rewrite this title Catching Up With 11 Gene Editing Stocks – Nanalyze

What if you could pay a reasonable amount of money to ensure your baby would never have an incurable disease? A recent paper suggests that using a technology called “base editing” would allow doctors to edit the genes of your future offspring to remove any genetic diseases prior to birth. All genetic diseases could be eliminated this way, but what about Down’s Syndrome or dwarfism? How about intelligence – not just addressing low IQ but perhaps raising it? Lots of debate can be had around these controversial topics, but there’s no question that gene editing – basically changing the recipe of life – is one of the most powerful technologies mankind has developed. The pioneering gene editing technique that started it all is called CRISPR.

It stands for Clustered Regularly Interspaced Short Palindromic Repeats, and it’s based on a system used naturally by bacteria to protect themselves from viruses by essentially “cleaving off” parts of their DNA. In 2012, scientists realized this technology could theoretically be used in humans, and they won a Nobel Prize for it. Twelve short years later, the first ever gene editing therapy hit the market and three leaders have emerged.

Three Gene Editing Leaders

CRISPR Sees Commercial Success

Aptly named CRISPR Therapeutics $CRSP became the first gene editing company to achieve commercial success. Their drug Casgevy targets hereditary blood disorders by altering a patient’s blood stem cells and restoring red blood cell function. This presents an opportunity to treat roughly 60,000 patients at $2.2 million a pop which is actually less than what the disease costs to treat over a patient’s lifetime. That’s why insurance companies will foot the bill for the lengthy and complicated “ex vivo” procedure which involves chemotherapy and blood infusions to extract stem cells from bone marrow so they can be edited and then infused back into the patient.

Since commercialization in 2024, Casgevy has treated 500 patients and generated $170 million in total revenue, $43 million of which came in Q1-2026. CRISPR has a 60/40 revenue split agreement with their partner Vertex Pharmaceuticals $VRTX, with the latter receiving 60%. That should mean CRISPR saw $17 million in Q1-2026 revenue, but they recorded just a tenth of that. Why?

CRISPR is recording these payments not as revenue, but as a reduction in “collaboration expense,” an operating expense affecting the company’s bottom line. This convoluted structure makes us wonder what happens when collaboration expenses hit $0. Do we then finally get to see some actual revenue? If so, we have about $46 million of reduced expenses to go, or $115 million in additional Casgevy sales.

Thankfully CRISPR has $2.4 billion in cash, giving them a runway of six years at their current $100 million per quarter burn rate from operating expenses. This includes nearly $600 million from the sale of convertible notes in Q1-2026. These are debt instruments that can be converted to shares, leading to potential dilution for current shareholders.

Outside of Casgevy, CRISPR has four other drug candidates in clinical trials right now: three wholly owned and one collaboration with privately held Sirius Therapeutics. Their wholly owned zugo-cel candidate is currently enrolled in two different Phase 1 trials for treatment of autoimmune diseases. Trial results are expected “in the second half of 2026,” so that’s something for investors to watch closely. Wholly owned candidates promise more benefit if successful, but they carry more risk.

The other three candidates are what’s known as “in vivo” approaches, where CRISPR treatments are delivered directly into a patient’s body as opposed to outside the body via a cell extraction. While ex vivo treatments are tedious, in vivo treatments promise less laboratory processing and a more simple and seamless patient experience – a few shots and you’re done. That brings us to the pioneer of in vivo CRISPR therapies: Intellia Therapeutics $NTLA.

Intellia Resumes Trials

Intellia currently has two key drugs in clinical trials: lonvo-z and nex-z which are both in vivo (the way forward). The former is wholly owned and treats a genetic disorder that causes swelling attacks. The latter is being developed with Regeneron Pharmaceuticals $REGN and can treat one of two hereditary disorders affecting the heart and other organs.

Lonvo-z is currently expected to launch commercially in the first half of 2027. Morningstar estimates that it could generate $1 billion in annual sales in the “later years” of their 10-year forecast period. As for Nex-z, Phase 3 trials have resumed after being halted last November. Unfortunately, a patient died after taking Nex-z, but the FDA determined it was due to outside factors and not the fault of Intellia. Trials resumed in January and are expected to be completed in 2028. Intellia currently has a cash runway through 2028 after a recent $180 million secondary offering in April 2026.

Beam Leans on Collaborations

Base editing promises cleaner edits with fewer side effects, and Beam provides the platform that makes that happen. The company makes it a point to emphasize that their commercial partnerships are seeing progression alongside their own internal development efforts. Pfizer $PFE is leading clinical development of an undisclosed drug while they partnered with Apellis (recently acquired by Biogen) on another new drug. Technology rights were sold to Eli Lilly $LLY who also purchased Verve last year which licensed Beam’s technology.

Investors in CRISPR will want to pay close attention to commentary in Beam’s latest investor deck that points to the challenges arising from current ex vivo therapies that result from limited manufacturing capacity and low process efficiency. Beam’s own risto-cel therapy is said to solve these challenges and consequently becomes a direct competitor to Casgevy which is one of the two approved gene therapies for sickle cell disease (SCD) alongside Bluebird Bio’s Lyfgenia which doesn’t use gene editing. When approved, Beam expects to offer a best-in-class ex vivo treatment with an in vivo treatment to follow (base editors would be delivered through intravenous infusion of lipid nanoparticles). In other words, this represents a two-pronged threat to CRISPR and their Casgevy drug.

With $1.2 billion in cash, Beam believes they’re fully funded into mid-2029 through the anticipated risto-cel launch. A key milestone for any drug is the biologics license application or BLA which could happen as early as year end for risto-cel.

Five Gene Editing Laggards

The three largest gene editing stocks we’ve talked about so far all surpass our one billion dollar market cap threshold, while the remaining names in our catalog don’t.

Editas is Still a Zombie

Since we exited Editas $EDIT four years ago, shares have fallen 72% as the company continues to kick the can down the road with a $300 million secondary offering in May and a market cap of just $500 million. Despite their origins as an intellectual property powerhouse, we ran for the exit when their key pharma partner backed out. Editas tried to run with the old “our pharma partner ditched us and we’re really stoked to go at it alone” explanation but that’s almost always a sign of underlying turmoil – not to mention the revolving door of C-level exits that continued even after we bailed. With a cash runway through 2027 and a lead candidate that hasn’t even been tested in humans yet (slated for later this year), the company just keeps spinning wheels.

Caribou Burning Cash

Caribou Biosciences $CRBU is an early gene editing pioneer with their own proprietary version of CRISPR called chRDNA.

Their goal was to increase specificity in the gene editing process by utilizing both DNA and RNA reads, but it hasn’t resulted in much progress. They have just two candidates in Phase 1 trials and are burning cash like mad. They used up roughly $100 million in cash in the past four quarters and are sitting on just $118 million today. With a market cap of $180 million, the market is clearly not confident about Caribou’s future prospects, and neither are we. While they’ve been advertising the recent positive data from their blood cancer (CAR-T) candidates, with over 80% overall response rate (how many patients see shrinking tumors), they still have to clear Phase 2 trials, where most drugs crash and burn. They also have a history of delayed trials, which is the reason we started avoiding the company in the first place. They only have a short cash runway through 2027, and raising capital will be difficult for this minuscule company.

Prime is Going Nowhere Fast

Google-backed Prime Medicine $PRME had a lot of promise with their patented “search and replace” gene editing technology which was said to be able to target 90% of all human genetic conditions. Their appropriately named “Prime Editing” technique builds on traditional CRISPR technology while adding additional precision, avoiding “double strand breaks” which can happen with traditional CRISPR editing and can cause unintended side effects.

Their lead candidate PM359 recently entered clinical trials for a rare white blood cell disorder called granulomatous disease. As their $135 million cash stockpile won’t get them very far in the expensive trial process, they’re seeking a new pharmaceutical partner – or buyer – to continue development. Their only other promising candidate is PM577a, an in vivo treatment for a specific variant of Wilson’s Disease which has afflicted roughly 10,000-15,000 patients. It only recently got regulatory approval in New Zealand to begin pre-clinical studies, so there’s not much to see here yet.

The bright side for Prime is their sugar daddy pharma partner, Bristol-Myers Squibb $BMY. Back in 2024, BMS gave Prime a $55 million cash payment and $55 million investment in exchange for use of their gene editing technology. While Prime has the potential to earn “up to $3.5 billion” in future royalty payments, this is completely dependent on Bristol-Myers Squibb passing the rigorous clinical trials needed to commercialize this complex ex vivo treatment. BMS currently has 50 drugs in development, meaning Prime needs BMS way more than BMS needs Prime.

Precision Biosciences

It’s now been 20 years since Precision Biosciences $DTIL was founded around their ARCUS gene editing platform which claims to have advantages over CRISPR methods such cleaner and more complex edits. We last covered the company seven years ago when they went public and noted their exclusive license with Gilead $GILD to develop a treatment for Hepatitis B. A year later, that relationship was terminated and Precision moved forward with the HBV program independently where it sits today in Phase 1 trials. Servier, a large private French pharmaceutical company, also bailed on Precision since their IPO and so did Eli Lilly (via subsidiary Prevail Therapeutics) which walked away and returned their assets to Precision.

Another deal with Novartis that spawned in 2022 has since been dissolved which means numerous large pharma partners have seemingly found no value in the platform. It’s a $200 million company with $115 million in cash which means the market also ascribes minimal value to their platform and so do we.

Cellectis SA

Cellectis $CLLS is one of the original gene-editing pioneers with their proprietary gene editing platform called TALEN and the company began trading in the USA over a decade ago. So when will we finally see a drug commercialized? The soonest would probably be their most advanced candidate lasme-cel which is expected to see a BLA application by 2028. Late this year they expect to see the first interim analysis of the “pivotal” Phase 2 trial. Note that the word “pivotal” refers to the definitive proof of safety and efficacy that the FDA needs for approval. While this usually happens in Phase 3 trials, it can happen earlier for rare diseases or small patient populations.

With $188 million in cash, they’re expected to have runway through 2027 before they’ll have to raise capital by issuing debt (current debt sits at $76 million), diluting shareholders, or hitting some payable milestones to provide working capital. Oftentimes drug developers will raise money when concurrently announcing positive trial progress which implies any negative news can be problematic for survival. With a market cap of just $234 million, the market isn’t placing much value on the business when you subtract cash. For such a small company, the trajectory often continues downwards until the business has no choice but to declare bankruptcy which is a good segue into our next three gene editing stocks.

Three Gene Editing Losers

Nothing good ever happens at a bar after 2:00AM, and nothing good ever happens to companies with market caps that fall below $100 million. Small does not equal a bargain, because if there were value to be found, any large pharma company could acquire if for a pittance. The below three companies will be removed from our catalog because they’ve gotten far too small for us to bother with.

Metagenomi (Slowly) Heads for Bankruptcy

After just two short years as a public company, Metagenomi $MGX has gone from a $600 million company to a sub-$50 million company. Turns out the whole “we’ll use AI to discover new gene editing tools” value proposition didn’t hold much water. While they currently have more cash on hand than their total market cap, they burned about $100 million last year with no drugs in clinical trials and none expected to be until 2027. Drug development is expensive, and Metagenomi will likely need to raise cash quickly to progress their lead candidate. With easy funding drying up and their share price at a historic low, Metagenomi’s odds of survival look bleak, which explains the “free” company. Seems like they’re inevitably going to pull a Sangamo.

Sangamo Therapeutics

Last week Sangamo $SGMOQ filed for bankruptcy providing a good example of what can happen to companies when large pharma partners back out. Despite positive Phase 3 trial results, Pfizer backed out of their relationship with Sangamo last year. Now it seems like Eli Lilly might buy what assets are left for $50 million or so which doesn’t do much for shareholders who lost 88% in the last year alone.

Synlogic

The Synlogic story starts with Mirna Therapeutics, a company that avoided bankruptcy by merging with a synthetic biology company called Synlogic $SYBX that had partnerships with Abbvie and Gingko Bioworks $DNA. After bringing a drug candidate to Phase 3 trials they ended up discontinuing their efforts and announced plans to wind down the operation and eliminate 90% of their staff. Why this eight million dollar company still trades is a mystery to us. All that’s left to do is add their name to the long list of synthetic biology stocks that have failed miserably.

Breaking News

Right before publishing this piece we noted that gene editing company Scribe Therapeutics has filed for an IPO (S-1 here). The company was founded by gene editing pioneer Jennifer A. Doudna and plans to (wait for it…) use AI to “optimize and tailor CRISPR technologies.” Should the IPO go through, we’ll give it a deeper analysis when we check in with gene editing stocks a year from now (or sooner if some major event happens).

Conclusion

After removing the three smallest gene editing companies from our catalog we’re left with eight names to watch going forward. It’s now been 12 years since we first wrote about gene editing and just one therapy has been commercialized. Progress is slow and the rewards seem uncertain for the first commercial drug to debut. Maybe after a few more success stories we’ll see just how much potential gene editing holds for investors who continue to wait patiently for more therapies to be commercialized, especially simple applications such as in vivo treatments which ultimately seem like the way forward.