GC Biopharma Corp. announced today that GC1126A, its drug candidate for Thrombotic Thrombocytopenic Purpura (TTP), received an Orphan Drug Designation on September 27 from the U.S. Food and Drug Administration (FDA).
When designated as an orphan drug, there are incentives such as tax credits for clinical development costs and exemptions from PDUFA user fees Initial Pediatric Study Plan. Additionally, upon receiving marketing approval, a 7-year period of market exclusivity is granted.
TTP is a rare blood disorder that affects approximately 3 to 11 individuals per 1 million people. It is a life-threatening condition characterized by the formation of small blood clots throughout the body, obstructing blood flow to critical organs, including the brain and heart. If left untreated, TTP can result in a mortality rate as high as 90%. The development of TTP is associated with a deficiency of the proteolytic enzyme ADAMTS13 in the patient’s body (congenital TTP) or the sudden production of antibodies that inhibit the enzyme activity (immune-mediated TTP).
GC1126A is a novel ADAMTS13 mutein designed to evade autoantibodies with an extended half-life. As presented at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in June, non-clinical data demonstrated efficacy of the drug candidate in disease models while maintaining higher activity levels when compared to existing treatments or wild-type ADAMTS13.
Speaking on this achievement, GC Biopharma stated, “We are dedicated to collecting data to develop a Best-in-Class treatment for such rare disorders. We remain committed to our mission of providing patients with new treatment options by continuously developing innovative drugs.”
About GC Biopharma Corp.
GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma Corp. is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.
This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma’s management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.
Check out our AAV CDMO service to expedite your gene therapy research
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.
Another Two-Plasmid AAV Gene Therapy Enters Clinical Trial: Gene Vector Biotechnology’s JWK001 Pioneers in Treating nAMD
On November 30, 2023, Gene Vector Biotechnology Co., Ltd. in Chengdu, China, received approval from the National Medical Products Administration's Center for Drug Evaluation (CDE) for the clinical trial of JWK001, a Class I gene therapy drug for treating neovascular...
Accelerating Gene Therapy Production: Combining next-generation AAV vectors and artificial intelligence (AI)
Recently, the France-based WIDGeT consortium emerged from a collaboration between Sanofi, WhiteLab Genomics, the TaRGeT Laboratory at Nantes University, and Institut Imagine. Scientists and companies in this consortium hope to speed up the development of gene...
First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin On track for pivotal dose determination and initiation of pivotal program in 2024...
Hodgkin lymphoma, nodular lymphocyte predominant (high-power view) Credit: Gabriel Caponetti, MD./Wikipedia/CC BY-SA 3.0The University of Pennsylvania plans to continue offering CAR-T therapy, a cancer treatment pioneered at Penn, after the Food and Drug...
Plasmids GMP Services
Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.
AAV GMP Services
Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.