatively small—the funds from its IPO can be consumed within 1–2 years of intensive trials. It may need a partner (e.g., for global commercialization if approved) or additional equity raises, which introduces uncertainty for investors.
Bottom line: Caribou has done an excellent job so far and is often cited as a “dark horse” of the CRISPR race—a company that could achieve major success in the oncology niche.
The rest of the field: from “holy grail” to “lipid reset”
Prime Medicine was founded in 2021 by David Liu and colleagues to develop prime editing, a next-generation form of genome engineering. Although younger and not yet in the clinic, the company deserves attention because prime editingis often regarded as the “holy grail” of gene editing. This method combines a modified Cas9 (which cuts only one DNA strand) with a reverse transcriptase enzyme, enabling the precise insertion, deletion, or replacement of virtually any DNA sequence at a chosen genomic site [9]. In theory, it can correct mutations that neither base editing nor classical CRISPR can fix—such as large deletions or insertions.
Prime Medicine went public in 2022, raising capital to support its research programs. So far, its work remains preclinical, as the company selects its first therapeutic targets. Nevertheless, 2025 brought the first real-world glimpse of prime editing’s potential: physicians in the United Kingdom reported the case of a teenage patient with a rare DNA-dependent protein kinase deficiency, whose own T cells were edited ex vivo using prime editing to repair the mutation [9][10]. The results were positive—his immune function improved [10]. This marks the first evidence that prime editing can work not only in vitro, but also in living human tissue.
In the coming years, Prime Medicine plans to advance its first therapies into clinical trials. Despite not yet having any approved products or human data, the company was valued at approximately USD 1.1 billion in 2025 [43] — a reflection of the transformative potential investors see in this “CRISPR 3.0” platform.
Verve Therapeutics aims to prevent cardiovascular disease by editing genes in otherwise healthy but at-risk individuals. Verve focuses on genes that regulate cholesterol and lipid levels. Its first therapy, VERVE-101, uses a base editor delivered in LNP nanoparticles to the liver to knock out the PCSK9 gene. People with naturally inactive PCSK9 have very low levels of “bad” LDL cholesterol and are essentially protected against heart attacks. Verve reasoned that instead of daily statins or periodic antibody injections, one could lower LDL once and for all through gene editing. In 2022, the company began a Phase 1 study in New Zealand and the UK in patients with genetically driven hypercholesterolemia (HeFH). The first results, presented in 2023, showed that in three patients who received ascending doses, LDL fell by 40–55%, and this effect persisted for at least half a year [81]—more than is typically achieved with statins [82]. At the same time, plasma PCSK9 levels nearly disappeared—evidence that the gene edit worked [83]. Unfortunately, the study also saw serious incidents: two participants experienced cardiovascular events (including one death), but analysis indicated these stemmed from pre-existing advanced coronary disease[84]. One person may have had a therapy-related adverse effect (cardiac arrhythmia), though this is uncertain[84]. These events prompted caution from the U.S. FDA, which initially withheld permission for U.S. sites to join the trial, requesting additional safety data (especially regarding any potential editing in germ cells) [85]. Verve supplied those data, and in October 2023 the FDA lifted the hold, allowing U.S. participation [86]. The company plans in H2 2025 to start a placebo-controlled Phase 2 for VERVE-102, after completing dose escalation in Heart-2 and obtaining regulatory approvals.
What’s more, Verve is developing a second editor (VERVE-201) that knocks out ANGPTL3, which lowers triglycerides and other lipids. Both genes (PCSK9, ANGPTL3) target relatively small patient subsets (primarily those with familial mutations), but Verve is thinking ahead: in the future, once safety is proven, such one-and-done therapies might be used prophylactically in a broader high-risk population. That scenario is distant but not impossible—Fyodor Urnov draws an analogy: statins were first tested in patients with genetic hypercholesterolemia, then in those with heart disease, and finally became a mass preventive therapy [87][88].
Verve and CRISPR Tx are taking similar paths. In 2023, CRISPR Therapeutics launched its own in vivo programs editing ANGPTL3 (CTX310) and Lp(a) (CTX320). In September 2025, the company presented first data—a single dose of CTX310 strongly reduced ANGPTL3 and lipids, and CTX320 lowered Lp(a).
In the future, this could unlock a huge market: a single treatment that simplifies control of cholesterol or other risk factors, potentially for millions of people [91][92]. Of course, the precondition is demonstrating long-term safety—the bar is highest here because we intervene in people who might otherwise live for decades and who are relatively healthy at the time of therapy.
Sangamo Therapeutics and Cellectis—worth mentioning as pioneers of the previous generation, still active today.
Sangamo (USA) has developed zinc-finger nucleases (ZFNs) since the 1990s. It conducted some of the first human gene-editing trials—for example, in 2017 it delivered ZFNs directly to the liver of patients with Hunter syndrome (MPS II), attempting to repair the IDS gene. Unfortunately, the therapy did not yield meaningful improvement (enzyme levels did not rise significantly). Sangamo also tested gene editing in T cells to treat HIV (modifying the CCR5 receptor, analogous to what He Jiankui did in embryos)—some patients temporarily suppressed the virus, but it found other ways to persist. Sangamo has since shifted toward using gene editing to create regulatory T cells (CAR-Treg) to dampen autoimmune disease and in transplantation. It also has partnerships (e.g., with Pfizer on PCSK9 editing, though Pfizer has now prioritized base editing with Beam). With the oldest technology, Sangamo suffered after CRISPR’s rise—its market cap fell sharply, and the company is fighting for niches where it can remain competitive (e.g., epigenetic editing instead of direct DNA cuts).
Cellectis, meanwhile, is a pioneer of TALEN. Its editing method underpinned the first autologous CAR-T therapies (developed in Europe for a young leukemia patient as early as 2015). Today, Cellectis, in collaboration with U.S. firm Allogene, is developing allogeneic CAR-T for lymphomas and myeloma, using TALEN to knock out receptors. Allogene’s clinical results have been moderate—there were responses, but also issues (e.g., in 2021 the FDA placed a hold due to chromosomal aberrations in modified cells, which were ultimately deemed benign clonal events). Cellectisalso has its own Phase 1 CAR-T programs. Overall, however, TALEN is more labor-intensive, and in the era of CRISPR/base editing/TCR, it faces the challenge of proving its continuing value.
Note on Partnerships
The gene-editing industry runs on partnerships with Big Pharma. Vertex backs CRISPR Therapeutics, Regeneronsupports Intellia, and Pfizer has teamed up with Beam (having previously partnered with Sangamo). Novartis invested early in several startups, while Bristol Myers Squibb (BMS) collaborated with Editas. Eli Lilly has partnered with both Precision BioSciences and Verve Therapeutics, signaling an interest in expanding into cardiometabolic targets. Meanwhile, institutional investors such as ARK Invest and BlackRock have built significant exposure to the space.
These alliances provide capital, know-how, and access to manufacturing infrastructure, but they also come with trade-offs — reduced autonomy, rights of first refusal, and commercial dependencies that can narrow strategic flexibility.
As scientific risk declines, the spotlight shifts to execution quality and corporate governance. Inefficient management can undervalue a company relative to its scientific merit, or even erode liquidity, leaving it in a weakened negotiating position — a setup that often precedes unfavorable takeovers.
One could also say that Big Pharma’s investments in gene editing strengthen the credibility of the entire field — when the giants start betting real money, it’s no longer just a scientific dream but a sector maturing toward industrial scale.
Conclusion — Part II
In Part I, we saw how Editas, Intellia, and CRISPR Therapeutics opened the era of CRISPR commercialization. By now, one thing is clear: CRISPR is no longer a promise — it’s a bill — clinically payable, but still commercially negotiated. The next 24 months will determine who truly has the technology, and who merely has a presentation. Some will learn the gravity of capital, others — the scale of manufacturing and the patience of regulators.
With that, we conclude the mapping of the second wave of players. Part III (final) will no longer ask who holds the technology, but rather where the field stands clinically in 2023–2025, what approval milestones lie ahead, and what scaling, cost, and regulatory barriers remain. We’ll also ask the key question: who can realistically turn CRISPR from a promise into a fully-fledged market for precision medicine.
To be continued… and with it comes ✨
🧬 The Gene Editing Revolution — Part III (Final)
🦄💎 The Opening of the Unicorn Vault
“He peeked inside — and felt the magic of rarity, the magic of discovery.”
🦄💎 Unicorn Vault
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This material is provided for educational and informational purposes only. It does not constitute investment advice or an offer within the meaning of applicable law. The author bears no responsibility for investment decisions made on the basis of this content.
📚 Selected References
· U.S. Food and Drug Administration – press release on the approval of the first CRISPR therapy (Casgevy) for sickle cell disease [2][3].
· Innovative Genomics Institute, CRISPR Clinical Trials: A 2024 Update – overview of ongoing CRISPR trials, including results in SCD/TDT, ATTR, HAE, and CAR-T [111][48][53][70].
· STAT News, Jason Mast, The CRISPR companies are not OK (2025) – analysis of the sector’s challenges and the gap between scientific breakthroughs and financial success [1][40].
· Nanalyze, When Will Gene Editing Stocks Finally Take Off? (2025) – examination of correlations and valuations across gene-editing companies [109][43].
· Nature News, Heidi Ledford, World first: ultra-powerful CRISPR treatment trialled in a person (2025) – coverage of the first use of prime editing in a human patient [9][10].
· The Guardian, Ian Sample, Chinese scientist who edited babies’ genes jailed for three years(2019) – report on He Jiankui’s case and its ethical fallout [11][12].
· Fierce Biotech, James Waldron, Graphite’s hopes for sickle cell ‘cure’ blunted after first patient dosed experiences serious event (2023) – report on Graphite Bio’s halted trial [103].
· U.S. FDA – background on indications and mechanism of action for Casgevy and the parallel lentiviral therapy (Lyfgenia) [2][3].
(Sources: high-reputation English-language publications with up-to-date scientific and financial data as of August 2025.)
📖 References
[1] [40] [59] The CRISPR gene editing revolution loses its mojo. STAT News. (Feb 2025). Link
[2] [3] [4] [5] [18] [116] FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. FDA Press Release. (Dec 2023). Link
[6] [7] CRISPR Illustration. National Institute of General Medical Sciences. Link
[8] [13]–[15], [19]–[21], [22]–[39], [41], [44]–[49], [50]–[58], [61]–[63], [64]–[111] CRISPR Clinical Trials: A 2024 Update. Innovative Genomics Institute. (2024). Link
[9] [10] Ledford H. World first: ultra-powerful CRISPR treatment trialled in a person. Nature. (2025). Link
[11] [12] Sample I. Chinese scientist who edited babies’ genes jailed for three years. The Guardian. (Dec 2019). Link
[42] [43] [79] [109] When Will Gene Editing Stocks Finally Take Off? Nanalyze. (Jul 2025). Link
[60] Editas, changing course again, looks to partner lead CRISPR therapy. BioPharma Dive. (2024). Link
[103]–[106] Waldron J. Graphite’s hopes for sickle cell ‘cure’ blunted after first patient dosed experiences serious event.Fierce Biotech. (2023). Link
[110] Allogene Therapeutics Presents Updated ALLO-501/501A Phase 1 Data. ASCO Annual Meeting. (2024). Link
[111] Exploring FDG PET + MRI for multiple myeloma staging. J Clin Oncol. ASCO (2024). Link
[112] [113] [115] Caribou Biosciences Reports Q2 2025 Results and Pipeline Update. Caribou Investor Relations. (Aug 2025). Link
[114] CB-010 (ANTLER trial) clinical update. J Clin Oncol. ASCO (2024). Link
[117] Prime Medicine announces breakthrough clinical data (PM359, CGD). Prime Medicine Investor Relations. (2025). Link