Around the Helix: Cell and Gene Therapy Company Updates – March 18, 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. Jerel A. Banks, MD, PhD, on Evaluating Gene Therapy BB-301 for OPMD-Related Dysphagia

At the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8-11 in Orlando, Florida, CGTLive® sat down with Jerel A. Banks, MD, PhD, the chief executive officer and chairman of the board of Benitec Biopharma, to learn more about the results a phase 1b/2a clinical trial (NCT06185673) evaluating Benitec Biopharma’s BB-301, an investigational adeno-associated virus (AAV) vector-based silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related dysphagia.

2. DTX301 Gene Therapy Lowers Ammonia Levels in Ornithine Transcarbamylase Deficiency at 36 Weeks

Interim results from the phase 3 Enh3ance clinical trial (NCT05345171) suggest that Ultragenyx’s investigational adeno-associated viral (AAV) vector-based gene therapy avalotcagene ontaparvovec (also known as DTX301) may reduce plasma ammonia levels in patients with ornithine transcarbamylase (OTC) deficiency, a rare inherited metabolic disorder associated with life-threatening hyperammonemia.

3. John F. Brandsema, MD, on the Real-World Use of Givinostat in DMD

At MDA’s 2026 conference, John F. Brandsema, MD, a child neurologist at Children’s Hospital of Philadelphia, spoke with CGTLive®’s sister site NeurologyLive® about his presentation on the now-completed phase 3 EPIDYS trial’s (NCT02851797) open-label extension (OLE; NCT03373968), which is still underway and continues to assess the long-term safety, tolerability, and efficacy of givinostat among patients who either finished or were screened but not randomized in previous studies.

4. Real-World Study Suggests Motor Gains in SMA After Switching to Onasemnogene Abeparvovec From Nusinersen or Risdiplam Treatment

Children with spinal muscular atrophy (SMA) who transitioned from SMN2-targeted therapies—nusinersen or risdiplam—to the gene therapy onasemnogene abeparvovec demonstrated modest, but clinically meaningful improvements in motor function in a real-world observational study conducted at a single center in Poland.

5. Gene Therapy RGX-202 Demonstrates Favorable Safety Profile in AFFINITY DUCHENNE Trial

An investigational AAV gene therapy, RGX-202, demonstrated favorable tolerability and robust microdystrophin expression in boys with Duchenne muscular dystrophy (DMD) in interim findings from the ongoing phase 1/2 AFFINITY DUCHENNE trial (NCT05693142). The therapy, designed to deliver an optimized microdystrophin gene using a NAV AAV8 vector, is being evaluated as a one-time treatment intended to address the underlying genetic cause of the disease.

6. iECURE's ECUR-506 Picked for FDA CMC Development and Readiness Pilot Program

iECURE announced that ECUR-506, its investigational in vivo targeted gene insertion therapy for neonatal-onset ornithine transcarbamylase (OTC) deficiency, has been selected for the FDA's Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot program, making it 1 of up to 9 of the programs chosen annually by the agency. Participation is intended to facilitate increased FDA engagement on CMC strategy ahead of a potential biologics license application submission. ECUR-506, which also holds regenerative medicine advanced therapy and fast track designations, is currently under evaluation in the phase 1/2/3 OTC-HOPE clinical trial (NCT06255782) in infant boys.