Around the Helix: Cell and Gene Therapy Company Updates – April 1, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. FDA Approves Higher Strength, More Effective Nusinersen Dose for Spinal Muscular Atrophy

Nearly 10 years after its original approval, the FDA has approved a new higher dose strength for nusinersen (Spinraza; Biogen) as a treatment for patients with spinal muscular atrophy (SMA). This new dosing regimen, which is comprised of 50 mg/5 mL and 28 mg/5 mL doses, is designed to deliver a higher concentration of drug through both the loading and maintenance dosing phases, leading to more efficacious results for patients with the disease.

2. Exploring One-Time Gene Regulation Therapy EXT101 in Dravet Syndrome: The Phase 1/2 POLARIS Program

This month NeurologyLive^®'s Clinical Trial in Focus covered the phase 1/2 POLARIS program for EXT101 (Encoded Therapeutics), an adeno-associated virus (AAV)-mediated candidate for gene regulation therapy, for the treatment of SCN1A+ Dravet syndrome (DS).

3. Ocugen Completes Dosing in Phase 2/3 GARDian3 Trial of OCU410ST for Stargardt Disease

Ocugen has completed enrollment and dosing of all 63 participants in the GARDian3 pivotal confirmatory clinical trial evaluating OCU410ST (AAV5-hRORA), an investigational single-dose subretinal modifier gene therapy for Stargardt disease, in less than 9 months, which the company noted was ahead of schedule. The multicenter, randomized, masked phase 2/3 study is assessing atrophic lesion size reduction at 12 months as its primary end point, with best corrected visual acuity and low luminance visual acuity compared to controls among key secondary end points. An interim analysis is planned for the third quarter of 2026, with topline results expected in the second quarter of 2027 and a biologic license application submission targeted for mid-2027.

4. AskBio Completes Enrollment in Phase 2 GenePHIT Trial of AB-1002 Gene Therapy for HFrEF

AskBio has randomized the final participant in GenePHIT (NCT05598333), its 173-patient phase 2 adaptive, double-blind, placebo-controlled trial evaluating AB-1002, an investigational AAV-based gene therapy for adults with nonischemic cardiomyopathy and New York Heart Association Class III heart failure symptoms with reduced ejection fraction (HFrEF). Administered as a single intracoronary infusion on top of standard of care, AB-1002 is designed to enable production of a modified form of inhibitor-1 protein to inhibit protein phosphatase 1, an enzyme associated with heart failure. Initial data from the trial are anticipated in the first half of next year.