Three factors will shape development of gene- and other edited therapies over the coming year and beyond.
First, commercially speaking, edited therapies still see lower than expected uptake among patients, primarily the result of payers balking at reimbursement costs for the treatments, which carry sky-high list prices—though therapy developers say their access programs have kept costs far lower for many of those in need of treatments who are unable to pay. Companies without marketed therapies have struggled to contain costs.
Those realities help explain why several public and private companies ranked by GEN reduced their headcounts last year—and why the sixth ranked privately held edited therapy developer, Tome Biosciences, went out of business last November, just 11 months after emerging from stealth mode with an eye-popping $213 million in venture capital.
The second influential factor is the long-running legal wrangle over who invented CRISPR gene-editing technology in eukaryotic cells. That battle was rekindled by a May 12 decision from the U.S. Court of Appeals for the Federal Circuit in Washington, D.C., which ordered the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (PTAB) to reconsider its 2022 interference decision, which sided with The Broad Institute of MIT and Harvard in a second challenge to its CRISPR-Cas9 patents from the University of California (UC), the University of Vienna, and CRISPR pioneer and Nobel co-laureate Emmanuelle Charpentier, PhD.
PTAB’s judgment and decision in the second interference determined that the Broad Institute, MIT, and President and Fellows of Harvard College had priority over the Regents of the UC, University of Vienna, and Charpentier, who is director and scientific member at the Max Planck Institute of Infection Biology, Berlin—known collectively as CVC—in the invention of a single RNA CRISPR-Cas9 system that functions in eukaryotic cells.
“We vacate the Board’s determination as to conception and remand for further proceedings,” the PTAB decided. “On remand, we instruct the Board to reconsider the issue of conception in a manner consistent with this opinion.”
In response, the Broad Institute issued a statement saying in part: “Broad is confident the PTAB will reach the same conclusion and will again confirm Broad’s patents, because the underlying facts have not changed.”
However, from a clinical standpoint, therapy developers are showing a growing number of successes. The world’s first CRISPR-edited therapy, Casgevy® (exagamglogene autotemcel or “exa-cel”), is slowly gaining momentum based on number of patients being treated and number of treatment centers up and running.
Casgevy’s initial developer, CRISPR Therapeutics, is among companies appearing on this A-List, which includes 10 publicly-traded developers of therapies that involve editing of genes, genomes, RNA, and other genetic materials, The companies have been ranked on a composite scale based on:
• Portfolio: Number of approved treatments and clinical programs. (Portfolio assessments included consideration of how far the clinical programs had advanced).
• Cash position: Cash, cash equivalents, and marketable securities, as disclosed by the companies in regulatory filings and press releases.
• Market capitalization: Product of the share price and the number of outstanding shares.
Because most publicly owned edited therapy developers do not yet have products that have reached the market, revenue figures are not included among the criteria used for determining company rankings, though our A-List reports their most recent quarterly revenue figures (mostly first quarter or “Q1”).
Just missing the list at No. 11 was Precision BioSciences, a developer of in vivo gene editing therapies based on its ARCUS® genome editing platform. At the European Association for the Study of the Liver (EASL) Congress held May 7-10 in Amsterdam, Precision presented initial safety data from the Phase I ELIMINATE-B trial (NCT06680232) evaluating its PBGENE-HBV program for the treatment of chronic hepatitis B.
That data showed PBGENE-HBV to be well tolerated with no dose-limiting toxicities and no serious adverse events after multiple dose administrations: “These data support the continued evaluation of multiple dose administrations per dose cohort and higher dose level cohorts of PBGENE-HBV, with the goal of achieving HBV cure,” Precision researchers concluded.
1. CRISPR Therapeutics(NASDAQ: CRSP)
Casgevy® (exagamglogene autotemcel or “exa-cel”), the CRISPR-Cas9 gene edited therapy CRISPR Therapeutics co-developed with Vertex Pharmaceuticals, finished Q1 with $14.2 million, which Vertex collects. Vertex leads global development, manufacturing, and commercialization of Casgevy, indicated for sickle cell disease and transfusion-dependent β-thalassemia. More than 65 authorized treatment centers were activated globally for Casgevy as of May 1, with 90+ patients having cells collected. In May, the company reported positive Phase I data for CTX310™ showing peak reduction of up to 82% in triglycerides and up to 81% in low-density lipoprotein. With $865,000 in grant revenue in Q1, CRISPR Therapeutics led across clinical activity (two additional Casgevy programs and seven programs across five non-Casgevy candidates), cash position ($1.855 billion), and market cap ($3.075 billion).
2. Intellia Therapeutics (NASDAQ: NTLA)
Intellia Therapeutics’ MAGNITUDE trial (NCT06128629) assessing nexiguran ziclumeran (nex-z, formerly NTLA-2001), a Regeneron Pharmaceuticals-partnered candidate for ATTR amyloidosis with cardiomyopathy, is expected to exceed 550 patients by year-end, the company said on May 8. MAGNITUDE-2 (NCT06672237), which assesses nex-z in hereditary ATTR amyloidosis with polyneuropathy, has dosed its first patient, with enrollment set to be completed in 2026. NTLA-2002, a wholly-owned hereditary angioedema candidate, is being evaluated in the HAELO trial (NCT06634420), for which Intellia dosed the first patient in January and expects enrollment completion in Q3. With $16.627 million in Q1 collaboration revenue ($57.9 million in 2024), Intellia places second in clinical activity (2 candidates in 3 Phase III trials) and cash position ($707.1 million in Q1) but fourth in market cap with $816.234 million.
3. Beam Therapeutics(NASDAQ: BEAM)
Beam Therapeutics made history in March by announcing the first-ever clinical genetic correction of a disease-causing mutation for alpha-1 antitrypsin deficiency (AATD) among positive initial safety and efficacy data from a global Phase I/II trial (NCT06389877). BEAM-302 for AATD has since won clearance for a U.S. Investigational New Drug (IND) application. Beam recently advanced its second of three clinical programs by dosing the first patient in its U.S.-based Phase I/II trial (NCT06735755) studying BEAM-301 as a potential treatment for patients with glycogen storage disease type Ia. With $7.47 million in Q1 license and collaboration revenue, Beam ranked second in market cap ($1.734 billion) and cash position ($1.22 billion, a 43% leap from Q4 2024), and fourth in clinical activity (3 candidates, all in Phase I/II).
4. Wave Life Sciences (NASDAQ: WVE)
Wave Life Sciences expects to announce data in Q3 from the 200 mg multidose and single dose cohorts of the Phase Ib/IIa RestorAATion-2 trial (NCT06405633) assessing GSK-licensed WVE-006 in AATD with the PiZZ genotype. The 400 mg single dose cohort is set to report data this fall. In 2026, Wave plans to file a New Drug Application for accelerated approval with monthly dosing of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy amenable to exon 53 skipping, following positive data in the Phase II FORWARD-53 trial (NCT04906460). Singapore-registered Wave (U.S. headquarters in Cambridge, MA) garnered $9.175 million in Q1. Wave ranked third in clinical activity (five trials for four candidates) and market cap ($978.751 million), and fifth in cash position with $243.075 million.
5. Verve Therapeutics(NASDAQ: VERV)
Verve Therapeutics announced positive initial data April 14 from the Phase Ib Heart-2 trial (NCT06164730) assessing VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease. Among 14 participants across three dose levels, a single VERVE-102 infusion yielded dose-dependent decreases in blood PCSK9 protein levels and low-density lipoprotein cholesterol (LDL-C), with a mean reduction in blood LDL-C of 53% and a maximum of 69% seen among four participants in the 0.6 mg/kg dose cohort. Verve expects to dose the first patient in its Phase II trial of VERVE-102 in 2H 2025. Reporting Q1 collaboration revenue of $32.976 million (), Verve placed fourth in cash position ($497.077 million), fifth in market cap ($384.207 million), and sixth in pipeline activity with VERVE-102 and a second LDL-C fighting clinical program, VERVE-201 (program update expected in 2H).
6. Editas Medicine (NASDAQ: EDIT)
Editas Medicine is doubling down on CRISPR-based in vivo gene editing therapies, based on positive preclinical data it shared at the American Society of Gene and Cell Therapy (ASGCT)’s annual meeting. Editas presented in vivo data showing that targeted lipid nanoparticles successfully delivered HBG1/2 promoter editing cargo to hematopoietic stem and progenitor cells (HSPCs). The company also shared in vivo preclinical proof of concept for functional upregulation of an undisclosed target liver protein to reduce a disease biomarker by >80%. With $4.658 million in Q1 collaboration and R&D revenue, Editas has no clinical programs at present, leaving the company ranking ninth. But Editas places higher in cash position, where it is sixth with $220.964 million; and ranks seventh in market cap with $115.524 million.
7. Caribou Biosciences (NASDAQ: CRBU)
Caribou Biosciences expects to report early clinical data during 2H 2025 on its co-lead candidates CB-010 for large B-cell lymphoma and CB-011 for multiple myeloma. The company narrowed its pipeline to those programs in a “prioritization” or restructuring that included chopping its workforce approximately 32% (47 jobs) and ending a Phase I trial (NCT06752876) of CB-010 for lupus, a Phase I trial of CB-012 in relapsed or refractory acute myeloid leukemia, and all preclinical research. Caribou sliced 21 jobs last year. Generating $2.353 million in Q1 revenue, all from licensing and collaborations, Caribou placed seventh in cash position ($212.452 million) and clinical pipeline (two Phase I candidates), and ninth in market cap ($76.161 million).
8. Metagenomi (NASDAQ: MGX)
Metagenomi expects to submit an IND and clinical trial application in 2026 to advance its first clinical program, wholly owned MGX-001 in hemophilia A, into first-in-human studies. MGX-001 includes a bioengineered Factor VIII (FVIII) construct with higher FVIII activity levels vs. wild type. In May, Metagenomi released 19-month FVIII durability data from a preclinical nonhuman primate durability study showing FVIII levels of 80%, 10% and 32% correlating with gene integration frequency in each of three animals studied (one died prematurely, unrelated to treatment). With $4.127 million in Q1 revenue, all from collaborations, the company finished eighth in cash position ($225.970 million) and 10th in market cap ($56.074 million). But with no clinical-phase candidates, Metagenomi tied for ninth in pipeline activity.
9. Prime Medicine (NASDAQ: PRME)
Prime Medicine plans to report data later this year from its Phase I/II trial (NCT06559176) evaluating PM359 in p47phox (also called neutrophil cytosolic factor 1 or NCF1) chronic granulomatous disease. PM359 is Prime’s sole clinical candidate, for now: The company envisions launching clinical trials for its Wilson’s Disease and AATD programs in 2026. The AATD program, unveiled in March, reflects Prime’s commitment to building a liver franchise of Prime Editors designed to cure major genetic diseases, President and CEO Keith Gottesdiener, MD, said on May 8. Reporting $1.454 million in Q1 collaboration revenue, Prime finished the quarter sixth in market cap ($158.866 million) but eighth in clinical pipeline (PRME-359 alone) and ninth in cash position ($144.256 million).
10. Sangamo Therapeutics (NASDAQ: SGMO)
Sangamo Therapeutics said on May 6 that all patients dosed with its Fabry disease gene therapy candidate isaralgagene civaparvovec (ST-920) in the Phase I/II STAAR trial (NCT04046224) passed the one-year milestone under the FDA’s Accelerated Approval pathway. In April, Sangamo licensed its neurotropic adeno-associated virus (AAV) capsid STAC-BBB to Eli Lilly under an up-o-$1.4 billion agreement to develop up to five intravenous genomic medicines for central nervous system diseases. Sangamo says STAC-BBB has shown potent blood-brain barrier penetration and neuronal transduction in nonhuman primates. With $6.437 million in Q1 revenue, Sangamo is fifth in clinical pipeline (ST-920 and ST-503 for idiopathic small fiber neuropathy, also in Phase I/II) but ranks lower in market cap (eighth with $100.387 million) and cash position (10th with $25.18 million).
Read GEN‘s Top Five Privately Held Edited Therapy Companies.