Therapies to Watch in 1H 2026

As 2026 kicks off, CGTLive® is taking a look at some of the notable investigational cell and gene therapy candidates in the pipeline. Whether they target a disease area with high unmet need, have inventive mechanisms, or are otherwise notable, here are a few of the investigational products we are keeping our eye in the first half of this year.

Click the “read further” links for past coverage of these therapies.

RGX-202

Indication: Duchenne muscular dystrophy (DMD)

Patients Treated With REGENXBIO’s DMD Gene Therapy RGX-202 Exceed Expected Disease Trajectory on NSAA

January 15, 2026 — Among 4 patients treated with the pivotal dose of REGENXBIO’s RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), in the phase 1/2 portion of the AFFINITY DUCHENNE clinical trial (NCT05693142), all 4 surpassed expected disease trajectory on the North Star Ambulatory Assessment (NSAA) with utilization of the Collaborative Trajectory Analysis Project (cTAP) disease progression model at 18 months posttreatment.

Specifically, REGENXBIO noted that the patients treated with the gene therapy product showed an average improvement of 7.4 points in comparison to cTAP. The company also pointed out that at 12 months posttreatment, the 4 treated patients had improved an average of 6.6 points in comparison to cTAP.

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Deramiocel

Indication: Duchenne muscular dystrophy (DMD) cardiomyopathy

In Response to FDA’s Release of Full CRL for Deramiocel, Capricor Publishes Full Response to the Agency

September 9, 2025 — Following the FDA’s public release of the full complete response letter (CRL) that it issued to Capricor Therapeutics for the company’s biologics license application (BLA) for deramiocel, an investigational allogeneic cardiosphere-derived cell therapy intended for the treatment of DMD cardiomyopathy, Capricor has responded by publishing its full response to the agency.

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website. Furthermore, the FDA did not publish Capricor’s response to the CRL, which, according to the company, provides clarifications on the feedback received and insight on its proposed plans to address remaining issues. As such, Capricor published its response to the CRL on its own website.

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DTX401

Indication: Glycogen storage disease type Ia (GSDIa).

Ultragenyx Finishes Rolling Submission of BLA for Glycogen Storage Disease Gene Therapy DTX401

January 5, 2026 — Ultragenyx has completed its submission of a rolling BLA to the FDA for DTX401, an investigational AAV serotype 8 (AAV8) vector gene therapy expressing the human G6PC gene that is intended to treat Glycogen Storage Disease Type Ia (GSDIa).

The BLA is supported by 96-week data from the randomized, placebo-controlled phase 3 GlucoGene clinical trial (NCT05139316). Ultragenyx noted that the nonclinical and clinical modules of the BLA were submitted to the FDA in August 2025; it has now also completed submission of the chemistry, manufacturing, and controls (CMC) module of the BLA, thus finishing the BLA package.

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SGT-212

Indication: Friedreich ataxia (FA)

Phase 1b Trial for Solid Biosciences’ Friedreich Ataxia Gene Therapy SGT-212 Doses First Patient

January 15, 2026 — The first patient has been dosed in the first-in-human phase 1b FALCON clinical trial (NCT07180355), which is evaluating Solid Biosciences’ SGT-212, an investigational gene therapy product, for the treatment of FA. The open-label, dose-finding study will include patients with FA aged 18 to 40 years.

SGT-212 is intended to address neurologic, cardiac, and systemic manifestations of the disease through a dual-route administration. The cerebellar dentate nuclei will first be targeted with an intradentate nucleus (IDN) infusion of SGT-212, and patients will also receive a subsequent intravenous infusion of the gene therapy. Pending patient enrollment, Solid expects to announce initial data from the trial in the second half of 2026.

“We want to recognize the extraordinary leadership and partnership of the Friedreich’s Ataxia Research Alliance (FARA), whose ongoing guidance and support continue to shape and strengthen our clinical program,” Solid wrote in an open letter to the FA Community. “We also wish to acknowledge the many scientists, clinicians, and partners whose collaboration made the development of SGT-212 and this dual-route gene therapy approach possible. This work reflects our shared determination to advance clinically meaningful therapies that address the urgent unmet needs of this community – to improve both survival and quality of life for people living with FA.”

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Isaralgagene civaparvovec

Indication: Fabry disease

Sangamo Starts Putting BLA for Fabry Disease Gene Therapy Isa-Vec in Front of FDA

December 19, 2025 — Sangamo Therapeutics has begun submission of a rolling BLA application to the FDA for investigational Fabry disease gene therapy isaralgagene civaparvove (isa-vec, ST-920).

The BLA is supported by data from the phase 1/2 STAAR clinical trial (NCT04046224). Across all patients who were treated in STAAR, a positive mean annualized estimated glomerular filtration rate (eGFR) slop was observed at 52 weeks posttreatment. Notably, in an October 2025 meeting with Sangamo, the FDA onfirmed its agreement that eGFR slope can be used as an end point for the support of an accelerated approval pathway for isa-vec. Shortly after this meeting, in November 2025, Sangamo received a green light from the FDA to begon submitting a rolling BLA for the therapy.

“The initiation of our BLA submission marks an important milestone for Sangamo and for Fabry patients in need,” Nathalie Dubois-Stringfellow, PhD, the chief development officer of Sangamo, said in a statement. “The compelling data from our STAAR study shows the potential of ST-920 to provide safe and long-lasting clinical benefits to a wide range of Fabry disease patients. We look forward to working with the FDA as we continue to advance the regulatory process.”

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