US DoD awards 2-year contract to CytoSorbents for development of Life-saving Universal Plasma

The HemoDefend-BGA Adsorber is not yet approved in the U.S. or elsewhere. This award is in support of the US Army Medical Research and Development Command (USAMRDC)/ Congressionally Directed Medical Research Programs (CDMRP)/ Combat Casualty Care Research Program (CCCRP/JPC-6) under Contract No. W81XWH20C0087. This follows the successful completion of Phase I and II STTR contracts of approximately $1.15 million with researchers at Penn State University, previously funded by the U.S. Army Medical Research Acquisition Activity (USAMRAA) and the U.S. Army Medical Research and Development Command (USAMRDC). This award is in addition to two recently awarded contracts to develop HemoDefend-BGA for universal plasma and whole blood transfusions announced earlier this year, including a $4.4 million contract from U.S. Army Medical Research Acquisition Activity (USAMRAA) and the Congressionally Directed Medical Research Programs (CDMRP), and a $2.9 million contract from the Defense Health Agency STTR Program/U.S. Army Medical Research and Development Command (USAMRDC) and CDMRP.  

CytoSorbents’ purification technologies are based on biocompatible, highly porous polymer beads that can actively remove toxic substances from blood and other bodily fluids by pore capture and surface adsorption. Its technologies have received non-dilutive grant, contract, and other funding of more than $38 million from DARPA, the Defense Health Agency, the National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), the U.S. Army, U.S. Special Operations Command (USSOCOM), the U.S. Air Force, Air Force Material Command (USAF/AFMC), the U.S. Department of Health and Human Services, and others. The Company has numerous products under development based upon this unique blood purification technology protected by many issued U.S. and international patents and multiple applications pending, including CytoSorb-XL™, HemoDefend™, VetResQ™, K+ontrol™, ContrastSorb, DrugSorb, and others. 

Axovant AXO-AAV-GM1 gets Rare Pediatric Disease Designation for GM1 Gangliosidosis

Axovant is on-track to report 6-month Stage 1 data from the low dose juvenile cohort (Type II) by the fourth quarter of 2020, and expects to initiate the high dose cohort which includes infantile (Type I) and juvenile (Type II) patients in the second half of 2020.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

GM1 Gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the β-galactosidase enzyme. Currently, there are no approved treatment options for GM1 Gangliosidosis.

AXO-AAV-GM1 is an investigational gene therapy that delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring β-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to improve β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.