Cell therapy weekly: Eikonoklastes’ first-in-class gene therapy for ALS receives orphan drug designation
This week: Eikonoklastes’ first-in-class gene therapy for ALS receives orphan drug designation, Charles River increases cell therapy manufacturing capacity with expanded CDMO facility and AVITA Medical’s RECELL® System trial achieves co-primary endpoints.
The news highlights:
Eikonoklastes’ first-in-class gene therapy for ALS receives orphan drug designation
Eikonoklastes Therapeutics (OH, USA) has received orphan drug designation from the FDA for ET-101, a first-in-class AAV9-based gene therapy treatment for sporadic and familial amyotrophic lateral sclerosis (ALS). ALS is a progressive neurodegenerative disease affecting the motor neurons connecting the brain and spinal cord with no available cure. The investigational gene therapy employs targeted overexpression of Caveolin-1, a protein that regulates synaptic receptors essential for neuromuscular signaling and function, to induce neuroprotection and compensate for neurodegeneration caused by ALS.
Bruce Halpryn, chairman and CEO of Eikonoklastes Therapeutics stated: “The FDA’s Orphan Drug Designation for ET-101 reflects the compelling data underlying the ET-101 program and its potential as a 1st-in-class treatment option for both familial and sporadic ALS. Our goal is to significantly increase survival and improve quality of life for patients with this terrible disease.”
Charles River increases cell therapy manufacturing capacity with expanded CDMO facility
Charles River Laboratories (MA, USA) announced the expansion of its Memphis cell therapy contract development and manufacturing (CDMO) facility to enable more cell therapy developers to streamline and accelerate their programs. The expansion includes nine state-of-the-art processing suites with advanced cleanroom technology, which are fully compliant with international regulatory standards and good manufacturing practices.
Birgit Girshick, Corporate Executive Vice President and Chief Operating Officer at Charles River stated: “This expansion builds on the Memphis site’s demonstrable expertise and commitment to supporting cell and gene therapy clients from early target identification through clinical and commercial-scale manufacturing. We look forward to continuing close partnerships with our clients and supporting projects to bring these potentially curative therapies to patients.”
AVITA Medical’s RECELL® System trial achieves co-primary endpoints
AVITA Medical (CA, USA) has announced the achievement of co-primary endpoints following an updated analysis of data from its clinical trial evaluating the safety and effectiveness of its RECELL System for soft tissue repair. The technology platform has previously been approved by the FDA for the treatment of acute thermal burns in adults and children and harnesses autologous cellular therapies to create Spray-On Skin™ cells for tissue regeneration.
Jim Corbett, CEO of AVITA Medical stated: “These results reinforce the potential for RECELL to become a new standard of care for soft tissue repair. We look forward to sharing the soft tissue repair outcomes with the FDA via our PMA submission expected in December 2022.”
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