Around the Helix: Cell and Gene Therapy Company Updates – March 4, 2026

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. AOC Del-desiran Demonstrates Target Engagement in Phase 1/2 MARINA Trial for Myotonic Dystrophy Type 1

An investigational antibody–oligonucleotide conjugate (AOC), delpacibart etedesiran (AOC 1001; del-desiran), demonstrated skeletal muscle delivery of small interfering RNA (siRNA) and reductions in mutant DMPK messenger RNA (mRNA) in adults with myotonic dystrophy type 1 (DM1) in the phase 1/2 MARINA clinical trial (NCT05027269). Results from the study were published in The New England Journal of Medicine.

2. The Potential of Bringing Hemophilia B Gene Therapy to Younger Patients

Steven W. Pipe, MD, discussed the potential of bringing hemophilia B gene therapy etranacogene dezaparvovec (marketed as Hemgenix) to younger patients. Currently the therapy is only FDA-approved for patients aged 18 years and older.

3. Rare Disease Day 2026: The Latest Updates in Rare Neurological Disease Therapy

Since 2008, the last day of February has been observed as Rare Disease Day by the patient and clinician communities. This year, CGTLive’s sister site NeurologyLive® put together a roundup of their latest news and interviews in several rare neurological diseases. The list includes items related to several gene therapy products for rare disease indications.

4. Investigating Gene Therapy Nexiguran Ziclumeran for ATTRv-PN: Phase 3 MAGNITUDE-2 Trial

For the month of February, as part of its Clinical Trial in Focus series, NeurologyLive spotlighted the phase 3 MAGNITUDE-2 trial (NCT06672237), assessing Intellia Therapeutics’ investigational gene therapy nexiguran ziclumeran (nex-z) for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic neuromuscular condition.

5. FDA Recommends uniQure Conduct Phase 3 Trial for Huntington Disease Gene Therapy

In a Type A meeting with uniQure regarding the path forward for the company’s investigational Huntington disease gene therapy AMT-130, the FDA reaffirmed its position previously expressed in a pre–biologics license application (preBLA) meeting last year that data from phase 1/2 clinical trials would not be sufficient to support a BLA for the product. Instead, the FDA “strongly recommended” in the Type A meeting the uniQure carry out a “prospective, randomized, double-blind, sham surgery-controlled” phase 3 clinical trial. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130,” Matt Kapusta, MBA, the CEO of uniQure, said in a statement.

6. FDA Lifts Hold on Intellia’s MAGNITUDE Trial for Nex-Z in ATTR-CM

The FDA has lifted a clinical hold on Intellia Therapeutics’ phase 3 MAGNITUDE clinical trial (NCT06128629) evaluating nex-z for the treatment of ATTR with cardiomyopathy (ATTR-CM). The hold was originally placed in October 2025 after a patient treated in MAGNITUDE experienced a grade 4 liver adverse event. The hold has been lifted as a result of Intellia aligning with the FDA on several mitigation measures for implementation in the remainder of the trial.