ALS Awareness Month 2025: A Review of the Cell and Gene Therapy Pipeline

According to the ALS Association, a person is diagnosed with amyotrophic lateral sclerosis (ALS), progressive neurodegenerative disease affecting brain and spinal cord nerve cells, every 90 minutes. Diagnosis with ALS can take a substantial period of time—months or years—and although about 10% of patients can have their condition linked to an inherited mutated gene, 90% of cases are not known to be related to any genetic cause or family history. There is currently no cure for the condition.

An important area of interest for new therapeutic development in ALS is advanced modalities, such as cell therapy, gene therapy, and RNA-based therapies. A number of companies and academic institutions are now pursuing the development of such advanced treatments, with several clinical trials currently active. In honor of ALS Awareness Month, observed annually in May by the patient and clinician communities, CGTLive® is taking a look at the pipeline of advanced therapeutics currently in clinical development for the condition. Click the "READ MORE" buttons for more details and information about each item.

uniQure Makes Progress in Trial for SOD1-ALS Gene Therapy AMT-162

January 30, 2025 — An independent data monitoring committee (IDMC) has given uniQure the green light to begin enrolling patients in the second dose cohort of the phase 1/2 EPISOD1 clinical trial (NCT06100276), which is evaluating AMT-162, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of ALS caused by mutations in superoxide dismutase 1 (SOD1-ALS).

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 light of the recommendation, uniQure stated that it anticipates enrollment activities for the second dose cohort will begin before the end of the first quarter of this year.

BrainStorm’s New Trial May Support ALS Cell Therapy BLA

July 10, 2024 — BrainStorm Cell Therapeutics has reached alignment with the FDA on chemistry, manufacturing, and control (CMC) aspects of a phase 3b clinical trial evaluating debemestrocel (Nurown) cell therapy for the potential treatment of ALS.

"This in-person Type C meeting with the FDA was an important step as we finalize preparations for our pivotal Phase 3b trial of NurOwn. We are very pleased with the outcome and are now aligned with the FDA on resolving all previously outstanding CMC questions," Chaim Lebovits, President & Chief Executive Officer, BrainStorm, said in a statement. "As with any cell therapy products, there are additional complexities in the manufacturing process, and it is important that we have alignment in advance with all the relevant groups within the FDA. We are committed to working closely with the FDA and to helping the ALS community, and are excited to move forward with the Phase 3b trial. Our ultimate goal is to achieve approval for NurOwn in order to address the unmet needs of patients."

ALS REGALS Trial Continues to Evaluate Cellenkos’ CK0803 Treg Therapy

May 13, 2024 — CK0803 is a novel, allogeneic, neurotrophic, clinical-grade umbilical cord blood-derived cell therapy that consists of regulatory T-cells developed by Cellenkos. The cells have a high cell surface expression of CD11a that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia in patients with ALS.

CK0803 is being evaluated in patients with ALS in the phase 1 REGALS trial (NCT05695521). The first patient was dosed in the REGALS trial in May 2023. Cellenkos announced that the trial was proceeding to dose its second cohort following a positive recommendation by the Data Safety Monitoring Board (DSMB) in March 2024.

Ractigen Therapeutics' RAG-17

January 8, 2025 — Ractigen Therapeutics is currently evaluating RAG-17, a small interfering RNA (siRNA) therapy, for the treatment of SOD1-ALS in a phase 1 clinical trial (NCT06556394). According to the clinicaltrials.gov page, the study launched on December 24, 2024, and recruitment is ongoing.

Hong Chen's VGN-R13

March 17, 2025 — Hong Chen, Tongji Hospital is currently evaluating VGN-R13, an AAV vector-based gene therapy, for the treatment of ALS in an early phase 1 clinical trial (NCT06849609). According to the clinicaltrials.gov page, the study launched on Janauary 16, 2025, and recruitment is active.

Regeneron's ALN-SOD

February 26, 2025 — Regeneron Pharmaceuticals is currently evaluating ALN-SOD, an RNA interfering (RNAi) therapy, for the treatment of SOD1-ALS in a phase 1 clinical trial (NCT06351592). According to the clinicaltrials.gov page, the study launched on August 28, 2024, and recruitment is active.

Peking University Third Hospital's SNUG01

October 16, 2024 — Peking University Third Hospital is currently evaluating SNUG01, an AAV vector-based gene therapy, for the treatment of ALS in an early phase 1 clinical trial (NCT06645197). According to the clinicaltrials.gov page, the study launched on October 16, 2024, and recruitment is active.

Rapa Therapeutics' RAPA-501

May 1, 2025 — Rapa Therapeutics is currently evaluating RAPA-501, an autologous T-stem cell therapy, for the treatment of ALS in a phase 2/3 clinical trial (NCT04220190). According to the clinicaltrials.gov page, the study launched on January 2, 2025, and recruitment is active.

Corestemchemon's Lenzumestrocel

January 10, 2025 — Corestemchemon is currently evaluating Lenzumestrocel (Neuronata-R Inj.), an autologous bone marrow derived mesenchymal stem cell therapy, for the treatment of ALS in a phase 3 clinical trial (NCT04745299). According to the clinicaltrials.gov page, the study launched on March 23, 2021, and is active, but not recruiting new patients.

Novabio's NP001 Cell Injection

May 25, 2025 — Novabio Therapeutics is currently evaluating NP001, an autologous human polyclonal regulatory T-cell injection, for the treatment of neurodegenerative diseases including ALS in a phase 1 clinical trial (NCT06671236). According to the clinicaltrials.gov page, the study launched on November 21, 2024, and is actively recruiting.

Shanghai East Hospital's iPSC-MNP

January 9, 2025 — Shanghai East Hospital is currently evaluating a human induced pluripotent stem cell derived motor neuron precursor (iPSC-MNP) therapy for the treatment of ALS in a clinical study (NCT06765564). According to the clinicaltrials.gov page, the study launched on March 13, 2024, and is actively recruiting.

REFERENCE
1. What is ALS? ALS Association. Website. Accessed May 29, 2025. https://www.als.org/understanding-als/what-is-als