Around the Helix: Cell and Gene Therapy Company Updates – March 25, 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. Angela Lek, PhD, on the Scientific Priorities that Shaped the 2026 MDA Conference

At the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, CGTLive sat down with Angela Lek, PhD, the chief research officer of MDA, to get her insight on highlights and themes from the conference. “We're always covering the cutting edge topic—what’s important to have conversations around—not just the readouts for clinical trials, but the really exciting technologies that are emerging around the corner,” Lek told CGTLive.

2. What should the medical community know about Deramiocel?

At MDA’s 2026 conference, Linda Marbán, PhD, the chief executive officer of Capricor, explained the mechanism behind the company’s investigational allogeneic cardiosphere-derived cell therapy deramiocel, which is currently under review by the FDA for Duchenne cardiomyopathy.

3. Focused Ultrasound Enables Nonsurgical Brain Delivery of scFvMC1 Gene Therapy That Reduces Cortical Tau Pathology in Tauopathy Mice

A preclinical study presented at the 2026 AD/PD International Conference on Alzheimer’s and Parkinson’s Diseases, held March 17-21 in Copenhagen, Denmark, demonstrated that MRI-guided focused ultrasound (FUS) can be used to direct an intravenously administered adeno-associated virus (AAV) carrying an antitau gene therapy to discrete brain regions in a mouse model of tauopathy — and that doing so produces regionally specific reductions in both soluble and insoluble tau species.

4. Indirect Comparison Suggests Intrathecal Onasemnogene Abeparvovec Comparable to Other SMA Therapies

New data from an indirect comparison analysis suggest that intrathecal onasemnogene abeparvovec (Itvisma; Novartis) demonstrated motor function improvements comparable with currently approved disease-modifying therapies for spinal muscular atrophy (SMA), including nusinersen (Spinraza; Biogen) and risdiplam (Evrysdi; Genentech).

5. Ocugen Gene Therapy Shows Reduction in Lesion Growth in Phase 2 Geographic Atrophy Trial

Ocugen reported 12-month data from the phase 2 ArMaDa clinical trial (NCT06018558) evaluating OCU410 (AAV5-RORA), an investigational single-dose subretinal gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration. In 51 patients randomized 1:1:1 to medium dose, high dose, or control, the medium dose cohort demonstrated a statistically significant 31% reduction in GA lesion growth versus control (p < .05) and 27% slower ellipsoid zone loss, a structural biomarker of photoreceptor integrity. No OCU410-related serious adverse events were reported. Ocugen anticipates initiating a phase 3 registrational trial in Q3 2026.

6. Encoded Therapeutics Reaches Accord With FDA on Pivotal Study Design for ETX101 in SCN1A+ Dravet Syndrome

Encoded Therapeutics announced FDA alignment on the pivotal study design for ETX101 (an AAV9-based gene regulation therapy) in SCN1A+ Dravet syndrome following a completed Initial Comprehensive Multidisciplinary Regenerative Medicine Advanced Therapy (RMAT) meeting. The 30-patient ENDEAVOR Part 2 trial—now initiated—will evaluate a single intracerebroventricular administration versus sham control over 52 weeks in children aged 6 months to younger than 4 years, with monthly seizure frequency reduction and Bayley-4 cognitive scores as primary and key secondary end points, respectively. An open-label expansion cohort (part 1B) will assess safety and preliminary efficacy in patients aged 4 to younger than 18 years. Enrollment completion and initial data readouts are expected by end of 2026 and 2027, respectively.