Cell therapy weekly: FDA clears g-NK cell therapy for Phase I MS trial
This week: The US Food and Drug Administration (FDA; MD, USA) approved an Investigational New Drug application for a g-natural killer (g-NK) cell therapy in multiple sclerosis (MS), Vertex Pharmaceuticals (MA, USA) announced a reimbursement agreement with NHS England enabling patients with transfusion-dependent beta thalassemia to access the CRISPR/Cas9 gene-edited therapy, CASGEVY™ and MIP Discovery (Bedford, England) has shifted its focus to supporting viral vector analytics and purification and rebranded as Tozaro.
The news highlights:
- FDA clears Indapta’s g-NK cell therapy for Phase I MS trial
- CASGEVY approved for NHS use in England for beta thalassemia
- MIP Discovery rebrands as Tozaro to focus on viral vector innovation
FDA clears Indapta’s g-NK cell therapy for Phase I MS trial
The FDA has cleared Indapta Therapeutics’ (WA and TX, USA) Investigational New Drug application for its g-NK cell therapy IDP-023 in MS. This approval allows the company to commence a Phase I clinical trial to investigate the biologic effects of IDP-023 in people with MS. The therapy will be administered in combination with ocrelizumab, an anti-CD20 monoclonal antibody.
IDP-023 is currently being investigated in patients with non-Hodgkin’s lymphoma and multiple myeloma in a Phase I/II trial.
The g-NK cells utilized in IDP-023 arise naturally due to epigenetic changes triggered by exposure to cytomegalovirus. These cells are expanded from healthy donors and possess multiple mechanisms to kill target cells without requiring genetic engineering.
“I am excited to participate in this clinical trial because of the multiple potential mechanisms by which g-NK cells may impact the biology of MS,” said Lawrence Steinman, Professor of Medicine and Principal Investigator. “In addition to being able to achieve B cell depletion by combining with a B cell directed monoclonal antibody, g-NK cells have the ability to kill HLA-E expressing autoreactive T and B cells. In addition, g-NK cells have potent anti-viral activity, and therefore may also address the Epstein Barr Virus reservoir that contributes to the disease pathogenesis.”
CASGEVY approved for NHS use in England for beta thalassemia
The CRISPR/Cas9 gene-edited therapy CASGEVY will be available for eligible patients in England with transfusion-dependent beta thalassemia following a reimbursement agreement between NHS England and Vertex Pharmaceuticals. The reimbursement agreement follows the National Institute for Health and Care Excellence (London, UK) issuing positive guidance that endorses the use of CASGEVY within the NHS.
Vertex’s Senior Vice President, Ludovic Fenaux, stated: “Securing access to CASGEVY is a historic moment for people living with transfusion-dependent beta thalassemia who, for too long, have had limited options for this life-shortening disease. Through collaboration with NHS England and [the National Institute for Health and Care Excellence], we have reached this landmark agreement that recognizes the value a one-time treatment can provide to patients, their families and the health care system.”
MIP Discovery rebrands as Tozaro to focus on viral vector innovation
MIP Discovery has rebranded to Tozaro following the decision to transition the company’s focus to enhancing the development of viral vectors for cell and gene therapy applications. This rebrand comes after a Series A funding round in February that secured £7 million for the company.
Tozaro’s smart polymers are designed using computational chemistry. By analyzing whole viruses, unique surface epitopes are identified for binding by synthetic affinity reagents. The identified epitopes are then compared to a library of over 600 monomers to select optimal candidates for new smart polymer development. The chemical manufacturing process allows precise control over affinity and other features, providing scalable, safe and effective solutions for viral vector purification and analytics.
Guy Leaver, Interim CEO at Tozaro said, “We have already seen positive results with our first wave of lentivirus smart polymers designed for both analytics and capture chromatography. We are excited to see this trend continue as we head into the latter part of the year and expand our portfolio under our new identity, Tozaro.”
