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LIfT BioSciences’ N-LIfT Cell Therapy Successfully Infiltrates Pancreatic Tumours.

LIfT BioSciences has successfully demonstrated migration to the human pancreatic tumor site in a mice model using its cell therapy, N-LIfT.  

The tumor was grown over two weeks before the mice were given a single dose of two billion N-LIfT cells via the tail-vein.

Neutrophil Only Leukocyte Infusion Therapy (N-LIfT) is produced from the stem cells of exceptional cancer-killing donors. It is allogeneic and utilizes the innate immune system of these exceptional donors to directly attack the tumor and recruit the patients’ immune system to join the attack.

Pancreatic cancer is known as the most difficult to treat, with only five-year survival rates of under 5% in most countries. Pancreatic cancer is often diagnosed late and the site of the tumor is difficult to infiltrate because it is surrounded by a network of non-malignant cells that act as a protective barrier.

(Source: Global News Wire, 2020)

Ultragenyx to Build Large-scale Gene Therapy Manufacturing Facility.

Ultragenyx Pharmaceutical recently announced its plans to build a new large-scale gene therapy manufacturing facility in Bedford, Massachusetts. The new facility will enable in-house manufacturing of clinical-stage adeno-associated virus (AAV)- based gene therapies for various diseases.

Ultragenyx will use both of its gene therapy manufacturing platforms at the new facility: the HeLa producer cell line (PCL) platform which enables large 2,000-liter commercial-scale manufacturing and their HEK293 transient transfection system.

The planned Phase I facility will cover an area of 100,000 square feet and provide the important internal capacity to develop and manufacture a supply of Ultragenyx’s gene therapies for both clinical stage and approved products. The facility will be able to support two independent manufacturing suites with a capacity of 30 runs per year.

The facility is expected to be completed in 2023.

(Source: Globe News Wire, 2020)

Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy 

Axovant Gene Therapies recently announced that the U.S. Food and Drug Administration (FDA) has lifted its clinical hold and cleared the Investigational New Drug (IND) Application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease. 

AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff diseases, which are rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode β-Hexosaminidase A, HEXA, and HEXB

AXO-AAV-GM2 delivers two vectors encoding the HEXA and HEXB genes directly to the central nervous system to produce a fully functional β-Hexosaminidase A enzyme. 

AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA.

The next phase of the study will include both infantile and juvenile participants with GM2 gangliosidosis. The trials are divided into two parts, one will consist of (1) a dose-ranging cohort evaluating the safe and efficacious dose of the gene therapy, followed by (2) an efficacy cohort, both of which form the basis of the registrational program.

(Source: Globe News Wire, 2020)