Cell therapy weekly: new integrated cell and gene therapy facility
This week: The US Food and Drug Administration (FDA; MD, USA) cleared an Investigational New Drug (IND) application for an amyotrophic lateral sclerosis (ALS) gene therapy, while a Phase II trial for an exon-skipping Duchenne muscular dystrophy (DMD) gene therapy yielded positive results. Plus, a new integrated cell and gene therapy facility opens in Genome Valley (Hyderabad, India).
The news highlights:
- FDA clears IND for ALS gene therapy
- New integrated cell and gene therapy facility opens in Genome Valley
- Positive Phase II results for DMD exon-skipping gene therapy
FDA clears IND for ALS gene therapy
The FDA has cleared SineuGene Therapeutics‘ (Beijing, China) IND application for SNUG01, a targeted intrathecal gene therapy for ALS that utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) capsid.
SNUG01 targets the TRIM72 (Tripartite Motif Protein 72) gene, identified as a multifunctional neuroprotectant by researchers at Tsinghua University’s Yichang Jia laboratory (Beijing, China). Preclinical studies have demonstrated SNUG01’s ability to reduce oxidative stress, restore mitochondrial homeostasis, inhibit neuroinflammatory cascades and enhance neuronal membrane repair.
By leveraging multiple neuroprotective mechanisms, SNUG01 offers a critical advantage for over 90% of ALS patients with sporadic (non-familial) disease. This multi-target approach positions SNUG01 as a first-in-class candidate for the majority of ALS cases.
SineuGene is now looking to conduct a multi-regional clinical trial to validate the safety and efficacy across a diverse range of ALS patients.
New integrated cell and gene therapy facility opens in Genome Valley
Bharat Biotech (Hyderabad, India) is expanding into cell and gene therapy and viral vector production at Genome Valley, broadening its focus from vaccines to regenerative and personalized treatments. Its new facility will develop therapies for cancer, hematological malignancies and genetic disorders such as hemophilia.
“Bharat aims to democratize gene therapies, traditionally considered prohibitively expensive and available primarily in developed nations or premium institutions,” said Raches Ella, Chief Development Officer, Bharat Biotech. “Our established expertise in producing viral vectors are essential for cell and gene therapy applications – the crucial material for anti-cancer and genetic disorders and robust clinical development abilities for [quality control] release. Additionally, it has capabilities to manufacture multiple platform products for various disease indications, including blood cancers, solid organ cancers and genetic disorders.”
Positive Phase II results for DMD exon-skipping gene therapy
Wave Life Sciences (MA, USA) has announced positive results from its Phase II FORWARD-53 trial of WVE-N531, an exon-skipping gene therapy being investigated in young males with DMD. DMD is an X-linked genetic neuromuscular disorder caused by out-of-frame deletions in the dystrophin gene. WVE-N531 is designed to promote the production of dystrophin protein to manage or slow disease progression.
After 48 weeks, WVE-N531 demonstrated a significant 3.8-second improvement in Time-to-Rise, the largest positive impact on muscle function observed compared to any other approved DMD treatment. The therapy was well-tolerated with no serious adverse events reported and led to reductions in muscle fibrosis, inflammation and necrosis, while enhancing functional outcomes. Additionally, dystrophin expression remained stable, averaging 7.8%, with 88% of boys achieving dystrophin levels above 5%.
Following feedback from the FDA, Wave plans to file a New Drug Application for accelerated approval in 2026 and submit Clinical Trial Applications for other DMD candidates targeting different exons.
“We expect WVE-N531 to become the first-line treatment of choice for boys amenable to exon 53 skipping, including the 40-50% in the US who are not being treated with approved exon skippers due in part to the burden of weekly infusions coupled with limited efficacy,” commented Paul Bolno, CEO of Wave Life Sciences.
