Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, announced today that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Glioblastoma (GBM).
Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner. If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review.
GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. RZ-001, the RNA replacement enzyme-based cancer gene therapy for the treatment of GBM, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. RZ-001 has demonstrated very promising responses in preclinical studies in which an enhanced anti-cancer efficacy and a higher survival rate was observed.
“We are proud to receive Fast Track Designation from the FDA,” said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics. “This designation signifies an important milestone in developing a safe and effective treatment for patients with GBM who are in need of new therapeutic options.”
Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial will investigate the safety, tolerability, and efficacy of RZ-001 in patients with GBM. Subjects will be treated with RZ-001 according to the planned dose escalation design.
As a biopharmaceutical company founded in the laboratory of Professor Seong-Wook Lee, Dankook University Department of Bioconvergence Engineering, Rznomics is researching with the goal of developing new RNA-based gene therapeutic bio-drugs for cancer and incurable diseases. Rznomics’ core platform technology is based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus, selectively inducing therapeutic gene activity in cells expressing the target RNA.
Rznomics’ lead candidate (RZ-001) is a treatment for Hepatocellular Carcinoma & Glioblastoma (Phase 1/2a IND approval from both in South Korea and in the U.S.), with other treatments for Alzheimer’s disease and hereditary retinal dystrophy (RD)
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
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