VectorBuilder supported Lantu Biopharma's gene therapy drug for Menkes Disease to receive FDA Orphan Drug Designation
August 15, 2024, VectorBuilder Inc. - a global leader in gene delivery technologies, announced that its partner, Lantu Biopharma, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its gene therapy drug LTGT06 targeting Menkes Disease, a congenital disorder of copper metabolism caused by mutations in the ATP7A gene. VectorBuilder provided comprehensive gene delivery services, including gene delivery contract research organization (CRO) and gene drug contract development and manufacturing organization (CDMO) services, for the development of this therapy.
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. ODD grants the drug potential seven years of market exclusivity upon approval, tax credits for qualified clinical trials and exemption from user fees.
LTGT06 is an innovative gene therapy based on an AAV vector designed to treat congenital Menkes Disease. It contains the necessary AAV packaging sequences and the complete ATP7A coding sequence. Preclinical studies have demonstrated that LTGT06 achieves exogenous ATP7A protein expression in disease model mice and has shown no significant toxicity in safety evaluations conducted in mice and non-human primates. Furthermore, in clinical trials, the first patient administered with LTGT06 has shown no serious adverse reactions related to the therapy after two years of follow-up.
Dr. Bruce Lahn, Chief Scientist of VectorBuilder, stated: "We are thrilled to see Lantu Biopharma's innovative gene therapy, LTGT06, receive FDA Orphan Drug Designation. This milestone not only recognizes the significant preclinical research achievements of this project but also highlights the collaborative efforts of both our teams. We are confident in the future and will continue to fully support Lantu Biopharma in advancing the clinical development of LTGT06. Congratulations once again to Lantu Biopharma!"
About Lantu Biopharma
Lantu Biopharma is an innovative gene therapy company founded in 2020. Committed to helping and curing patients with unmet medical needs worldwide, our preclinical research and clinical translational teams with years of experience are dedicated to developing novel genetic medicines and therapies on our own technology platforms for the treatment of genetic disorders and degenerative diseases.
VectorBuilder is a global leader in gene delivery technologies. As a trusted partner in thousands of labs and biotech/pharma companies around the world, VectorBuilder is a one-stop shop for the design, development and optimization of gene delivery solutions from basic research to clinical applications. Its award-winning Vector Design Studio is a transformative innovation that allows researchers to easily design and order custom vectors online, freeing them from the tedious work of cloning and packaging vectors in the lab. The global company boasts high-throughput vector production capacity, vast vector and component inventories, one-on-one CRO solutions that include advanced AAV capsid engineering capabilities and state-of-the-art GMP facilities. With leading R&D and CDMO manufacturing capacity, the VectorBuilder team strives to provide the most effective gene delivery solutions and develop new tools for life sciences research and genetic medicine.