Gene Editing in 2026 and the ASX Cell and Gene Therapy Companies

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Samso News theme banner. Gene Editing in 2026. ASX Cell and Gene Therapy Companies.

Samso taxonomy strip. Samso News, Biotech and Health, Gene Editing, Part 4 of 6.

In 2026 gene editing done inside the body produced its first positive Phase 3 result, for Intellia Therapeutics' lonvo-z.

All prices in this piece are as at the close of ASX trading on 30 October 2026.

What Gene Editing, Gene Therapy and Cell Therapy Are

Gene editing changes a person's DNA at a chosen spot. The best-known tool is CRISPR, which works like molecular scissors guided to one stretch of DNA. Gene therapy adds a working copy of a gene, usually carried by a harmless virus, and leaves the faulty one in place. Cell therapy uses living cells as the medicine.

IN PLAIN ENGLISH

CAR-T. T cells, a type of immune cell, fitted with a receptor that locks onto cancer cells. In vivo and ex vivo. An edit made inside the body, or to cells taken out and given back. Off-the-shelf. Made in advance from donor cells, not from each patient's cells.

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Intellia's Lonvo-z for Hereditary Angioedema

Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, was the first CRISPR medicine approved in the United States, in December 2023, for sickle cell disease. It is made ex vivo, from the patient's edited blood stem cells.

Intellia Therapeutics edits inside the body. Its treatment, lonvo-z, is one infusion for hereditary angioedema, a rare inherited condition that causes painful and sometimes dangerous swelling attacks. It switches off a liver gene called KLKB1, which lowers kallikrein, a protein that drives the attacks [R1]. Patients already have preventive medicines, taken regularly.

Intellia reported in April 2026 that its Phase 3 trial, HAELO, had met its primary endpoint, the main result it set out to measure [R14]. Full results were published on 13 June 2026 in the New England Journal of Medicine [R1]. Of 80 patients, 52 received one 50 milligram dose and 28 a placebo. Over weeks 5 to 28, attacks fell by 87% against placebo, and 62% of treated patients had no attacks and needed no other treatment, against 11% on placebo. Intellia reported no serious adverse events in the treated group [R1] [R14].

TRIAL TERMS

Phase 1, 2 and 3. The stages of human testing, from safety in a small group to a large comparison that a regulator usually approves on.

On 8 September 2026 Intellia said the US Food and Drug Administration (FDA) had accepted its application under Priority Review, a faster timetable, and aims to decide by 10 March 2027. Intellia says lonvo-z would be the world's first approved in-body CRISPR therapy [R2].

CRISPR Therapeutics tested the same in-body approach for cholesterol, in a Phase 1 trial run partly in Australia [R15] [R3].

ASX Companies in Cell and Gene Therapy

ASX exposure comes through gene therapy, gene-edited cell therapy and cell therapy. We found no ASX-listed company whose lead medicine is a CRISPR treatment.

CSL (ASX: CSL)

CSL sells Hemgenix, a one-time gene therapy for adults with haemophilia B, a bleeding disorder caused by too little of a clotting protein called factor IX. In March 2026 CSL announced a temporary global stockout, which it said was a manufacturing issue, not a safety one [R5]. CSL says it re-established limited supply in April, and that more than 100 people have been treated commercially since approval [R4].

Mesoblast (ASX: MSB)

Mesoblast makes Ryoncil, a cell therapy from donor bone marrow cells called mesenchymal stromal cells, grown in a laboratory. It is FDA approved for children with acute graft versus host disease that steroids have not controlled. In this transplant complication, donor immune cells attack the patient. Mesoblast reported US$115 million of Ryoncil net revenue in the year to 30 June 2026 [R6], and US$28 million in the September 2026 quarter [R7]. Mesoblast says a registration trial in adults is enrolling patients [R6].

Imugene (ASX: IMU)

Imugene's azer-cel is an off-the-shelf, gene-edited CAR-T cell therapy for blood cancers. It is made with ARCUS, a gene editing tool that is not CRISPR, from Precision BioSciences. ARCUS edits the donor cells so they target cancer and do not attack the patient [R16] [R8]. On 22 May 2026 Imugene reported that 13 of 16 evaluable patients in a Phase 1b group new to CAR-T had responded [R9]. In June the FDA gave azer-cel Fast Track, which allows closer contact with the agency, in two more blood cancers [R10].

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Arovella Therapeutics (ASX: ALA)

Arovella Therapeutics is at an earlier stage. Its ALA-101 uses iNKT immune cells from healthy donors, fitted with a CAR that targets CD19, a marker on many blood cancer cells. On 27 October 2026 it dosed the first patient in its Phase 1 trial, SAPPHIRE, at The Alfred in Melbourne, and expects initial data in early 2027 [R11].

Where the Share Prices Sit

TABLE 01 Closing share prices of the four ASX companies, against the close a year earlier. Columns: Company, Code, Close 30 October 2026, Close a year earlier, Change. Company CSL; Code CSL; Close 30 October 2026 A$200.73; Close a year earlier A$179.56; Change up 12%. Company Mesoblast; Code MSB; Close 30 October 2026 A$2.26; Close a year earlier A$2.51; Change down 10%. Company Imugene; Code IMU; Close 30 October 2026 3.5 cents; Close a year earlier 37 cents; Change down 91%. Company Arovella Therapeutics; Code ALA; Close 30 October 2026 5.3 cents; Close a year earlier 9.2 cents; Change down 42%. Source: Yahoo Finance daily closes [R17], Samso calculation. Year-earlier close is the last session on or before 30 October 2025.

Over the year the trials moved forward while three of the four share prices fell. The two companies still in early trials fell the most. Our reading is that the market is waiting for trial results before it pays for them. We can probably safely assume Imugene's placements, at 18 and 9.5 cents, added to its fall [R19] [R18]. CSL is 12% higher than a year ago. Mesoblast is 10% lower while Ryoncil sales grew. Imugene and Arovella trade below 10 cents, so a small move in price is a large move in percentage terms.

What Comes Next for Gene Editing

What we still need to know is how an in-body edit holds up over many years, and how health systems will pay for one treatment that replaces years of medicine. The next date is the FDA decision on lonvo-z, due by 10 March 2027.

THE OTHER WAY TO READ THIS

In October 2025 the FDA put Phase 3 trials of a second Intellia treatment, nex-z, on hold after a patient had a serious liver reaction. Enrolment resumed in 2026 [R13] [R12]. The Hemgenix stockout shows supply can be interrupted after approval, and early responses such as Imugene's come from small groups and may not hold in larger trials.

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Samso Concluding Comments

Lonvo-z is the first positive Phase 3 result for an edit made inside the body, with an FDA decision due by March 2027. CSL and Mesoblast already sell approved therapies. Imugene and Arovella are still in trials.

SAMSO TAKE

For me, lonvo-z is the point where in-body gene editing became a medicine waiting on a regulator. I will be watching March 2027, and Arovella's first data. Will health systems pay for one-time treatments like this?

THE VOCABULARY, IN PLAIN ENGLISH

CRISPR.

A gene editing tool that cuts DNA at a chosen spot.

Gene therapy.

A treatment that adds a working copy of a gene without editing the faulty one.

Cell therapy.

A treatment that uses living cells as the medicine.

CAR-T.

Immune cells fitted with a receptor that targets cancer.

In vivo, ex vivo.

Inside the body, and outside the body.

Off-the-shelf (allogeneic).

Made in advance from donor cells rather than the patient's cells.

Phase 1, 2, 3.

The stages of human trials, from safety in a small group to a large comparison.

Placebo.

A dummy treatment used for comparison.

Primary endpoint.

The main result a trial is designed to measure.

Priority Review.

An FDA review with a shorter target time.

Fast Track.

An FDA designation that allows more contact with the agency during development.

Evaluable patient.

A patient with enough follow-up to be assessed.

Response rate.

The share of patients whose cancer shrank or disappeared.

References and Sources

[R1] Intellia Therapeutics, "Intellia Therapeutics Reports Additional Positive Phase 3 Results for Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema", 13 June 2026. Link.

[R2] Intellia Therapeutics, "Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review" (lonvo-z), 8 September 2026. Link.

[R3] American Heart Association, "First-in-human trial of CRISPR gene-editing therapy safely lowered cholesterol, triglycerides", 8 November 2025 (CTX310, Scientific Sessions 2025). Link.

[R4] CSL, "Our Continued Commitment to the Hemophilia B Community, An Update on HEMGENIX Supply and Patient Access", 24 July 2026. Link.

[R5] CSL, "Our Commitment to the Hemophilia B Community, An Update on HEMGENIX Availability", 17 March 2026. Link.

[R6] Mesoblast, ASX announcement, "Ryoncil Delivers Net Revenue of US$36M for the Fourth Quarter Ended 30 June 2026", 10 July 2026, and Financial Results and Operational Update for FY2026, 27 August 2026 (https://announcements.asx.com.au/asxpdf/20260827/pdf/0739c7szlttrdy.pdf). Link.

[R7] Mesoblast, ASX announcement on Ryoncil net revenue for the quarter ended 30 September 2026, 16 October 2026. Link.

[R8] Gupta S. and others, "Safety and Efficacy of Azer-cel, an Allogeneic CD19 CAR T, Plus Low-Dose Interleukin-2", ASH 2025 oral presentation, published on imugene.com. Link.

[R9] Imugene, ASX announcement, "Azer-cel Demonstrates Promising Response Rates in CAR T naive cohort in ASCO 2026 abstract", 22 May 2026. Link.

[R10] Imugene, ASX announcement, "Imugene's Azer-cel Granted FDA Fast Track Designation for CLL/SLL and MZL", 9 June 2026. Link.

[R11] Arovella Therapeutics, ASX announcement, "First Patient Dosed with ALA-101", 27 October 2026. Link.

[R12] Intellia Therapeutics, "Intellia Therapeutics Announces Second Quarter 2026 Financial Results and Business Updates", 6 August 2026 (nex-z enrolment reinitiated). Link.

[R13] Intellia Therapeutics, "Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE Phase 3 Clinical Trial in ATTR-CM", 2 March 2026. Link.

[R14] Intellia Therapeutics, "Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing", 27 April 2026. Link.

[R15] CRISPR Therapeutics, "CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310", 8 November 2025. Link.

[R16] Precision BioSciences, "Precision BioSciences Announces the Presentation of Initial Clinical Data Supporting the Safety and Clinical Activity of PBCAR0191", 6 November 2019. Link.

[R17] Yahoo Finance, daily closing prices for CSL.AX, MSB.AX, IMU.AX and ALA.AX, read 31 October 2026. Link.

[R18] Imugene, ASX announcement, "Imugene Completes $11.1 Million Placement to Advance azer-cel", 7 July 2026. Link.

[R19] Imugene, Quarterly Activities and Appendix 4C, quarter ended 31 March 2026 (11 March 2026 placement), 30 April 2026. Link.

Cover photo: Monoclonal Antibodies, a scientist's hands pipetting samples in a laboratory hood, Linda Bartlett (photographer), National Cancer Institute, 1980 (NCI Visuals Online ID 2031). Public domain, via Wikimedia Commons. Reproduced photograph, shown under the Samso News glass on the article thumbnail.

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