SineuGene Garners Orphan Drug Designation for ALS Gene Therapy
SNUG01 is intended to deliver a copy of the human TRIM72 gene to neurons via an rAAV9 vector.
SineuGene's SNUG01, an adeno-associated virus (AAV) vector-based gene therapy being evaluated for the treatment of amyotrophic lateral sclerosis (ALS), has been granted orphan drug designation by the FDA.1
SNUG01 is intended to deliver a copy of the human Tripartite Motif Containing 72 (TRIM72) gene to neurons via an rAAV9 vector. It is to be administered intrathecally. Reduction of oxidative stress via the scavenging reactive oxygen species, restoration of mitochondrial homeostasis, suppression of stress granule dysregulation, inhibition of neuroinflammatory cascades, and improved capacity for neuronal membrane repair are expected to be among potential benefits of TRIM72 expression based on preclinical research, and SineuGene states that these effects may help to slow the degeneration of motor neurons and thus work against ALS pathogenesis. The therapy is not intended to target a specific mutation related to ALS but rather to provide neuroprotection in a broad manner, and thus the company believes it may have the potential to benefit over 90% of patients with ALS who have sporadic disease.
SNUG01 has previously been evaluated in an investigator-initiated trial in China that was completed recently, according to SineuGene. The company noted that early indications of clinical efficacy and improvements in biomarkers were observed in this study, as were favorable safety and tolerability. The gene therapy product also previously received clearance of an investigational new drug application from the FDA in March of this year, and SineuGene plans to launch a phase 1/2a clinical trial for the candidate that will take place at sites in the United States and China.
”SineuGene will partner with a leading global consortium of academic and clinical institutions to conduct a multiregional clinical trial, expediting validation of SNUG01’s safety and therapeutic efficacy profile across a diverse ALS patient populations,” the company wrote in a March 24, 2025, press release.2
SNUG01 is one of many gene and cell therapy products currently being evaluated for the treatment of ALS by companies and academic institutions across the world. CGTLive® recently reviewed the state of the cell and gene therapy pipeline for ALS for ALS Awareness Month, which is observed annually in May by the patient and clinician communities.
Several other notable therapies covered in the pipeline review were uniQure’s AMT-162, an investigational AAV vector-based gene therapy for the treatment of ALS caused by mutations in superoxide dismutase 1 (SOD1-ALS); BrainStorm Cell Therapeutics’ debemestrocel (Nurown) cell therapy; and Cellenkos’ CK0803, a novel, allogeneic, neurotrophic, clinical-grade umbilical cord blood-derived cell therapy that consists of regulatory T-cells.
Notably, in January 2025, an independent data monitoring committee (IDMC) gave uniQure the green light to begin enrolling patients in the second dose cohort of the phase 1/2 EPISOD1 clinical trial (NCT06100276) for AMT-162.3 The recommendation to proceed with enrollment of the next dose cohort followed a review of 28 days-posttreatment data from patients who received AMT-162 in the first cohort, in which no significant safety concerns were observed.
In July 2024, BrainStorm reached alignment with the FDA on chemistry, manufacturing, and control (CMC) aspects of a phase 3b clinical trial evaluating Nurown for the potential treatment of ALS.4 The news came a couple of months after the company announced that it had reached an accord with the FDA on a phase 3b trial design for debemestrocel, the end points of which would support a biologics license application approval for the therapy.
In May 2024, CGTLive covered the phase 1 REGALS clinical trial (NCT05695521) evaluating CK0803 as part of our Clinical Trial in Progress series.5 The first patient was dosed in the REGALS trial in May 2023.
