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.<\/p>\n
Of particular interest to analysts is Intellia Therapeutics (NASDAQ: NTLA)<\/strong>. Intellia stock surged 19%<\/strong> from October 6\u201310 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<\/a>. 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%<\/strong> this past week from $23.76 on Monday to $24.75 on Friday.<\/p>\n Over the past month, through Wednesday, Intellia shares nearly doubled,<\/strong> soaring 109%<\/strong> compared with just a 12% increase<\/strong> for the SPDR S&P Biotech Exchange-Traded Fund (NYSE Arca: XBI)<\/strong>,\u00a0the second-largest biotech ETF and the largest ETF whose holdings include the company\u2019s stock, Mani Foroohar, MD, senior managing director, genetic medicines with Leerink Partners, observed Thursday in a research note.<\/p>\n That one-month jump shrank to a 99% gain<\/strong> by Friday. But over six months, Intellia shares more than tripled, zooming 242%<\/strong> from $7.23 on April 17.<\/p>\n \u201cNTLA\u2019s recent outperformance,\u201d Foroohar commented, \u201chas largely been driven by broader gene editing strength (as investors\u2019 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.\u201d<\/p>\n 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\u2013December 2024 reported by Evaluate Pharma. Intellia\u2019s forecast, in turn, was based on a 2008 projection<\/a> of HAE having a worldwide prevalence of 150,000 patients.<\/p>\n At the recent European Society of Gene and Cell Therapy (ESGCT) 32nd\u00a0Annual Congress\u00a0in Seville, Spain, Birgit Schultes, PhD, Intellia\u2019s executive vice president and CSO,\u00a0presented positive data from a Phase I\/II trial (NCT05120830<\/a>) 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.<\/p>\n Lonvo-z for HAE is now under study in the Phase III HAELO trial (NCT06634420<\/a>), 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.<\/p>\n Smaller gains <\/strong><\/p>\n Intellia\u2019s 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)<\/strong>, whose $25.98 close Friday is up 16%<\/strong> from $22.45 on September 17 and 53% above<\/strong> the company\u2019s $17 close on April 17.<\/p>\n The smaller increases reflect investors holding off while the company continues two key clinical studies. By year\u2019s end, Beam is expected to share updated data from the Phase I\/II BEACON trial (NCT05456880<\/a>) 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<\/a>) of BEAM-302 in alpha-1 antitrypsin deficiency (AATD).<\/p>\n Seventeen patients had been dosed across four cohorts as of Beam\u2019s 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\u2019s initial safety and efficacy data established clinical proof-of-concept for in vivo base editing<\/a> as well as for treating AATD: \u201cWe 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,\u201d Beam CEO John Evans stated.<\/p>\n Leerink\u2019s Foroohar said the Q3 results should be routine pending future updates on the trials, though he\u2019s awaiting clinical data expected by year\u2019s end on another editing therapy being developed to treat AATD\u2014the RNA editing oligonucleotide KRRO-110<\/a> being developed by Korro Bio (NASDAQ: KRRO)<\/strong>, under study in a Phase I\/IIa trial (BTX-302-001;\u00a0NCT06389877<\/a>).<\/p>\n The next-lowest one-month gainer was CRISPR Therapeutics (NASDAQ: CRSP)<\/strong>, which rose 17% <\/strong>over the past month (from $59.07 on September 17) and jumped 83% <\/strong>over the past six months (from $37.77 on April 17).<\/p>\n New platform, positive publicity<\/strong><\/p>\n Those include a 5% uptick<\/strong> 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\u2122\u00a0editing platform and its first SyNTase-based candidate CTX460\u2122, an in vivo\u00a0gene correction candidate designed to treat AATD.<\/p>\n 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.<\/p>\n \u201cWe expect the platform to be a source of add\u2019l programs for CRSP\/partners,\u201d Maury Raycroft, PhD, equity research analyst with Jefferies, wrote in an October 10 research note.<\/p>\n At the high end of six-month gainers is Prime Medicine (NASDAQ: PRME)<\/strong>, whose shares more than quadrupled<\/strong>, catapulting nearly 340%<\/strong> from $1.24 on April 17 to $5.45 at Friday\u2019s close. Over the past month, however, Prime saw a much more modest 17% increase<\/strong> (from $4.65 on September 17), the difference likely reflecting the absence of news announcements since the company released second-quarter results in August.<\/p>\n However, Prime made some news outside of press releases\u2014namely, some positive publicity from an article in\u00a0Wired<\/a> about Prime\u2019s namesake editing technology, summing up remarks made at the magazine\u2019s 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.<\/p>\n Prime shares jumped 28%<\/strong> 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<\/a>, which he calls disease-agnostic therapeutic genome editing,\u00a0on \u201cBehind the Breakthroughs,\u201d a podcast of GEN sister publication Inside Precision Medicine.<\/p>\n \u201cSentiment is improving\u201d<\/strong><\/p>\n \u201cFive years ago, valuations were unsustainable. Then everything crashed\u2014companies were trading below cash,\u201d Prime Medicine CEO Allan Reine, MD, told Inside Precision Medicine in an interview at the recent Meeting on the Mesa conference<\/a> in Phoenix. \u201cNow, sentiment is improving for the right reasons. We\u2019re seeing real clinical data, real approvals, and pharma partnerships that validate the space. The dream is becoming reality.\u201d<\/p>\n Reine was promoted from CFO in May, succeeding Keith Gottesdiener, MD, at the company\u2019s helm. Under Reine, Prime Medicine eliminated 25% of its workforce\u2014about 50 jobs\u2014in a restructuring<\/a> that included the company pivoting its prime editing-based pipeline focus to liver disease and programs funded by external collaboration partners.<\/p>\n The job cutting overshadowed some good news from Prime\u2019s 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\u00a0(also called neutrophil cytosol factor 1) variant of autosomal recessive CGD.<\/p>\n 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\u2019s Disease and AATD.<\/p>\n Also surging among gene editing therapy developers is Editas Medicine (NASDAQ: EDIT)<\/strong>, which closed Friday at $3.77. Editas has seen its stock climb 39%<\/strong> over the past month from $2.72 on September 17, and nearly triple, rocketing 195% <\/strong>over the past six months from $1.28 on April 17.<\/p>\n The past month\u2019s surge reflected Editas\u2019 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 \u226590% LDL-C reduction in nonhuman primates within 48 hours of a single dose of EDIT-401, as well as a \u226590% LDL-C reduction in mice with high baseline LDL-C and reduced function of the LDL receptor (LDLR).<\/p>\n Initial in vivo human data set for 2026<\/strong><\/p>\n 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.<\/p>\n \u201cTranslation of EDIT-401\u2019s 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,\u201d Jack K. Allen, a senior research analyst with Baird, wrote last month in a summary of presentations from the firm\u2019s 2025 Healthcare Conference. \u201cManagement is also working to advance the preclinical efforts surrounding in vivo gene editing in extrahepatic tissues in tandem.\u201d<\/p>\n Addressing analysts at the H.C. Wainwright Genetic Medicines Virtual Conference on October 14, Editas president and CEO Gilmore O\u2019Neill, 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.<\/p>\n \u201cExceeding that, we think, actually is meaningfully differentiated,\u201d O\u2019Neill said, according to a transcript published by Investing.com. \u201cNot only are we mechanistically differentiated \u2026 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.\u201d<\/p>\n He added: \u201cWe want to be transformative. We want to differentiate from other medicines.\u201d<\/p>\n Leaders and laggards<\/strong><\/p>\n\n