CRISPR Stocks: The Second Wave of Gene Editing

Everyone remembers December 2023. The FDA approved Casgevy, the first CRISPR-edited therapy in history, for sickle cell disease. Vertex and CRISPR Therapeutics made the cover of every business publication. The stock ran. The narrative was simple: gene editing is here, and it works.

Two and a half years later, the story has moved on. Casgevy is approved, but the commercial ramp is slower than anyone expected. The second wave of gene editing companies is in the clinic now, and the investment thesis has shifted from “who has the first approval” to “who has the platform that scales.” That is a much harder question.

Here are the five CRISPR and gene editing stocks that matter in H2 2026, ranked by pipeline progress, cash runway, and platform moat. The ranking is not what you would expect.

1. CRISPR Therapeutics (CRSP): The Only One With Revenue

CRSP closed at $47.55 with a market cap of $4.6 billion. The company trades at roughly 40x its 2025 Casgevy revenue of $115.8 million. That sounds expensive, but the ramp is real. Casgevy generated $43 million in Q1 2026 alone, and Vertex confirmed more than 500 patients globally have initiated the treatment journey. Vertex projects combined Casgevy and Journavx revenue of $500 million in 2026, a 185% increase year-over-year.

The bottleneck has never been efficacy. It has been logistics. Each patient requires cell collection, ex vivo editing, myeloablative conditioning, and reinfusion. The process takes months. Vertex has activated 75 authorized treatment centers globally, which helps, but the patient journey from referral to infusion to revenue recognition still spans two to three quarters. The 300 patients who initiated treatment in 2025 are the pipeline for 2026 revenue.

At $2.2 million per patient, Casgevy is the most expensive drug in mainstream use. Reimbursement is slowly expanding, with coverage now secured in multiple European markets. But the addressable population is narrow. Sickle cell disease affects roughly 100,000 people in the US. Even if CRSP and Vertex treat 500 patients per year at full price, that is $1.1 billion in gross revenue before discounts. CRSP gets 40% under the collaboration agreement. At a $4.6 billion market cap, the stock is pricing in sustained ramp plus pipeline optionality from CRISPR’s CAR-T programs. The ramp is real but the multiple is full. Hold for the revenue trajectory, not for the pipeline.

2. Intellia Therapeutics (NTLA): The Real Bet on In Vivo Editing

NTLA closed at $10.89 with a market cap of $1.5 billion. This is the stock to own if you can only pick one gene editing company.

The thesis is simple: in vivo CRISPR is a bigger opportunity than ex vivo. Casgevy edits cells outside the body in a lab. Intellia’s nex-z (NTLA-2001) edits cells inside the body with a single IV infusion. If in vivo editing works at scale, it eliminates the manufacturing bottleneck that has crippled Casgevy’s launch.

The Phase 1 data is strong. A single dose of nex-z produced a 92% mean serum TTR reduction at 24 months across 33 patients. Among 18 patients with 24-month neuropathy assessments, 72% showed clinically meaningful improvement of at least 4 points on the mNIS+7 scale. The drug has Orphan Drug and RMAT designation from the FDA.

The problem is safety. The FDA placed a clinical hold on both Phase 3 trials in October 2025 after a patient in MAGNITUDE experienced Grade 4 liver toxicity. That patient subsequently died. The hold on MAGNITUDE-2 was lifted in January 2026, and the hold on MAGNITUDE was lifted in March 2026. Both trials are now enrolling again with enhanced liver monitoring protocols. MAGNITUDE is testing nex-z in approximately 1,200 ATTR-CM patients (NCT06128629). MAGNITUDE-2 is testing approximately 60 ATTRv-PN patients.

The BLA filing is expected by 2028. That means no revenue until 2029 at the earliest. But the competitive picture is favorable. The approved ATTR drugs, tafamidis (Pfizer) and acoramidis (BridgeBio), stabilize TTR protein. They do not stop production. Nex-z silences the gene permanently. If the Phase 3 data confirms the Phase 1 signal, Intellia has a best-in-class mechanism in a market projected to exceed $10 billion by 2030.

At $1.5 billion market cap with a Regeneron-funded program and a Phase 3 trial enrolling, NTLA is the most asymmetric risk-reward in this group. The binary is MAGNITUDE data, expected in 2027. If it hits, the stock doubles. If the liver toxicity signal worsens, the stock loses 60%.

3. Beam Therapeutics (BEAM): Base Editing’s Quiet Progress

BEAM closed at $26.29 with a market cap of $2.6 billion. Beam uses base editing, a CRISPR derivative that changes individual DNA letters without cutting the double helix. The science is elegant. The commercial question is whether it matters.

Beam’s lead program, risto-cel (BEAM-101), treats sickle cell disease by editing the gamma-globin promoter to increase fetal hemoglobin. The BEACON Phase 1/2 trial showed mean HbF levels above 60% and HbS below 40% post-treatment, with no investigator-reported severe vaso-occlusive crises after engraftment. The data was published in the New England Journal of Medicine in April 2026.

One patient died in the trial. Beam attributed the death to myeloablative conditioning, not to the gene editing itself. This is the same conditioning regimen Casgevy uses. The risk is known and shared across the field.

The problem for Beam is that it is entering a sickle cell market Casgevy already occupies. Risto-cel’s advantage is manufacturing: base editing produces high, predictable yields, and patients required a median of one stem cell collection cycle. That could mean lower cost of goods and faster patient throughput. But Vertex has a multi-year head start, 75 activated treatment centers, and the brand recognition that comes with being first.

Beam is also exploring in vivo base editing for sickle cell and beta-thalassemia. That program is preclinical. At $2.6 billion, the stock is pricing in risto-cel commercialization plus in vivo optionality. The in vivo program is where the real value is, but it is years away. The stock is a hold until in vivo data arrives.

4. Prime Medicine (PRME): Science First, Revenue Later

PRME closed at $3.09 with a market cap of $544 million. Prime editing is the third generation of gene editing after CRISPR and base editing. It can make all 12 types of point mutations and small insertions or deletions without double-strand breaks. The science is genuinely novel.

The financials are tight. Prime Medicine reported $149.2 million in cash as of March 31, 2026, with runway into 2027. The company secured a $110 million upfront payment from Bristol Myers Squibb in a strategic collaboration. That extends the clock but does not solve the fundamental problem: no clinical data yet.

The lead program, PM359, treats chronic granulomatous disease, an ultra-rare immunodeficiency. It is in preclinical development. The company expects to file an IND, but human proof of concept is at least a year away.

At $544 million, PRME is a spec play on platform technology. The BMS deal validates the science, but validation is not revenue. If you buy PRME, you are betting that prime editing produces clinical data that base editing and CRISPR cannot. That is possible. It is also unproven. Do not size this position for more than 2% of a portfolio.

5. Editas Medicine (EDIT): The Cautionary Tale

EDIT closed at $2.62 with a market cap of $411 million. This is what failure looks like in gene editing.

Editas discontinued development of reni-cel, its lead sickle cell program, after failing to find a partner. The company laid off 65% of its workforce, including the chief medical officer. The pivot is to in vivo gene editing, with a goal of human proof of concept in approximately two years.

The problem is that Editas had the same sickle cell opportunity as CRISPR Therapeutics and Beam. It had clinical data from the RUBY trial. It could not find a partner because the data was not differentiated enough to justify the commercial cost. That tells you something about the bar for gene editing programs. Good science is not sufficient. You need best-in-class data or a manufacturing advantage.

At $411 million, EDIT is cheap. But cheap is not the same as undervalued. The company has no clinical-stage programs, a gutted workforce, and a two-year timeline to first human data. The in vivo editing thesis is real, but Intellia is already there with Phase 3 data. Editas is late to its own pivot.

Risks

The sector risk is reimbursement. Every gene therapy company faces the same question: can payers sustain $2 million per patient pricing? Casgevy’s slow launch proved that approval is not the same as access. If CMS or commercial payers push back on pricing, every company in this group loses revenue projections.

The technology risk is off-target edits. CRISPR, base editing, and prime editing all carry the possibility of unintended genetic modifications. Intellia’s liver toxicity signal is a reminder that in vivo editing is still early. The FDA will require long-term follow-up data for any approved gene editing therapy.

The competitive risk is RNA therapeutics. Ionis and Alnylam can achieve similar protein reductions with antisense oligonucleotides and RNAi without permanent DNA modification. Their drugs are already approved and reimbursed. Gene editing’s advantage is durability: one treatment, lifelong effect. But durability is a claim, not a proven fact, until we have 10-year follow-up data.

Ranking

1. NTLA is the best risk-reward. Phase 3 enrolling, partnered with Regeneron, in vivo mechanism, $1.5 billion market cap. The binary is 2027 data.
2. CRSP is the safest hold. Revenue is ramping, partnership with Vertex is working, but the multiple is full at 40x revenue.
3. BEAM is a credible platform play. Base editing data is strong, but the stock needs in vivo proof to justify $2.6 billion.
4. PRME is a science bet. Prime editing is real, but clinical data is years away. Size accordingly.
5. EDIT is a warning, not an investment. The company had its chance and missed. The pivot to in vivo editing is the right strategic move, but it is two years late.

If you want exposure to gene editing, the biotech investing framework is straightforward: own the company with Phase 3 data and a partnered program. That is Intellia. Everyone else is either pre-revenue, pre-clinical, or pre-proof. For a broader look at the gene therapy space beyond CRISPR, see our gene therapy catalysts roundup. Gene therapies file BLAs, not NDAs, and the BLA vs. NDA distinction matters when you are timing catalysts. The PDUFA date for the next gene editing approval is not until 2028 at the earliest. Until then, the trade is about trial data, not FDA decisions.