Further elevating its clinical promise, RRG-OTOF received the Orphan Drug Designation (ODD) from the US FDA on December 21, 2023. This distinction marks RRG-OTOF as the first Chinese independently developed in vivo gene therapy product for OTOF-mediated hearing loss to be recognized by the FDA.
PackGene Biotech, a strategic partner in this endeavor, played a crucial role by providing vital virus samples for the project. This collaboration has been instrumental in propelling the development of RRG-OTOF, contributing significantly to the advancement of gene therapies for hereditary deafness and bridging the gap from research to clinical application.
OTOF-mediated hearing loss, also known as DNFB9 congenital deafness, is an autosomal recessive disorder caused by mutations in the OTOF gene. The gene encodes otoferlin, a key protein essential for the function of cochlear hair cells in responding to sound stimuli. The dual-vector approach of RRG-OTOF, an innovative gene therapy, delivers the otoferlin gene to the cochlea’s inner hair cells. This single administration aims to restore auditory function by ensuring the long-term expression of functional otoferlin protein.
This breakthrough in gene therapy for hearing loss marks a significant step forward in treating genetic disorders and demonstrates the power of international collaboration in advancing medical science and patient care.
Related News
A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What’s Possible in Gene Therapy with Help from Weill Cornell Medicine
HOUSTON, TX – Two Australian parents, faced with the unimaginable prospect of losing their 14-month-old daughter to the ultra-rare neurological disease Hereditary Spastic Paraplegia Type 56 (SPG56), sold their home and everything they owned to save their daughter....
NeuExcell’s NXL-004 Secures FDA Orphan Drug Designation for Glioma Treatment
On December 7, 2023, Beijing time, NeuExcell Therapeutics announced that its proprietary AAV gene therapy product, NXL-004, designed for treating malignant glioma, has been awarded Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This...
PackGene Biotech Receives Exclusive C+ Round Investment from SDIC Fund, and Hosts a CGT Industry Salon to Discuss Global Strategies
December 1, 2023, PackGene Biotech, a frontrunner in gene therapy delivery, has successfully closed a landmark C+ funding round, securing over 100 million RMB exclusively from SDIC Fund Management Co., Ltd. This funding, a remarkable accomplishment in the current...
Tauopathy’s Role in Aβ Oligomer Formation: New Insights from Transgenic Primate Models on Alzheimer’s Pathogenesis
Tauopathy, characterized by protein tau and Aβ oligomer accumulation, is a significant aspect of Alzheimer's disease. The causal relationship between these accumulations and neurodegeneration remains unclear despite their links to various cognitive and movement...
Related Services
Plasmid CDMO Services
We provide high quality plasmids that are ideal for any phase of the drug development cycle.
READ MORE
AAV cGMP Manufacturing
READ MORE
AAV Capsid Engineering
Proven technology paving your path to effective therapies for cancer or genetic disorder
READ MORE
