Published on: Jan 21, 2026

Author: Amy Liu

Gene editing technology, which directly modifies DNA, offers revolutionary potential for treating and even curing diseases caused by genetic variations. It is predicted that the global gene editing market could reach $30.8 billion by 2032. In this promising sector, several leading biotechnology companies are advancing therapies toward the market, attracting significant investor attention.

Intellia Therapeutics: Dedicated to In Vivo Editing Therapies

Co-founded by Jennifer Doudna in 2014, Intellia Therapeutics (NTLA) focuses on developing potentially curative gene editing therapies. In 2021, in collaboration with partner Regeneron Pharmaceuticals (REGN), the company released the first clinical data supporting the use of CRISPR-Cas9 gene editing in humans, marking a significant milestone. Currently, the two are collaborating on late-stage studies for transthyretin amyloidosis (ATTR), evaluating the drug nexiguran ziclumeran (formerly known as NTLA-2001). Additionally, Intellia’s wholly owned late-stage candidate drug NTLA-2002 is planned to be submitted to U.S. regulators in the second half of 2026 for the treatment of hereditary angioedema (HAE). If the subsequent progress of its hemophilia B therapy licensed to Regeneron proceeds smoothly, Intellia will also be eligible for milestone payments and royalties.

CRISPR Therapeutics: From the First Approval to Pipeline Expansion

CRISPR Therapeutics (CRSP) was co-founded by Emmanuelle Charpentier in 2013. In collaboration with Vertex Pharmaceuticals (VRTX), its gene editing therapy Casgevy gained regulatory approval in December 2023 for treating sickle cell disease, followed by approval for transfusion-dependent beta-thalassemia, achieving a commercial breakthrough in the field. As Casgevy’s commercialization advances, the company’s research pipeline includes several innovative projects, such as two allogeneic CAR-T therapies, CTX112 and CTX131, currently in clinical trials, two in vivo gene editing therapies in early clinical studies, and an experimental therapy for type 1 diabetes.

Beam Therapeutics: Exploring Precision Base Editing

Beam Therapeutics (BEAM) was co-founded by David Liu and Feng Zhang in 2017, focusing on base editing technology. In March 2025, the company announced positive preliminary results from a Phase I/II clinical study of BEAM-302 for alpha-1 antitrypsin deficiency (AATD). Meanwhile, its candidate drug risto-cel (formerly known as BEAM-101) for sickle cell disease is also in Phase I/II clinical studies, with plans to submit a marketing application as early as the end of 2026. Additionally, the company expects to release more clinical trial data for BEAM-301, targeting glycogen storage disease type 1a, and BEAM-302 for AATD in 2026.

Investment Risks and Challenges Should Not Be Overlooked

Although the gene editing sector offers considerable long-term return potential, investing in related stocks also comes with significant risks. These include high stock price volatility, potential dilution of existing stock value due to capital needs, the possibility of clinical trial failures, risks of failing to gain regulatory approval, and potential reimbursement hurdles after therapies are approved.