Cell therapy weekly: Exclusive license agreement for novel technology allowing safer spinal delivery of gene therapies
This week: Exclusive license agreement for novel technology allowing safer spinal delivery of gene therapies, Orphan drug designation granted to stem cell therapy for Duchenne Muscular Dystrophy and Exothera appoints new Chief Business Officer.
The news highlights:
Exclusive license agreement for novel technology allowing safer spinal delivery of gene therapies
AviadoBio (London, UK) and Neurgain Technologies (DE, USA) have entered into an exclusive license and collaboration agreement on a novel spinal delivery technology for gene therapies. Under the terms of the agreement AviadoBio, a gene therapy company focusing on the development of treatment options for neurodegenerative disorders, has been granted an exclusive worldwide license for the development, manufacture and licensing of Neurgain Technologies’ subpial delivery technology. The delivery system is minimally invasive and has the potential to provide precise and safe gene therapy delivery via injection under the membrane that surrounds the spinal cord.
Lisa Deschamps, CEO of AviadoBio, stated: “We are taking a neuroanatomy-led approach to drug delivery, which should have utility beyond ALS, and across neurological diseases that involve the spinal cord. Whether through our own research and development efforts or through partnerships, we want to enable all possibilities to deliver effective treatments to patients.”
Orphan drug designation granted to stem cell therapy for Duchenne Muscular Dystrophy
GIVI-MPC, a first-in-class induced pluripotent stem cell therapy for the treatment of Duchenne Muscular Dystrophy, has been granted an orphan drug designation by the US FDA. Duchenne Muscular Dystrophy is a rare, fatal neuromuscular genetic disease caused by a mutation in the gene for the protein dystrophin. There is no cure for Duchenne Muscular Dystrophy and, while gene therapies have provided some promise, they have not yet successfully reversed the underlying disease as they can only deliver approximately 30% of the large gene encoding dystrophin.
IPS HEART’s (TX, USA) stem cell therapy, GIVI-MPC, utilizes Givinostat to reprogram human induced pluripotent stem cells and has successfully generated new human skeletal muscle with 100% full-length dystrophin in both mouse and pig models.
Rauf Ashraf, CEO of IPS HEART, commented: “Given our successful pre-[investigational new drug] meeting with the FDA and our ongoing developmental efforts including GMP manufacturing, we believe that we will be the first IPS stem cell company with a disease-modifying therapy to advance both drugs into human clinical trials whereby all current drugs largely only provide symptomatic relief.”
Exothera appoints new Chief Business Officer
Darren Leva has been appointed as Chief Business Officer of Exothera (Jumet, Belgium), a CDMO specializing in viral vector-based cell and gene therapies and vaccines. Leva previously held the role of Head of Commercial Operations for Plasmid and Cell Therapy business units at Thermo Fischer Scientific (CA, USA). He will leverage his experience of scaling cell and gene therapy businesses along with his insights from various prior roles at start-ups and growth-stage companies to help Exothera accelerate its growth in viral vector manufacturing.
Commenting on his appointment, Leva said: “I have been truly impressed with the team at Exothera and their commitment to excellence across the organization. Combined with their state-of-the-art GMP facility, I am not surprised by their stellar track record with customer experience. As part of the Exothera family, I couldn’t be more humbled to help deliver cell and gene therapies to patients.”