Cell therapy weekly: One-time gene therapy for Hemophilia B gets approved by the FDA
This week: The US Food and Drug Administration (FDA; MD, USA) cleared the first-ever open Investigational New Drug (IND) for prime editing technology and issued ‘safe to proceed’ IND clearance for a cell therapy targeted at relapsed/refractory myeloid malignancies. Plus, a gene therapy for adults with Hemophilia B was also approved by the FDA.
The news highlights:
- First-ever ex vivo prime editing technology granted IND clearance from the FDA
- FDA approves one-time gene therapy, BEQVEZ™, for adults with Hemophilia B
- IND clearance issued by the FDA for cell therapy for relapsed/refractory myeloid malignances
First-ever ex vivo prime editing technology granted IND clearance from the FDA
The FDA has approved Prime Medicine’s (MA, USA) IND application for PM359, the company’s ex vivo product candidate designed to correct a prevalent disease-causing mutation of chronic granulomatous disease (CGD). CGD is a rare inherited hematologic disorder caused by mutations in any of the subunits comprising the NADPH oxidase complex, which is required for phagocytic cells, in particular neutrophils, to destroy many invasive microorganisms. PM359 uses prime editing to correct the ex vivo in autologous hematopoietic stem cells.
The approval of this IND application will enable the company to initiate its global Phase I/II clinical trial in the United States. Keith Gottesdiener, President and CEO of Prime Medicine, reflected on the data from the company’s preclinical studies and the significance of this advancement for next-generation gene editing, stating, “We believe PM359 has the potential to sufficiently correct a prevalent disease-causing mutation of CGD, leading to amelioration of disease for these patients.”
FDA approves one-time gene therapy, BEQVEZ™, for adults with Hemophilia B
Pfizer’s (NY, USA) one-time gene therapy, BEQVEZ™(fidanacogene elaparvovec-dzkt), has been approved by the FDA for adult patients with moderate to severe hemophilia B, a rare genetic bleeding disorder that prevents normal blood clotting due to a factor IX (FIX) deficiency. To be eligible for this treatment, a patient must be currently using FIX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes and not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid, as detected by and FDA-approved test.
BEQVEZ™ is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous prophylactic infusions of FIX that are often administered multiple times a week or multiple times a month.
“This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with hemophilia, with the delivery of a medicine that has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration,” said Aamir Malik, Chief US Commercial Officer and Executive Vice President, Pfizer.
IND clearance issued by the FDA for cell therapy for relapsed/refractory myeloid malignances
Genome writing company Replay (CA, USA) and The University of Texas MD Anderson Cancer Center (TX, USA) announced that the FDA has issued a ‘safe to proceed’ letter for the IND application for their engineered T-cell receptor natural killer (TCR-NK) cell therapy, PRAME TCR/IL-15 NK (SY-307).
Syena (CA, USA), an oncology-focused product company launched by Replay and MD Anderson, holds an exclusive license to Miltenyi Biotec’s (Bergisch Gladbach, Germany) PRAME (PReferentially expressed Antigen in MElanoma) TCR. Syena is developing the engineered PRAME-targeted TCR-NK from cord blood-derived NK cell therapy using Miltenyi’s PRAME TCR.
Adrian Woolfson, Executive Chairman, President, and Co-Founder of Replay, elaborated on the capability of PRAME. He stated that “PRAME is expressed at high levels in multiple different tumor types, making it a compelling target for engineered TCR-NK cancer immunotherapy. The recent IND clearance of our PRAME TCR/IL-15 NK (SY-307) program for AML and MDS represents a significant expansion of Syena’s growing pipeline of ‘off-the-shelf’ engineered TCR-NK therapies and complements our existing NY-ESO-1 targeted programs in myeloma and sarcoma.”
The Phase I/II open-label study will assess the safety, tolerability and preliminary efficacy of PRAME TCR/IL-15 NK (SY-307) in patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndromes.
