As developers of gene editing therapies and other genetic medicines get set to report third quarter earnings in the coming days, analysts and especially investors will keep their eyes on whether the companies maintain the momentum that has seen their stocks double or triple in price over the past year.

Of particular interest to analysts is Intellia Therapeutics (NASDAQ: NTLA). Intellia stock surged 19% from October 6–10 on news of positive clinical data in hereditary angioedema (HAE) and transthyretin amyloidosis (ATTR) that, in turn, lifted the shares of other gene editing companies. After a two-day dip mirroring an overall market decline over fears of higher tariffs on Chinese goods, Intellia shares resumed their climb, rising 4% this past week from $23.76 on Monday to $24.75 on Friday.

Over the past month, through Wednesday, Intellia shares nearly doubled, soaring 109% compared with just a 12% increase for the SPDR S&P Biotech Exchange-Traded Fund (NYSE Arca: XBI), the second-largest biotech ETF and the largest ETF whose holdings include the company’s stock, Mani Foroohar, MD, senior managing director, genetic medicines with Leerink Partners, observed Thursday in a research note.

That one-month jump shrank to a 99% gain by Friday. But over six months, Intellia shares more than tripled, zooming 242% from $7.23 on April 17.

“NTLA’s recent outperformance,” Foroohar commented, “has largely been driven by broader gene editing strength (as investors’ confidence builds that gene editing can co-exist alongside oligos) and positioning into pivotal HAE data in 2Q26, as NTLA looks to shift the narrative from clinical/TTR enrollment to a commercial story with a sizeable HAE commercial opportunity relative to its current valuation.”

That commercial opportunity could translate to global sales of $5 billion by 2028, Intellia told investors in August, citing a consensus forecast by analysts from October–December 2024 reported by Evaluate Pharma. Intellia’s forecast, in turn, was based on a 2008 projection of HAE having a worldwide prevalence of 150,000 patients.

At the recent European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress in Seville, Spain, Birgit Schultes, PhD, Intellia’s executive vice president and CSO, presented positive data from a Phase I/II trial (NCT05120830) showing that lonvoguran ziclumeran (lonvo-z), the gene editing candidate previously called NTLA-2002, successfully treated HAE by reducing kallikrein levels to less than 60% up to and beyond two years after treatment at all doses. The 50 mg dose offered the best results among the 27 patients studied in Phase II, with 70% of those patients showing a complete response.

Lonvo-z for HAE is now under study in the Phase III HAELO trial (NCT06634420), which completed patient enrollment last month. Intellia said it expects to release topline data in the first half of 2026, then submit a biologics license application (BLA) in the second half of 2026 and launch lonvo-z in the first half of 2027.

Smaller gains

Intellia’s one-month gain is at the high end among five leading publicly traded gene editing companies spot-checked by StockWatch in recent days. At the low end of both one- and six-month gains was Beam Therapeutics (NASDAQ: BEAM), whose $25.98 close Friday is up 16% from $22.45 on September 17 and 53% above the company’s $17 close on April 17.

The smaller increases reflect investors holding off while the company continues two key clinical studies. By year’s end, Beam is expected to share updated data from the Phase I/II BEACON trial (NCT05456880) assessing BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs). And in early 2026, Beam anticipates releasing data from both parts of a Phase I/II trial (NCT06389877) of BEAM-302 in alpha-1 antitrypsin deficiency (AATD).

Seventeen patients had been dosed across four cohorts as of Beam’s second-quarter earnings results in August. At the time, Beam said BEAM-302 continued to show positive data as it did in March, when the trial’s initial safety and efficacy data established clinical proof-of-concept for in vivo base editing as well as for treating AATD: “We have mounting evidence to suggest that BEAM-302 is fundamentally altering the disease to restore the key physiologic functions of alpha-1 antitrypsin with a single course of treatment,” Beam CEO John Evans stated.

Leerink’s Foroohar said the Q3 results should be routine pending future updates on the trials, though he’s awaiting clinical data expected by year’s end on another editing therapy being developed to treat AATD—the RNA editing oligonucleotide KRRO-110 being developed by Korro Bio (NASDAQ: KRRO), under study in a Phase I/IIa trial (BTX-302-001; NCT06389877).

The next-lowest one-month gainer was CRISPR Therapeutics (NASDAQ: CRSP), which rose 17% over the past month (from $59.07 on September 17) and jumped 83% over the past six months (from $37.77 on April 17).

New platform, positive publicity

Those include a 5% uptick from $68.29 to $71.82 that the stock enjoyed over three days ending Wednesday, after the company reported positive preclinical data in mouse and rat models for its new SyNTase™ editing platform and its first SyNTase-based candidate CTX460™, an in vivo gene correction candidate designed to treat AATD.

CTX460 targets the E342K mutation in SERPINA1, the gene that provides instructions for making alpha-1-antitrypsin, encapsulated in a de-risked lipid nanoparticle (LNP). According to CRISPR Therapeutics, CTX460 achieved >90% mRNA correction, a five-fold increase in total AAT levels, and >99% serum M-AAT:Z-AAT ratio in disease models of AATD. CTX460 maintained editing durability for up to seven weeks in rat models, and up to nine weeks in mouse models. The company said its results supported advancement of CTX460 and SyNTase into clinical trials in mid-2026.

“We expect the platform to be a source of add’l programs for CRSP/partners,” Maury Raycroft, PhD, equity research analyst with Jefferies, wrote in an October 10 research note.

At the high end of six-month gainers is Prime Medicine (NASDAQ: PRME), whose shares more than quadrupled, catapulting nearly 340% from $1.24 on April 17 to $5.45 at Friday’s close. Over the past month, however, Prime saw a much more modest 17% increase (from $4.65 on September 17), the difference likely reflecting the absence of news announcements since the company released second-quarter results in August.

However, Prime made some news outside of press releases—namely, some positive publicity from an article in Wired about Prime’s namesake editing technology, summing up remarks made at the magazine’s Wired Health summit by genome editing pioneer David R. Liu, PhD, whose Broad Institute lab developed both prime editing and base editing technologies over more than a decade.

Prime shares jumped 28% in the four days after the article came out. In it, Liu shared that his lab plans to report on an editing approach that could enable a single composition of matter to treat multiple unrelated genetic diseases. Liu shared the thinking behind that approach, which he calls disease-agnostic therapeutic genome editing, on “Behind the Breakthroughs,” a podcast of GEN sister publication Inside Precision Medicine.

“Sentiment is improving”

“Five years ago, valuations were unsustainable. Then everything crashed—companies were trading below cash,” Prime Medicine CEO Allan Reine, MD, told Inside Precision Medicine in an interview at the recent Meeting on the Mesa conference in Phoenix. “Now, sentiment is improving for the right reasons. We’re seeing real clinical data, real approvals, and pharma partnerships that validate the space. The dream is becoming reality.”

Reine was promoted from CFO in May, succeeding Keith Gottesdiener, MD, at the company’s helm. Under Reine, Prime Medicine eliminated 25% of its workforce—about 50 jobs—in a restructuring that included the company pivoting its prime editing-based pipeline focus to liver disease and programs funded by external collaboration partners.

The job cutting overshadowed some good news from Prime’s pipeline; its first clinical candidate, PM359, generated positive initial data, namely the first-ever clinical data supporting the safety and efficacy of prime editing in humans. PM359 is a treatment for the p47phox (also called neutrophil cytosol factor 1) variant of autosomal recessive CGD.

Prime is seeking a partner for PM359, instead focusing resources on internally developed in vivo programs designed to treat two liver diseases with large patient populations representing potentially lucrative commercial opportunities, Wilson’s Disease and AATD.

Also surging among gene editing therapy developers is Editas Medicine (NASDAQ: EDIT), which closed Friday at $3.77. Editas has seen its stock climb 39% over the past month from $2.72 on September 17, and nearly triple, rocketing 195% over the past six months from $1.28 on April 17.

The past month’s surge reflected Editas’ October 9 presentation of positive in vivo preclinical proof-of-concept data for EDIT-401, a one-time therapy designed to significantly reduce LDL-cholesterol (LDL-C). In data presented at ESGCT, researchers reported achieving a ≥90% LDL-C reduction in nonhuman primates within 48 hours of a single dose of EDIT-401, as well as a ≥90% LDL-C reduction in mice with high baseline LDL-C and reduced function of the LDL receptor (LDLR).

Initial in vivo human data set for 2026

Editas nominated EDIT-401 last month as its lead in vivo development candidate, saying it planned to submit an investigational new drug (IND) or clinical trial application (CTA) for the therapy by mid-2026, and aimed to achieve in vivo human proof-of-concept data by the end of next year.

“Translation of EDIT-401’s preclinical results into clinical models should be fairly derisked by both the utilization of the same guide molecules in NHPs and prior data showing strong translatability of in vivo CRISPR programs from NHPs to humans,” Jack K. Allen, a senior research analyst with Baird, wrote last month in a summary of presentations from the firm’s 2025 Healthcare Conference. “Management is also working to advance the preclinical efforts surrounding in vivo gene editing in extrahepatic tissues in tandem.”

Addressing analysts at the H.C. Wainwright Genetic Medicines Virtual Conference on October 14, Editas president and CEO Gilmore O’Neill, noted that EDIT-401 is aiming for a far higher LDL-C reduction than the 40% range achieved by statins, and 60% range delivered by PCSK9 inhibitors.

“Exceeding that, we think, actually is meaningfully differentiated,” O’Neill said, according to a transcript published by Investing.com. “Not only are we mechanistically differentiated … not only can we differentiate it by driving increases of LDLR by direct editing of regulatory elements of its genetics, but we actually have an efficacy differentiation here.”

He added: “We want to be transformative. We want to differentiate from other medicines.”

Leaders and laggards

Absci (ABSI) shares climbed 28% from $3.90 to $5 Wednesday after the developer of engineered biologic drugs based on its own generative AI platform said it now expects to initiate a Phase I/IIa trial in early December, ahead of prior plans to start in early 2026, for ABS-201, an anti-prolactin receptor (PRLR) antibody designed to treat androgenetic alopecia, also known as male and female pattern hair loss. ABS-201 is designed to stimulate hair follicle regeneration and promote durable hair regrowth. Absci said the antibody has shown statistically significant superior hair regrowth compared to minoxidil in preclinical mouse models. Absci anticipates releasing initial proof-of-concept data in the second half of 2026.
Omeros (NASDAQ: OMER) shares more than doubled, zooming 154% from $4.10 to $10.42 on Wednesday after the company joined Novo Nordisk to announce that the Danish biopharma giant entered into an up to $2.1 billion asset purchase and license agreement for the Seattle biotech’s rare blood and kidney disorder candidate zaltenibart (formerly OMS906). Novo Nordisk has been granted exclusive global rights to develop and commercialize zaltenibart in all indications. In return, Novo Nordisk agreed to pay Omeros $340 million in upfront and near-term milestone payments, plus additional payments tied to achieving development and commercial milestones, plus tiered royalties on net sales. Zaltenibart is Phase III ready, as Omeros presented positive Phase II data for the drug in paroxysmal nocturnal hemoglobinuria (PNH) last December at the 66th Annual Meeting of the American Society of Hematology (ASH)
Praxis Precision Medicines (PRAX) shares nearly tripled, soaring 184% from $57.35 to $162.71 on Thursday after the company announced positive topline results for its two-study, Phase III Essential3 program (NCT06087276) assessing ulixacaltamide in essential tremor. Study 1 met its primary endpoint as 199 ulixacaltamide patients achieved a statistically significant and clinically meaningful 4.3 point mean improvement in the Modified Activities of Daily Living 11 (mADL11) score at Week 8, vs 1.7 points for 233 placebo patients. The effect was sustained from Week 2 throughout the 12-week dosing period. Study 2 met its pre-specified primary endpoint, as 55% of patients in the 233-patient ulixacaltamide arm maintained response vs 33% in the 234-patient placebo group. “We look forward to the opportunity to have a pre-NDA meeting with the FDA soon to discuss the potential NDA,” Praxis president and CEO Marcio Souza said. Praxis develops treatments for CNS disorders characterized by neuronal excitation-inhibition imbalance.
Rani Therapeutics Holdings (NASDAQ: RANI) shares more than tripled, leaping 248% from 47 cents to $1.64 on Friday after announcing it entered into an up-to-$1.085 billion collaboration and license agreement with Chugai Pharmaceutical to develop and commercialize Chugai’s rare disease antibody in development and the RaniPill® oral delivery technology. Chugai agreed to pay Rani $10 million upfront, up to $75 million tied to achieving technology transfer and development milestones, up to $100 million tied to sales milestones, and single-digit royalties on sales. Chugai has an option to extend its rights to up to five additional drug targets under similar deal terms. Rani also entered into a securities purchase agreement for a private placement in public equity financing (PIPE) that is expected to result in gross proceeds of approximately $60.3 million to Rani. Those anticipated proceeds, the initial upfront payment, and $18 million in expected technology transfer milestones are expected to fund Rani’s operations into 2028.