Around the Helix: Cell and Gene Therapy Company Updates – January 28, 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. Jeffrey Goldberg, MD, PhD, on the Potential of Cell and Gene Therapy in Glaucoma

This year for Glaucoma Awareness Month, which is observed annually in January by the patient and clinician communities, CGTLive® reached out to Jeffrey Goldberg, MD, PhD, a professor and chair of ophthalmology at the Byers Eye Institute at Stanford University, to learn more about how cell and gene therapy might help to address unmet needs in glaucoma.

2. FDA Lifts Clinical Hold on Intellia’s Trial for Gene Editing Therapy Nex-Z in ATTRv-PN

The FDA has lifted a clinical hold it had previously placed on Intellia Therapeutics’ phase 3 MAGNITUDE-2 clinical trial (NCT06672237), which is evaluating gene editing therapy nexiguran ziclumeran (nex-z, also known as NTLA-2001) for transthyretin amyloidosis with polyneuropathy (ATTR-PN). The hold was placed in October 2025 after a patient being treated in the separate phase 3 MAGNITUDE clinical trial (NCT06128629), which is evaluating nex-z for ATTR with cardiomyopathy (ATTR-CM), experienced a grade 4 cases of liver transaminases and increased total bilirubin.

3. Integrating Gene Therapy into Clinical Care for Hemophilia B

CGTLive® interviewed Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, about what clinicians thinking about incorporating the commercial hemophilia B gene therapy etranacogene dezaparvovec (marketed as Hemgenix) into their practice need to know.

4. TIL expansion and more in vivo CAR data in 2026?

ImmunoLogic cohosts Janna Minehart, MD, and Joseph Fraietta, PhD, discussed their thoughts on the potential of 2026 to bring major new data on in vivo chimeric antigen receptor (CAR) T-cell therapies and the expansion of tumor infiltrating lymphocyte (TIL) therapy outside melanoma.

5. FDA Places Holds on REGENXBIO MPS Trials

The FDA has placed clinical holds on REGENXBIO’s clinical trials for RGX-111, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat severe mucopolysaccharidosis Type I (MPS I, also known as Hurler syndrome), and clemidsogene lanparvovec (RGX-121), an investigational AAV vector-based gene therapy intended to treat mucopolysaccharidosis type 2 (MPSII, also known as Hunter syndrome). The holds are related to an intraventricular central nervous system tumor that occurred in 1 patient who was treated with RGX-111 in a phase 1/2 clinical trial.

6. What CGT indications do you think will make major progress in 2026?

Minehart and Fraietta discussed the potential of advancements for a Claudin-directed CAR-T therapy and Orca Bio’s Orca-T in the new year.