4 Gene-Editing Biotech Stocks Worth Watching for Long-Term Gains

Gene editing is emerging as a potentially transformative approach to medicine because it seeks to address disease at its genetic source rather than primarily managing downstream symptoms. Unlike conventional therapies that may require chronic administration to suppress disease activity, genome-editing treatments are designed to make targeted changes to DNA that can potentially produce long-lasting therapeutic benefits after a single treatment. CRISPR has accelerated this field by making targeted genome modification more efficient and scalable, helping move gene editing from a research tool toward a clinical treatment modality.

The investment case rests on the potential for gene editing to expand treatment options for serious genetic diseases that have historically lacked curative therapies. At the same time, gene editing is still an emerging technology and faces challenges related to treatment delivery, long-term safety, manufacturing and regulatory approval. Concerns about unintended changes to DNA also remain an important consideration as these therapies move through clinical development, underscoring both the significant potential of gene editing and the risks involved.

Against this backdrop, CRISPR Therapeutics CRSP, Intellia Therapeutics NTLA, Beam Therapeutics BEAM and Editas Medicine EDIT stand out as comparable gene-editing players with differentiated platforms and meaningful clinical catalysts. The range of technologies and clinical stages across these companies offers investors exposure from proven commercial validation to higher-risk, potentially high-reward pipeline opportunities. All four companies currently carry a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

CRISPR Therapeutics

CRISPR Therapeutics has the strongest commercial validation among the four. Its core platform is based on CRISPR/Cas9 technology, with programs spanning ex vivo (out of the living organism) and in vivo (within the living organism) gene editing. Its flagship product, Casgevy (exagamglogene autotemcel), became the world's first approved CRISPR-based ex vivo gene-editing therapy in 2023 for two indications — sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). In July 2026, CRSP secured FDA approval to expand its U.S. use to eligible patients as young as two years old with either SCD or TDT.

Year to date, CRSP shares have gained 13.5% compared with the industry’s growth of 11.3%.

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CRISPR Therapeutics is also developing next-generation technologies, including its proprietary SyNTase platform and lipid nanoparticle (LNP) delivery systems, to expand its gene-editing pipeline beyond hematology into cardiovascular and rare diseases. The company’s larger long-term opportunity lies in its in vivo pipeline.

CTX310, targeting angiopoietin-related protein 3, is currently being evaluated in a phase Ib study for several cardiovascular disease indications. CTX340, directed toward angiotensinogen, recently entered phase I development for refractory hypertension, while CTX460, directed toward SERPINA1 using CRSP’s proprietary SyNTase editing platform, is also undergoing early-stage evaluation for alpha-1 antitrypsin deficiency. CTX321, which targets elevated lipoprotein(a), is yet to enter clinical development.

Intellia Therapeutics

Intellia Therapeutics is differentiated by its focus on systemically delivered, in vivo CRISPR editing. Rather than removing cells, editing them externally and reinfusing them, NTLA uses LNPs to deliver its editing payload directly to target tissues. Per management, it is the first company to advance in vivo genome-editing candidates into phase III clinical development.

Year to date, NTLA shares have surged 42.8% compared with the industry’s 11.3% growth.

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One of NTLA’s lead candidates, lonvoguran ziclumeran (lonvo-z), is an investigational, in vivo CRISPR-based therapy being developed for hereditary angioedema (HAE). In the phase III HAELO study, a one-time infusion of lonvo-z reduced HAE attacks by 87% compared with placebo during the six-month efficacy evaluation period. Intellia initiated a rolling biologics license application (BLA) in April 2026, expecting the FDA to accept the submission by year-end. Subject to approval, Intellia Therapeutics anticipates a potential U.S. launch of lonvo-z for HAE in the first half of 2027.

The second key candidate is nexiguran ziclumeran (nex-z), an in vivo CRISPR-based therapeutic candidate, currently being evaluated in the phase III MAGNITUDE and MAGNITUDE-2 studies for transthyretin amyloidosis with cardiomyopathy and hereditary transthyretin amyloidosis with polyneuropathy, respectively. Both studies resumed patient enrollment after FDA clinical holds were lifted in 2026, although safety monitoring remains a key investment consideration.

Beam Therapeutics

Beam Therapeutics offers a different technological proposition through base editing, which is designed to make precise single-base changes without creating the double-strand DNA breaks associated with conventional CRISPR/Cas9 editing. The company is combining this technology with LNP delivery and ex vivo editing to pursue potentially durable genetic medicines across hematology and rare diseases.

Year to date, BEAM shares have gained 7.4% compared with the industry’s 11.3% growth.

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Beam Therapeutics’ lead hematology candidate, risto-cel, is an investigational ex vivo base-edited therapy for SCD, currently undergoing development in the phase I/II BEACON study. Dosing has been completed in both the adult and adolescent patient cohorts of the BEACON study, and the company plans to submit a BLA for risto-cel to treat SCD by the end of 2026. BEAM is also advancing BEAM-302 in a phase I/II study to treat alpha-1 antitrypsin deficiency (AATD). Based on FDA feedback, it plans to pursue an accelerated approval pathway for the candidate using AAT biomarkers evaluated over 12 months, with a 60-mg dose selected. The first patient was dosed in the pivotal expansion cohort of the phase I/II AATD study in July 2026, with plans to enroll about 50 additional patients to support a future BLA submission.

Beam Therapeutics is also expanding its platform into additional genetic diseases. BEAM-301 is undergoing phase I/II development for glycogen storage disease type Ia in patients with the R83C mutation, while BEAM-304 is being developed for phenylketonuria with a phase I/II study initiation anticipated soon. This pipeline breadth strengthens the investment case by allowing validation of the same base-editing platform across multiple disease areas rather than relying on a single asset.

Editas Medicine

Editas Medicine has strategically shifted toward in vivo CRISPR-based gene editing, aiming to develop therapies administered directly to patients that can functionally upregulate normal gene expression. Its platform work includes targeted LNP delivery to tissues such as the liver and hematopoietic stem cells, potentially expanding the range of diseases addressable with in vivo editing.

Year to date, shares of Editas have soared 34.1% compared with the industry’s 2.5% growth.

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The key pipeline asset is EDIT-401, an investigational potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (LDL-C) through upregulation of the LDL receptor gene. The candidate is intended for heterozygous familial hypercholesterolemia and is progressing toward a phase I/II study initiation in Australia and New Zealand. In preclinical non-human-primate studies, a single dose produced approximately 90% or greater mean reductions in LDL-C, Lp(a) and ApoB, supporting its potential as a differentiated approach to cardiovascular risk reduction.

EDIT is the most speculative of the four stocks. CRSP has an approved gene-therapy product, while NTLA’s lead candidates are in late-stage development. BEAM’s several early-stage candidates provide greater pipeline diversification, unlike EDIT, which has just one gene-editing candidate that has yet to enter clinical-stage development.Importantly, Editas Medicine discontinued development of its former ex vivo reni-cel program for SCD and TDT in 2024 and is now concentrating resources on its in vivo pipeline. That creates greater execution risk but also makes EDIT a relatively pure bet on whether in vivo CRISPR editing can translate from preclinical validation into human proof of concept and, ultimately, commercial therapies.

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