NADPH

Prime Medicine Presents Preclinical Data Demonstrating Ability of PM359 to Efficiently, Reproducibly and Durably Correct Causative Mutation of Chronic Granulomatous Disease (CGD)

Retrieved on: 
onsdag, maj 8, 2024
FDA, Process, Chronic granulomatous disease, Mutation, Mouse, Cell, CGD, Food, Hematology, Life, U.S. FDA, IND, Neutrophil, NADPH, Patient, Society, Immunology, Stem cell, Fungus, Health, NCF1, Pharmaceutical industry

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of its ex vivo Prime Editing program, PM359, to correct a common disease-causing mutation of chronic granulomatous disease (CGD). The data will be presented today at an oral presentation during the American Society of Cell & Gene Therapy 27th Annual Meeting in Baltimore. Prime Medicine recently announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for PM359 for the treatment of CGD.

Key Points: 
  • The data will be presented today at an oral presentation during the American Society of Cell & Gene Therapy 27th Annual Meeting in Baltimore.
  • Prime Medicine recently announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for PM359 for the treatment of CGD.
  • Prime Medicine is advancing an ex vivo Prime Editing program, PM359, that aims to correct the predominant mutation in NCF1 in CGD patient CD34+ hematopoietic stem cells (HSCs) and restore NADPH oxidase function.
  • In today’s presentation at ASGCT, Prime Medicine highlighted data from a series of in vivo non-clinical studies using human CGD patient CD34+ HSCs.

Prime Medicine to Present New Preclinical Data Highlighting Broad Potential of Prime Editing Technology at ASGCT 26th Annual Meeting

Retrieved on: 
onsdag, maj 17, 2023
Chronic granulomatous disease, DNA, TRAC, Gene, CD19, Neoplasm, Bone marrow, Investigational New Drug, Deletion, Fungus, CD34, Stem cell, Health, Chromosomal translocation, Spleen, Genome, NADPH, Annual general meeting, Cancer, Cell, CGD, American Society of Gene and Cell Therapy, Society, Disease, NCF1, Vaccine, B2M

CAMBRIDGE, Mass., May 17, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the presentation of new preclinical data that further demonstrated the potential of Prime Editing to correct the causative mutation of chronic granulomatous disease (CGD) and preclinical data that showcased the potential application of the Prime Editing Assisted Site-Specific Integrase Gene Editing (PASSIGE™) platform to generate multiplex-edited CAR-T cells for the treatment of certain cancers and immune diseases. The data are being presented today during the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023, in Los Angeles, California.

Key Points: 
  • The data are being presented today during the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023, in Los Angeles, California.
  • “We are very pleased to present these new data for our CGD program and PASSIGE platform today at ASGCT, which underscore our belief in the breadth and potential of Prime Editing to offer curative treatments for many diseases,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine.
  • Further, while the benefits of autologous CAR-T therapies are well established, their full potential is often hindered by manufacturing and delivery challenges.
  • Prime Medicine has previously shared data from the CGD program that demonstrated the ability of Prime Editing to correct a CGD causative mutation in CD34+ cells ex vivo.