RAG-01 is a new therapy aimed at treating non-muscle invasive bladder cancer (NMIBC).
This regulatory milestone enables the commencement of clinical trials in the US.
NMIBC accounts for 50-80% of all bladder cancer cases. Despite the use of standard treatments such as transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, patients frequently experience recurrence, with rates estimated at 50-70% within the first five years post-treatment.
Ractigen said RAG-01 is at the forefront of bladder cancer therapy, being the first saRNA therapy designed to specifically activate the p21 tumour suppressor gene.
This gene plays a crucial role in controlling cell cycle progression and is instrumental in halting the proliferation of cancer cells.
Ractigen’s proprietary LiCOTM delivery technology is used to administer the drug through intravesical instillation.
Preclinical studies have demonstrated significant tumour suppression in mouse orthotopic bladder cancer models, the company noted.
The Phase I clinical trial of RAG-01 in Australia is currently underway, with the first three patients successfully enrolled and dosed.
Ractigen Therapeutics founder and CEO Long-Cheng Li said: “FDA IND approval for RAG-01 is a major achievement for Ractigen and a significant advancement for saRNA technology worldwide.
“This first-in-class saRNA therapy harnesses the power of RNAa to target the p21 gene, offering a promising new option for patients with limited treatment choices. This approval validates the potential of RAG-01 as a leading saRNA therapy and strengthens our position as innovators in RNA-based treatments.”